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FOR IMMEDIATE RELEASE
Lundbeck’s SABRIL® (vigabatrin) Now Approved by U.S. FDA as an Adjunctive Treatment Option for Children 10 and older with Refractory Complex Partial Seizures
Deerfield, Ill., October 28, 2013 – The U.S. Food and Drug Administration (FDA) approved SABRIL (vigabatrin) as add-on therapy for the treatment of refractory complex partial seizures (CPS) in children 10 years of age and older who have inadequately responded to several other treatments and if the possible benefit outweighs the risk of vision loss.1 This approval expands upon the age range of SABRIL’s previous indication as adjunctive therapy for adults with refractory CPS. SABRIL is not indicated as a first-line agent for refractory CPS.
Of the more than two million Americans affected by epilepsy,2 approximately 35 percent have CPS, which originates from a single region of the brain and can cause impaired consciousness.3 Approximately 30 to 36 percent of those with CPS continue to have seizures despite trying multiple therapies, and are considered to have refractory CPS.4,5,6
“It is crucially important for people with challenging seizures like refractory CPS to not give up and continue striving for improved seizure management, and this expanded Sabril indication provides another consideration for the treatment of those ten and older with refractory CPS,” said Philip Gattone, president and CEO of the Epilepsy Foundation. “We encourage people living with such challenging seizures and their loved ones to have ongoing conversations with their doctor about available options to help manage this intractable seizure disorder.”
When SABRIL was first approved in 2009, a patient registry was established to collect information on all patients who are prescribed SABRIL. To date, more than 5,600 patients have been treated with SABRIL, a substantial number of whom have been treated for refractory CPS.7 In evaluating whether to start SABRIL, doctors, patients and their caregivers work together to assess the risk of permanent vision loss versus the benefit of seizure reduction. There are other serious risks associated with SABRIL. Please see the important safety information below for more details.
“With so many children still having seizures due to refractory CPS, we are very pleased that the FDA has approved SABRIL for patients 10 and older who may benefit from a new add-on treatment option,” said Amy Magro, Director of Epilepsy Marketing at Lundbeck. “For those caring for a child as young as 10, we hope this new indication provides encouragement to speak with their child’s doctor about the risks and potential benefits of adding SABRIL for refractory CPS.”
In addition to its refractory CPS indication, SABRIL is approved for use in babies one month to two years of age with infantile spasms if the possible benefit outweighs the potential risk of vision loss.
For more information, please visit www.SABRIL.net.
About SABRIL® (vigabatrin) 1
SABRIL is a prescription oral antiepileptic drug developed in the United States by Lundbeck. SABRIL is available in 500-mg tablets or 500-mg packets of powder for oral suspension. Because of the risk of permanent vision loss, SABRIL is available only through a restricted program under a REMS called the SHARE Program. (1-888-45-SHARE).
SABRIL (vigabatrin) is a prescription medicine used with other treatments in adults and children 10 years of age and older with refractory complex partial seizures (CPS), who have not responded well enough to several other treatments, and if the possible benefits outweigh the risk of vision loss. SABRIL should not be the first medicine used to treat CPS.
SABRIL (vigabatrin) is a prescription medicine used in babies, 1 month to 2 years old, with infantile spasms (IS), if the possible benefits outweigh the possible risk of vision loss.
Important Safety Information
WARNING: VISION LOSS
See Medication Guide and full Prescribing Information for complete information
In all people who take SABRIL:
• You are at risk for vision loss with any amount of SABRIL
• Your risk of vision loss may be higher the more SABRIL you take daily and the longer you take it
• It is not possible for your healthcare provider to know when vision loss will happen. It could happen soon after starting SABRIL or any time during treatment. It may even happen after treatment has stopped.
Please see SABRIL Medication Guide, full Prescribing Information including Boxed Warning, and Instructions for Use; go to www.sabril.net, or call toll-free 1-888-45-SHARE (1-888-457-4273).
You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.
About Lundbeck in the U.S.
A wholly owned subsidiary of H. Lundbeck A/S of Denmark, Lundbeck in the United States is headquartered in Deerfield, Illinois, and is committed to providing innovative specialty therapies that fulfill unmet medical needs of people with central nervous system (CNS) disorders, including several therapies for people with challenging seizure disorders.
With a special commitment to the epilepsy community, Lundbeck actively supports and participates in hundreds of community-based initiatives. Learn more about our epilepsy community programs at http://www.lundbeck.com/us/our-commitment/community-involvement.
About H. Lundbeck A/S
Lundbeck is a global pharmaceutical company highly committed to improving the quality of life of people living with brain diseases. For this purpose, Lundbeck is engaged in the entire value chain throughout research, development, production, marketing and sales of pharmaceuticals across the world. The company’s products are targeted at disorders such as depression and anxiety, psychotic disorders, epilepsy, Huntington’s, Alzheimer’s and Parkinson’s diseases. Lundbeck’s pipeline consists of several mid- to late-stage development programs.
Lundbeck employs more than 5,800 people worldwide, 2,000 of whom are based in Denmark. We have employees in 57 countries and our products are registered in more than 100 countries. We have research centers in Denmark, China and the United States and production facilities in Italy, France, Mexico, China and Denmark. Lundbeck generated revenue of approximately DKK 15 billion in 2012. Lundbeck’s shares are listed on the stock exchange in Copenhagen under the symbol “LUN.” Lundbeck has a sponsored Level 1 ADR programme listed in the US (OTC) under the symbol “HLUYY.” For additional information, we encourage you to visit our corporate site www.lundbeck.com.
SABRIL is a registered trademark of Lundbeck.
Actelion receives U.S. FDA approval of Opsumit (macitentan) for the treatment of pulmonary arterial hypertension.
Allschwil, Switzerland, October 18, 2013 – Actelion Ltd (SIX: ATLN) announced today that the United States Food and Drug Administration (FDA) has approved the use of the orally available endothelin receptor antagonist Opsumit® (macitentan) 10 mg once daily for the treatment of pulmonary arterial hypertension (PAH) to delay disease progression.
Opsumit is indicated for the treatment of pulmonary arterial hypertension (PAH, WHO Group I) to delay disease progression. Disease progression included: death, initiation of intravenous (IV) or subcutaneous prostanoids, or clinical worsening of PAH (decreased 6-minute walk distance, worsened PAH symptoms and need for additional PAH treatment). Opsumit also reduced hospitalization for PAH.
Effectiveness was established in a long-term study in PAH patients with predominantly WHO Functional Class II-III symptoms treated for an average of 2 years. Patients were treated with Opsumit® monotherapy or in combination with phosphodiesterase-5 inhibitors or inhaled prostanoids. Patients had idiopathic and heritable PAH (57%), PAH caused by connective tissue disorders (31%), and PAH caused by congenital heart disease with repaired shunts (8%).
Dr. Vallerie McLaughlin, Director of the Pulmonary Hypertension Program in the Division of Cardiovascular Medicine at the University of Michigan, commented: “Over the past twenty years, great strides have been made in treating PAH patients. However, there has been a medical need for innovative treatments that improve long-term outcomes. Opsumit® is the first clinically proven and only oral treatment option indicated to delay disease progression and reduce the need for PAH hospitalization.”
Dr. McLaughlin concluded: “These effects were demonstrated in SERAPHIN, the first and largest PAH outcome study to date, where Opsumit® was given on average for 2 years, as a monotherapy or in combination with phosphodiesterase-5 inhibitors or inhaled prostanoids. I am very pleased that PAH patients will have this new treatment option.”
Jean-Paul Clozel, M.D. and Chief Executive Officer of Actelion commented: “Today’s approval of Opsumit® by the FDA is providing the PAH community with a unique treatment option, the only oral PAH medicine that has proven to delay disease progression. Over the last 14 years, Actelion has worked tirelessly to first discover and then develop Opsumit® in the largest, longest and first-ever outcome study in PAH. I would like to express my gratitude to all the members of the PAH community. Without their contribution, Opsumit® would not have become a reality. We will now leverage our existing PAH expertise and infrastructure to bring Opsumit® to patients within the coming weeks.”
The US label for Opsumit® carries a Boxed Warning alerting patients and health care professionals that the drug should not be used in pregnant women because it can harm the developing fetus. Female patients can receive the drug only through the Opsumit REMS Program. All female patients must be enrolled in the program, comply with pregnancy testing requirements and be counselled regarding the need for contraception.
The most common adverse reactions (more frequent than placebo by 3% or more) observed in patients treated with Opsumit® were anemia, nasopharyngitis/pharyngitis, bronchitis, headache, influenza, and urinary tract infection.
Physicians are advised to measure hemoglobin and liver enzymes prior to initiation of Opsumit® and repeat during treatment as clinically indicated.
In the United States, Actelion expects Opsumit® to become available to patients in November. Outside of the United States, Actelion continues to work with health authorities to obtain regulatory approval for Opsumit® .
The FDA approval was based in part on data from the landmark phase III SERAPHIN study. Published in the New England Journal of Medicine in August 2013, the SERAPHIN study showed the risk of the first occurrence of a morbidity or mortality event, the primary endpoint of the study, was reduced by 45% (p<0.0001) with macitentan 10 mg compared to placebo. This effect was observed irrespective of whether or not patients were already treated with other therapies for PAH. SERAPHIN also showed a risk reduction in PAH related hospitalization and death of 50% (p<0.0001) compared to placebo. .
Pulmonary arterial hypertension (PAH) is a chronic, life-threatening disorder characterized by abnormally high blood pressure in the arteries between the heart and lungs of an affected individual. The symptoms of PAH are non-specific and can range from mild breathlessness and fatigue during normal daily activity to symptoms of right heart failure and severe restrictions on exercise capacity and ultimately reduced life expectancy.
NOTES TO THE EDITOR
ABOUT OPSUMIT® (MACITENTAN)
Opsumit® (macitentan) is a novel dual endothelin receptor antagonist (ERA) that resulted from a tailored drug discovery process with the target of developing an ERA to address efficacy and safety .
ABOUT THE SERAPHIN STUDY
SERAPHIN (Study with an Endothelin Receptor Antagonist in Pulmonary arterial Hypertension to Improve cliNical outcome) was the largest and longest randomized, controlled study in PAH patients to include a clearly defined morbidity/mortality primary endpoint . The pivotal Phase III study was designed to evaluate the efficacy and safety of Opsumit®(macitentan) – a novel dual endothelin receptor antagonist that resulted from a tailored drug discovery process – through the primary endpoint of time to first morbidity and all-cause mortality event in patients with symptomatic PAH.
Global enrollment was completed in December 2009 with a total of 742 patients. Patients were randomized 1:1:1 to receive two different doses of macitentan (3 mg and 10 mg once daily) or placebo. Patients were allowed to receive PAH background therapy throughout the study, either PDE-5 inhibitors or oral/inhaled prostanoids. This event-driven study was conducted in 151 centers from almost 40 countries in North America, Latin America, Europe, Asia-Pacific and Africa and was completed in the first half of 2012, with 287 patients having an adjudicated event.
Dr. McLaughlin is a consultant to Actelion and was an investigator in the SERAPHIN trial.
ABOUT SERAPHIN STUDY DATA
Patients were randomized to placebo (n=250), macitentan 3 mg (n=250), or macitentan 10 mg (n=242). The primary end point occurred in 46.4%, 38.0%, and 31.4% of the patients in these groups, respectively. The hazard ratio for macitentan 3 mg versus placebo was 0.70 (97.5% CI, 0.52 to 0.96; p=0.0108) and the hazard ratio for macitentan 10 mg versus placebo was 0.55 (97.5% CI, 0.39 to 0.76; p<0.0001). Worsening of pulmonary arterial hypertension was the most frequent primary end point event. The effect of macitentan on this end point was observed irrespective of background therapy for pulmonary arterial hypertension. 
ABOUT THE SAFETY AND TOLERABILITY PROFILE
Opsumit is contraindicated in pregnancy because it may harm the developing fetus. Females of reproductive potential should be counselled on the use of reliable contraception and have a negative pregnancy test prior to initiating therapy and monthly thereafter.
Other ERAs have been associated with elevations of aminotransferases, hepatotoxicity, and liver failure. Liver enzyme tests should be obtained prior to initiation of Opsumit® and repeated during treatment as clinically indicated. If clinically relevant aminotransferase elevations occur, or if elevations are accompanied by clinical symptoms of hepatoxicity, discontinue Opsumit®.
Decreases in hemoglobin concentration and hematocrit occurred following administration of other ERAs and were observed in clinical studies with OPSUMIT. The decreases occurred early and stabilized thereafter. Decreases in hemoglobin seldom require transfusion. Initiation of Opsumit® is not recommended in patients with severe anemia. Hemoglobin should be measured prior to initiation of treatment and repeat during treatment as clinically indicated.
Should signs of pulmonary edema occur, consider the possibility of associated PVOD. If confirmed, discontinue Opsumit®.
Other ERAs have been associated with adverse effects on spermatogenesis. Men should be counseled about potential effects on fertility.
The use of Opsumit® with strong CYP3A4 inducers or inhibitors should be avoided.
The most common adverse reactions (more frequent than placebo by 3% or more) observed in patients treated with Opsumit were anemia, nasopharyngitis/pharyngitis, bronchitis, headache, influenza, and urinary tract infection.
ABOUT OPSUMIT® (MACITENTAN) SUBMISSIONS TO HEALTHCARE AUTHORITIES
Approval of the new drug application for Opsumit® (macitentan) was issued by the US Food and Drug Administration (FDA) on 18October 2013 for the treatment of pulmonary arterial hypertension (PAH, WHO Group I) to delay disease progression. Disease progression included: death, initiation of intravenous (IV) or subcutaneous prostanoids, or clinical worsening of PAH (decreased 6-minute walk distance, worsened PAH symptoms and need for additional PAH treatment). The need for PAH hospitalization was also reduced.
Regulatory reviews are ongoing in Europe, Canada, Switzerland, Australia, Taiwan, Korea and Mexico.
ABOUT PULMONARY ARTERIAL HYPERTENSION [9, 10]
Pulmonary arterial hypertension (PAH) is a chronic, life-threatening disorder characterized by abnormally high blood pressure in the arteries between the heart and lungs of an affected individual. The symptoms of PAH are non-specific and can range from mild breathlessness and fatigue during normal daily activity to symptoms of right heart failure and severe restrictions on exercise capacity and ultimately reduced life expectancy.
PAH is one group within the classification of pulmonary hypertension (PH). This group includes idiopathic PAH, heritable PAH and PAH caused by factors which include connective tissue disease, HIV infection and congenital heart disease.
The last decade has seen significant advances in the understanding of the pathophysiology of PAH, which has been paralleled with developments of treatment guidelines and new therapies. Drugs targeting the three pathways that have been established in the pathogenesis of PAH are endothelin receptor antagonists (ERAs), prostacyclins and phosphodiesterase-5 inhibitors. PAH treatments have transformed the prognosis for PAH patients from symptomatic improvements in exercise tolerance 10 years ago to delayed disease progression today. Improved disease awareness and evidence-based guidelines developed from randomized controlled clinical trial data have highlighted the need for early intervention, goal-oriented treatment and combination therapy.
In PAH, survival rates are unacceptably low and PAH remains incurable.
Actelion Ltd is a biopharmaceutical company with its corporate headquarters in Allschwil/Basel, Switzerland. Actelion’s first drug Tracleer® (bosentan), an orally available dual endothelin receptor antagonist, has been approved as a therapy for pulmonary arterial hypertension. Actelion markets Tracleer through its own subsidiaries in key markets worldwide, including the United States (based in South San Francisco), the European Union, Japan, Canada, Australia and Switzerland. Actelion, founded in late 1997, is a leading player in innovative science related to the endothelium – the single layer of cells separating every blood vessel from the blood stream. Actelion’s over 2,300 employees focus on the discovery, development and marketing of innovative drugs for significant unmet medical needs. Actelion shares are traded on the SIX Swiss Exchange (ticker symbol: ATLN) as part of the Swiss blue-chip index SMI (Swiss Market Index SMI®).
For further information please contact:
Senior Vice President, Head of Investor Relations & Public Affairs
Actelion Pharmaceuticals Ltd, Gewerbestrasse 16, CH-4123 Allschwil
+41 61 565 62 62
+1 650 624 69 36
The above information contains certain “forward-looking statements”, relating to the company’s business, which can be identified by the use of forward-looking terminology such as “estimates”, “believes”, “expects”, “may”, “are expected to”, “will”, “will continue”, “should”, “would be”, “seeks”, “pending” or “anticipates” or similar expressions, or by discussions of strategy, plans or intentions. Such statements include descriptions of the company’s investment and research and development programs and anticipated expenditures in connection therewith, descriptions of new products expected to be introduced by the company and anticipated customer demand for such products and products in the company’s existing portfolio. Such statements reflect the current views of the company with respect to future events and are subject to certain risks, uncertainties and assumptions. Many factors could cause the actual results, performance or achievements of the company to be materially different from any future results, performances or achievements that may be expressed or implied by such forward-looking statements. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those described herein as anticipated, believed, estimated or expected.
News Release Intended for U.S. Media Only
FDA Approves Bayer’s New Class of Drug Adempas® (riociguat) tablets to Treat Adults with PAH and Persistent, Recurrent or Inoperable CTEPH First and only drug approved in U.S. to Treat Two Forms of Pulmonary Hypertension (WHO Group 1 and 4)
Whippany, N.J., October 8, 2013 – Bayer HealthCare announced today that the United States Food and Drug Administration (FDA) has approved Adempas® (riociguat) tablets for: (i) the treatment of adults with persistent/recurrent chronic thromboembolic pulmonary hypertension (CTEPH) (WHO* Group 4) after surgical treatment or inoperable CTEPH to improve exercise capacity and WHO functional class; and (ii) the treatment of adults with pulmonary arterial hypertension (PAH) (WHO Group 1) to improve exercise capacity, improve WHO functional class and delay clinical worsening. In PAH, efficacy was shown in patients on Adempas monotherapy or in combination with endothelin receptor antagonists (ERAs) or prostanoids (inhaled, oral or subcutaneous). Studies establishing effectiveness included predominately patients with WHO functional class II-III and etiologies of idiopathic or heritable PAH (61%) or PAH associated with connective tissue diseases (25%). Adempas is the only treatment approved in the U.S. for use in two types of pulmonary hypertension (WHO Group 1 and 4). It is the first and only FDA-approved drug therapy for persistent/recurrent CTEPH after surgical treatment or inoperable CTEPH. It is also the only approved oral therapy in PAH with efficacy shown in monotherapy or in combination with ERAs or prostanoids. For all female patients, Adempas is available only through a restricted program called the Adempas Risk Evaluation and Mitigation Strategy (REMS) Program.
“CTEPH and PAH are serious and life-threatening diseases,” said Nick H. Kim, Associate Clinical Professor of Medicine, Division of Pulmonary and Critical Care Medicine; Director, Pulmonary Vascular Medicine; Director, Fellowship Program; University of California San Diego. “The approval of Adempas equips physicians with a new approach to treating PAH patients, and it gives us the first approved drug treatment for patients with inoperable CTEPH or with persistent/recurrent CTEPH after surgery. While surgery should always be considered as the first treatment option for CTEPH, the fact remains that up to forty percent of CTEPH patients are not eligible for surgery, and ten to thirty-five percent of CTEPH patients have disease that persists after surgery.” PAH is a disease characterized by elevated pressure in the pulmonary arteries. CTEPH is a form of pulmonary hypertension in which blood clots and thromboembolic occlusion of the pulmonary vessels leads to increased pressure in the pulmonary arteries. The standard treatment for CTEPH is pulmonary endarterectomy, a potentially curative surgery that clears clots and scar material from the blood vessels of the lung. “Bayer is deeply committed to bringing new treatment options to patients with life-threatening diseases. Adempas is an excellent example of this commitment, because it is the result of years of dedicated research in our Bayer laboratories into a new way of treating two forms of pulmonary hypertension,” said Pamela A. Cyrus, MD, Vice President and Head, U.S. Medical, Bayer HealthCare Pharmaceuticals. “We are pleased to bring this new class of treatment to patients with PAH or with inoperable CTEPH or persistent/recurrent CTEPH after surgical treatment.” Rino Aldrighetti, President and CEO, Pulmonary Hypertension Association added, “From a patient’s perspective, living with pulmonary hypertension remains difficult. We know that not all treatments work for all people. We get excited when there is a new treatment option for PAH patients, and we are thrilled there is finally an approved drug treatment for people living with persistent/recurrent CTEPH after surgical treatment or inoperable CTEPH.” Adempas, a stimulator of soluable guanylate cyclase (sGC), represents a new class of drug now available in the U.S. Pulmonary hypertension is associated with endothelial dysfunction, impaired synthesis of nitric oxide (NO) and insufficient stimulation of the NO-sGC-cGMP pathway. Adempas sensitizes sGC to endogenous NO by stabilizing the NO-sGC binding. Adempas also directly stimulates sGC via a different binding site independently of NO. Adempas restores the NO-sGC-cGMP pathway and leads to increased generation of cGMP with subsequent vasodialation. The most common adverse reactions occurring more frequently (>3%) on Adempas than placebo were headache (27% vs 18%), dyspepsia/gastritis (21% vs. 8%), dizziness (20% vs 13%), nausea (14% vs 11%), diarrhea (12% vs 8%), hypotension (10% vs 4%), vomiting (10% vs 7%), anemia (7% vs 2%), gastroesophageal reflux disease (5% vs 2%), and constipation (5% vs 1%). Other events that were seen more frequently in Adempas compared to placebo and potentially related to treatment were: palpitations, nasal congestion, epistaxis, dysphagia, abdominal distension and peripheral edema. About Patient Assistance Program Bayer offers patient assistance through the Adempas Aim Support Center program, which will assist with obtaining coverage and patient support of Adempas. Patients and providers may contact the program at 1-855-4ADEMPAS for additional information. IMPORTANT SAFETY INFORMATION
WARNING: EMBRYO-FETAL TOXICITY Do not administer Adempas (riociguat) tablets to a pregnant female because it may cause fetal harm.
Females of reproductive potential: Exclude pregnancy before the start of treatment, monthly during treatment, and 1 month after stopping treatment. Prevent pregnancy during treatment and for one month after stopping treatment by using acceptable methods of contraception.
For all female patients, Adempas is available only through a restricted program called the Adempas Risk Evaluation and Mitigation Strategy (REMS) Program.
Contraindications. Adempas is contraindicated in:
Warnings and Precautions Embryo-Fetal Toxicity. Adempas may cause fetal harm when administered during pregnancy and is contraindicated for use in women who are pregnant. In females of reproductive potential, exclude pregnancy prior to initiation of therapy, advise use of acceptable contraception and obtain monthly pregnancy tests. For females, Adempas is only available through a restricted program under the Adempas REMS Program. Adempas REMS Program. Females can only receive Adempas through the Adempas REMS Program, a restricted distribution program. Important requirements of the Adempas REMS program include the following:
Further information, including a list of certified pharmacies, is available at www.AdempasREMS.com or 1-855-4ADEMPAS. Hypotension. Adempas reduces blood pressure. Consider the potential for symptomatic hypotension or ischemia in patients with hypovolemia, severe left ventricular outflow obstruction, resting hypotension, autonomic dysfunction, or concomitant treatment with antihypertensives or strong CYP and P-gp/BCRP inhibitors. Consider a dose reduction if patient develops signs or symptoms of hypotension. Bleeding. In the placebo-controlled clinical trials program, serious bleeding occurred in 2.4% of patients taking Adempas compared to 0% of placebo patients. Serious hemoptysis occurred in 5 (1%) patients taking Adempas compared to 0 placebo patients, including one event with fatal outcome. Serious hemorrhagic events also included 2 patients with vaginal hemorrhage, 2 with catheter site hemorrhage, and 1 each with subdural hematoma, hematemesis, and intra-abdominal hemorrhage. Pulmonary Veno-Occlusive Disease. Pulmonary vasodilators may significantly worsen the cardiovascular status of patients with pulmonary veno-occlusive disease (PVOD). Therefore, administration of Adempas to such patients is not recommended. Should signs of pulmonary edema occur, the possibility of associated PVOD should be considered and if confirmed, discontinue treatment with Adempas. Most Common Adverse Reactions The most common adverse reactions occurring more frequently (>3%) on Adempas than placebo were headache (27% vs 18%), dyspepsia/gastritis (21% vs. 8%), dizziness (20% vs 13%), nausea (14% vs 11%), diarrhea (12% vs 8%), hypotension (10% vs 4%), vomiting (10% vs 7%), anemia (7% vs 2%), gastroesophageal reflux disease (5% vs 2%), and constipation (5% vs 1%). Other events that were seen more frequently in Adempas compared to placebo and potentially related to treatment were: palpitations, nasal congestion, epistaxis, dysphagia, abdominal distension and peripheral edema. For important risk and use information, please see the full Prescribing Information, including Boxed Warning, at www.adempas-us.com. About Bayer HealthCare Pharmaceuticals Inc. Bayer HealthCare Pharmaceuticals Inc. is the U.S.-based pharmaceuticals business of Bayer HealthCare LLC, a subsidiary of Bayer AG. Bayer HealthCare is one of the world’s leading, innovative companies in the healthcare and medical products industry, and combines the activities of the Animal Health, Consumer Care, Medical Care, and Pharmaceuticals divisions. As a specialty pharmaceutical company, Bayer HealthCare provides products for General Medicine, Hematology, Neurology, Oncology and Women’s Healthcare. The company’s aim is to discover and manufacture products that will improve human health worldwide by diagnosing, preventing and treating diseases. Bayer® and the Bayer Cross® are registered trademarks of Bayer. Intended for U.S. media only U.S. Media Contact: Marcy Funk, Communications, Bayer HealthCare Telephone: (862) 404-5385 E-Mail: firstname.lastname@example.org Forward-Looking Statements This release may contain forward-looking statements based on current assumptions and forecasts made by Bayer Group or subgroup management. Various known and unknown risks, uncertainties and other factors could lead to material differences between the actual future results, financial situation, development or performance of the company and the estimates given here. These factors include those discussed in Bayer’s public reports which are available on the Bayer website at www.bayer.com. The company assumes no liability whatsoever to update these forward-looking statements or to conform them to future events or developments.
News Release Intended for U.S. Media Only
FDA ADVISORY COMMITTEE UNANIMOUSLY RECOMMENDS APPROVAL OF BAYER’S RIOCIGUAT IN TWO PULMONARY HYPERTENSION INDICATIONS
If approved by the FDA, riociguat will be the first treatment for inoperable CTEPH or persistent/recurrent CTEPH after surgery and a new treatment for PAH
Whippany, N.J., August 6, 2013– Bayer HealthCare today announced that the U.S. Food and Drug Administration’s (FDA) Cardiovascular and Renal Drugs Advisory Committee recommended approval for investigational riociguat, proposed trade name Adempas™, in two forms of pulmonary hypertension. The Committee voted 11 to 0 that riociguat should be approved for the treatment of pulmonary arterial hypertension [PAH] of WHO1 Group 1. The Committee also voted 11 to 0 that riociguat should be approved for the treatment of chronic thromboembolic pulmonary hypertension (CTEPH) of WHO Group 4. In February 2013, Bayer submitted a new drug application for riociguat in two indications: (i) the treatment of PAH (WHO Group 1) to improve exercise capacity, improve WHO functional class and delay clinical worsening; and (ii) the treatment of persistent/recurrent CTEPH (WHO Group 4) after surgical treatment or inoperable CTEPH to improve exercise capacity and WHO functional class. “We appreciate the Committtee’s discussion today around the safe and appropriate use of riociguat and are pleased with the outcome of the votes,” said Pamela A. Cyrus, MD, Vice President and Head of U.S. Medical Affairs, Bayer HealthCare Pharmaceuticals. “If approved, riociguat will offer a new treatment option for patients with PAH and will also provide the first approved non-surgical treatment option for CTEPH patients who are inoperable or who have recurrent or persistent disease. We look forward to continued dialogue with the FDA in order to make riociguat available to patients.”
PAH and CTEPH are both life-threatening forms of pulmonary hypertension that cause significantly increased pressure in the pulmonary arteries. Riociguat is an investigational, oral medication for the treatment of adult patients with PAH or inoperable or persistent/recurrent CTEPH. If approved by the FDA later this year, it would create a new class of therapy available in the U.S. PH is associated with endothelial dysfunction, impaired synthesis of nitric oxide (NO) and insufficient stimulation of soluble guanylate cyclase (sGC). Riociguat stimulates sGC independent of NO and increases the sensitivity of sGC to NO. Data presented at today’s advisory committee meeting included results from the global Phase 3 clinical program, which enrolled 704 patients across two Phase 3 studies. Both studies met their primary endpoint by demonstrating a statistically significant improvement in the six-minute walk test (6MWT), after 16 and 12 weeks respectively. Riociguat was also associated with improvements across multiple, relevant, secondary endpoints in the studies. The most common treatment-emergent adverse events with riociguat were headache, dizziness, dsypesia, peripheral edema, nausea, diarrhea and vomiting. The advisory committee’s vote will be taken into consideration by the FDA when making its decision on the approvability of Bayer’s NDA for riociguat, which was submitted in February 2013. After acceptance of the NDA, the FDA granted riociguat priority review designation, which is given to drugs that have the potential to offer significant improvement in treatment or provide a treatment option where no adequate therapy exists. About Pulmonary Arterial Hypertension (PAH) In PAH, a rare and life-threatening disease, the blood pressure in the pulmonary arteries (the arteries that take de-oxygenated blood to the lungs from the heart) is significantly increased. PAH is characterized by morphological changes to the endothelium of the arteries of the lungs causing remodeling of the tissue, vasoconstriction and thrombosis-in-situ. As a result of these changes, the blood vessels in the lungs are narrowed, making it difficult for the heart to pump blood through to the lungs. In most cases, PAH has no known cause and, in some cases, it can be inherited. About Chronic Thromboembolic Pulmonary Hypertension (CTEPH) CTEPH is also a rare and life-threatening disease in which it is believed that thromboembolic occlusion (organized blood clots) of pulmonary vessels gradually lead to an increased pressure in the pulmonary arteries, resulting in an overload of the right heart. CTEPH may evolve after prior episodes of acute pulmonary embolism, but the pathogenesis is not yet completely understood. The standard treatment for CTEPH is pulmonary endarterectomy (PEA), a surgical procedure in which the blood vessels of the lungs are cleared of clot and scar material. However, CTEPH is inoperable in an estimated 20 to 40 percent of patients, and, in some cases, the disease persists or reoccurs after surgery. About Bayer HealthCare Pharmaceuticals Inc.Bayer HealthCare Pharmaceuticals Inc. is the U.S.-based pharmaceuticals business of Bayer HealthCare LLC, a subsidiary of Bayer AG. Bayer HealthCare is one of the world’s leading, innovative companies in the healthcare and medical products industry, and combines the activities of the Animal Health, Consumer Care, Medical Care, and Pharmaceuticals divisions. As a specialty pharmaceutical company, Bayer HealthCare provides products for General Medicine, Hematology, Neurology, Oncology and Women’s Healthcare. The company’s aim is to discover and manufacture products that will improve human health worldwide by diagnosing, preventing and treating diseases. Bayer® and the Bayer Cross® are registered trademarks of Bayer.
Intended for U.S. media only Media Contact: Marcy Funk, Communications, Bayer HealthCare Telephone: (862) 404-5385 E-Mail: email@example.com
Forward-Looking Statements This release may contain forward-looking statements based on current assumptions and forecasts made by Bayer Group or subgroup management. Various known and unknown risks, uncertainties and other factors could lead to material differences between the actual future results, financial situation, development or performance of the company and the estimates given here. These factors include those discussed in Bayer’s public reports which are available on the Bayer website at www.bayer.com. The company assumes no liability whatsoever to update these forward-looking statements or to conform them to future events or developments.Posted in About Us, Diseases, Featured | Tagged Adempas, Amy Magro, antiepileptic, Bayer, Bayer HealthCare, blood clots, child treatment, chronic, complex partial seizure, complex partial seizures, connective tissue diseases, CPS, CTEPH, disease, drug, drugs, dyspepsia, endothelial dysfunction, endothelin receptor antagonists, Epilepsy, Epilepsy Foundation, ERA, FDA, Food and Drug Administration, gastritis, Healthcare, Lundbeck, macitentan, Marcy Funk, Matt Flesch, monotherapy, News release, opsumit, partial seizure, partial seizures, Patient Assistance Program, PH, PHA, Pharmaceutical, Philip Gattone, prostanoids, pulmonary endarterectomy, Pulmonary Hypertension, Pulmonary Hypertension Association, Pulmonary Veno-Occlusive Disease, rare, refractory, refractory CPS, REMS, Rino Aldrighetti, riociguat, Risk Evaluation and Mitigation Strategy Program, Sabril, seizure, seizures, sgc, SHARE, SHARE program, soluable guanylate cyclase, thromboembolic occlusion, thromboembolic pulmonary hypertension, treating children, treatment, University of California San Diego, vigabatrin, vision loss, World Health Organization | Leave a comment October 18, 2013
Modern artist Paul Klee admired and was inspired by the spontaneous power of children’s art. Eva Leonard reports on Klee’s life, art, and creative perseverance as he fought the destructive forces of the Nazi party and the effects of systemic scleroderma.
One of the world’s most celebrated and influential modern artists, Paul Klee (pronounced “klay”), drew profound inspiration from the art of children. Because Klee believed that children created the purest art, he worked to make his art more child-like.
And he took very creative steps to do so. To reduce the impact of his artistic education on his art, he sometimes switched hands or closed his eyes when he drew, and he also used methods such as scraping layers of paint from his work to reveal the colors underneath. Klee also collected and studied the drawings of his son, Felix, and those of other children.
The influence of children’s art on his work, with its simple, bold lines, and vivid colors, is clear in his cheeky response to art critics of the time: “Those gentlemen, the critics, often say that my pictures resemble the scribbles and messes of children. I hope they do!”
Klee’s observation, “A line is a dot that went for a walk,” reflects his boundless sense of playfulness and imagination. He was prolific, producing more than ten thousand works of art, more than two thousand of which he created following the appearance of symptoms that would later be diagnosed as scleroderma. His desire to continually simplify his technique would serve him in his later years, as the symptoms of scleroderma began to limit his movement.
Born in 1879 near Bern, Switzerland, into a family of musicians, Klee first studied to become a violinist. He initially drew only in black and white, but a trip to Tunisia in 1914 awakened his sense of color and light. Klee also used elements of music, letters, numbers, and hieroglyphic-like symbols in his work, and was intrigued by the art of the mentally ill.
In 1906, he married the pianist Lili Stumpf, and the couple moved to Munich. In 1916, he joined the German army, painting camouflage on planes. Klee lectured at the Bauhaus from 1921 to 1931 and at the Art Academy in Dusseldorf until the Nazis dismissed him in 1933, when he refused to pledge allegiance to the party, and the Gestapo searched his home. He then fled Nazi Germany for Switzerland.
The Nazis declared Klee’s art “degenerate,” as they did most Modern art, including the works of Pablo Picasso, Joan Miró, and Salvador Dali, which they deemed counter to Nazi ideals. The Nazis seized more than a hundred of Klee’s works from public collections. Their so-called “Degenerate Art” exhibition in 1937 in Munich, meant to ridicule art they considered “un-German,” included 17 works by Klee.
After fleeing to Switzerland, Klee had his first exhibition in London and was visited by well-known artists, including Picasso, and Wassily Kandinsky. His star rose in the United States, where he earned kudos from artists Diego Rivera and Frida Kahlo, as well as from art dealers, collectors, and museum directors.
In 1936, the first symptoms of Klee’s scleroderma—heart irregularities, fatigue, weakness, chronic pneumonia, hardening of the skin, and gastrointestinal problems—became pronounced. Although he produced only 25 works of art that year, he rebounded as the disease stabilized. He also found new ways to create, using different styles and materials, creating 264 works the following year and more than twelve hundred pieces in 1939.
Klee’s scleroderma was likely diffuse systemic sclerosis, the most serious form of the disease. (There were no treatments for it at that time, and the condition went undiagnosed until after his death.) His art provided refuge and a way to express his struggles with the illness.
In his book, “Paul Klee and His Illness” (Karger, 2010), the author, Hans Suter, M.D., noted, “For Klee, drawing and painting were his personal form of meditation. It seems to me that this ability to sink into deep thought while working provided an excellent opportunity to get anxiety and distress out of his system.”
As Klee’s scleroderma progressed, it prevented him from doing two of the things he most enjoyed: hiking and playing the violin. However, he could still paint and draw, producing larger, simpler works, and using coarser materials such as burlap. Klee’s creative responses to the new limitations on his mobility and dexterity might have inspired his declaration: “He has found his style when he cannot do otherwise.”
During 1940, the year Klee died of heart failure from severe scleroderma at the age of 60, he created 366 works of art. Seventy-three years later, his art continues to inspire admirers, influencing not only visual artists, but also contemporary musicians all over the world, with its vibrant sense of rhythm, movement, imagination, and emotion.
The Norton Simon Museum in Pasadena, California, has more than fifty works by Klee, including paintings, drawings and prints, and one painting by Klee’s son, Felix, made when he was 12. Leah Lehmbeck, curator for the museum, said, “Very early on Klee recognized the value of children’s art and children’s drawings. He saw it as a sort of beginning of art.”
“Klee believed that creativity resided within. True things were drawn on from nature, and nothing was ever completely abstract. But the purest things were those that came from one’s self, and children are the best examples of that. They aren’t burdened by anything yet.
“There is also a really nice quote of how he saw himself. He considered himself ‘a child of this earth, yet also a child of the universe; the offspring of a star among stars.’”
The following museums have Paul Klee collections and/or exhibitions of his work scheduled. If you’re planning on visiting a museum to see its Klee collection, check to confirm that it will be on display, as art is often rotated. You can also view Klee’s works on many of the museum websites below.
The exhibition “Paul Klee: Making Visible” is on view at the Tate Modern from October 16, 2013 through March 9, 2014. www.tate.org.uk/whats-on/tate-modern/exhibition/ey-exhibition-paul-klee
In 2005, the Paul Klee Center (Zentrum Paul Klee) opened on the outskirts of Bern, Switzerland, designed by Italian architect Renzo Piano to look like a trio of rolling glass-and-steel hills, housing close to four thousand works by Klee. Among the works on display are nearly 50 hand puppets Klee made for his son, Felix. One of the center’s most popular areas is its Children’s Museum, Kindermuseum Creaviva.
Kindermuseum Creaviva Project Manager Sara Stocker said, “Paul Klee was not only a painter, musician and writer; he was also an excellent teacher. The idea for a Children’s Museum is to give children, teenagers and adults access to art and culture, and allow them to discover their own creativity on the basis of Paul Klee’s works.
“Creaviva’s workshops are its foundation. Children and adults can work in the Open Studio under the guidance of an art instructor, and participants can take home artwork they have created in the workshops. Creaviva’s ‘Klee Without Barriers’ offers workshops tailored to individual needs for people with disabilities.The interactive exhibition in Creaviva’s loft invites participation in playful discussions about art. It’s all about imagination, curiosity, the exciting journeys, and the joy that’s felt in one’s creation.
“The Children’s Museum Creaviva promotes Klee’s playful, experimental, humorous approach to art. ‘Creaviva’ was formed from the Latin words for ‘create’ and ‘lively.’ This can be translated as ‘a lively place of creativity.’ Long live creativity!” —Translation by Sabrina I. Parmelee
Posted in About Us, Diseases, Featured | Tagged Art, Art Education, Baltimore Museum, Bern, BMA, Children's Museum, Creativity, Creaviva, diffuse systemic sclerosis, Dusseldorf art academy, Felix Klee, Fire at Full Moon, Guggenheim, Hans Suter, Imagination, Karger, Leah Lehmbeck, Lili Stumpf, metmuseum, Metropolitan, Metropolitan Museum of Art, Miriam Loertscher, Modern Art, National Gallery, NGA, Norton Simon, Paul Clay, Paul Klay, Paul Klee, Paul Klee and His Illness, Paul Klee: Making Visible, Philadelphia Museum, Philamuseum, Sclero, scleroderma, Switzerland, Tate, Two Heads, Zentrum | Leave a comment September 27, 2013
Sixteen-year-old Elaine Kelley’s narcolepsy symptoms first appeared in July 2011, shortly before she started her freshman year of high school in Tulsa, Oklahoma.
“The first thing I noticed was the cataplexy,” she recalls. “It came on very suddenly. Laughing would make me very weak, and I thought that was strange. Then it just progressively got worse.
“I told my mom about it. Then I also started sleeping a lot during the day. We thought that was strange too.”
The cataplexy attacks first occurred at home and at her cousin’s house. Then, Elaine says, “It happened big time when we were shopping for school shoes, and I was yelling for my mom, asking her to come look.” Fortunately, Elaine wasn’t hurt when she collapsed in the shoe store, but she says, “That’s when we knew it was serious.
“One night I had a cataplexy attack, and my dad saw it for the first time. He wanted to take me to the hospital. We were just going to go to an urgent care center, but it was closed.
“The next day, we called my pediatrician, and she wanted me to check into the hospital. I checked in the next day and went through a 24-hour EKG and an EEG and spent the night there. They couldn’t find anything. They had a recording of me having cataplexy and still couldn’t figure it out, because they had never seen it before.
“They thought it was epilepsy, anemia, or something neurological, but everything came back normal. They might have said that it could be narcolepsy, but they never said ‘cataplexy.’
Elaine explains how she ended up essentially diagnosing herself. “I started googling ‘laughing and falling,’ and that’s when I came across cataplexy. So I looked into that because there was a story about a lady who had it.
“We called my pediatrician; she referred us to a sleep specialist, and I took the sleep test. I was lucky to figure it out pretty quickly, after a few months of the symptoms, because school had started.
Elaine did have cataplexy episodes at school, but luckily, she was in the bathroom with friends when they occurred. “All my friends knew about it and were supportive, which was nice,” she recalls.
She describes her initial attempts to prevent the cataplexy on her own. “It took a lot of self-control to try to not let it happen. I would clench my fists really tight if I felt it coming on and tighten my body so I wouldn’t lose muscle control. I noticed that it did help a little.
“But once I got the right medication and figured all that out, the cataplexy has never gone back to the way it was when it was at its worst.
“I still have daytime sleepiness, but a 30-minute nap once a day helps that. I still notice that when I laugh, sometimes I lose control of my facial muscles a little bit.
“I’m very lucky. I have a strong support system with my friends and family. I play soccer. I like to babysit. I like spending time with my friends.
“I want to go to college. I really love history, so I want to major in that. My reach school is Brown; that’s what I’m shooting for. If not, I’ll probably go to the University of Oklahoma or the University of Kansas—somewhere close to home.
“When I first was diagnosed, my dad sent out an email letting all my teachers know. They were all really curious. There was actually another kid who graduated last year who had narcolepsy, and I was able to talk to him about it.
“People ask me about it a lot. I have no problems explaining it.
There was a rumor at school that when I get excited, I fall asleep. That’s what some kids twisted cataplexy into. I set everyone straight.
Elaine says there are a couple of things she’d like people to know about narcolepsy: “I’d like to make it clear that I don’t fall asleep standing up, because a lot of people ask me that.”
She also says that if there’s one thing she’d like people to be aware of, it’s to be thankful to have a sleep cycle. “Because I sure do miss that.”
For those teens and children who’ve been diagnosed with narcolepsy, she adds, “Don’t be ashamed or embarrassed that you have it. I struggled with that. But if you’re confident about it, it makes it a lot easier.” —ELPosted in About Us, Diseases, Featured | Tagged anemia, Cataplexy, cataplexy attack, ECG, EEG, EKG, Elaine Kelley, electrocardiogram, Electroencephalography, Epilepsy, in your words, laughing and falling, narcolepsy, neurological disorder, patient profile, sleep, sleep specialist, sleep study, sleep test | 3 Comments September 25, 2013
A quick look at some of the people and the passion behind Caring Voice Coalition, and their experiences over the past ten years at CVC.
On July 12, 2013, the Department of Health and Human Services issued a final rule announcing new requirements for “Navigators” and other advocates who will help patients use the Health Insurance Exchanges.
First, the final rule added another resource to health care reform, “certified application counselors,” which each Exchange must offer. Second, the final rule explained the differing standards for Navigators and certified application counselors.
Certified application counselors are individuals (or entities) who will engage in many of the same duties as Navigators. Like Navigators, certified application counselors will provide information regarding and facilitate enrollment in health plans offered through the Exchanges. The main difference between a Navigator and a certified application counselor is the former has a broader range of duties than the latter.
The states or Exchanges may require Navigators complete a certification process; however, as the name suggests, certified application counselors must be certified according to their Exchange’s requirements.
Similarly, training may very between Navigators and application counselors. For example, Navigator training must include: information on qualified health plan options, insurance affordability programs, eligibility, and other government regulations. Conversely, certified application counselors’ training may be more limited simply because they have fewer duties than their Navigator counterparts.
Another difference is Navigators cannot have any conflicts of interest and must maintain a plan for remaining free of such conflicts. Certified application counselors, on the other hand, may have conflicts of interest; although, they must report such conflicts to the consumers they are assisting.
Further, the final rule requires Navigators to provide culturally and linguistically appropriate services (CLAS standards), which include: maintaining knowledge about racial, ethnic and cultural groups in the Navigator’s area, communicating with consumers in their preferred language, and promoting a staff representative of the Navigator’s consumer demographic. Certified application counselors need not comply with CLAS standards.
Despite the differences between Navigators and certified application counselors, the Final Rule compels competence and accountability from both. Overall, the Final Rule reflects an intrinsic goal of health care reform: assuring improved access to health care.
—Stephanie Posuniak, CVC Health Care Attorney
 A “Navigator” is an individual (or entity) who receives funds from a federally-facilitated Exchange and state partnership Exchanges. “Non-navigator assistance personnel” are persons (or entities) who receive funds from the federal government to assist with state-based Exchanges. For purposes of this article, this writer will refer to Patient Navigators and non-navigator assistance personnel collectively as “Navigators.”Posted in About Us, Diseases, Featured | Tagged application counselors, CLAS, CLAS standards, Department of Health and Human Services, health care, Health Insurance Exchanges, Healthcare, HHS, insurance, Navigators, patient navigator, stephanie posuniak | Leave a comment ← Previous post Next post →