Parent Project Muscular Dystrophy and Santhera Pharmaceuticals Team Up on Benefit/Risk Study in Duchenne

Collaboration results from Duchenne community's draft guidance to FDA

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Nov 25, 2014, 07:00 ET

HACKENSACK, N.J., Nov. 25, 2014 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD), the leading advocacy organization working to end Duchenne muscular dystrophy (Duchenne) and Santhera Pharmaceuticals (SIX: SANN), a Swiss specialty pharmaceutical company focusing on the development and marketing of innovative pharmaceutical products for the treatment of mitochondrial and neuromuscular diseases, will collaborate on a benefit/risk study in Duchenne. The study will focus specifically on patient and caregiver preferences regarding pulmonary therapies in the disease, and will be based on data from Santhera's successful phase III clinical trial of idebenone.

Duchenne muscular dystrophy is the most common fatal genetic disorder diagnosed in childhood, affecting approximately one in every 3,500-5,000 live male births.

Earlier this year, PPMD convened a broad coalition of over 80 stakeholders to draft and submit the first-ever patient advocacy-initiated guidance for a rare disease to the U.S. Food and Drug Administration (FDA) to help accelerate development and review of potential therapies for Duchenne. One of the recommendations in the guidance was to create partnerships between patient groups and industry to study the benefit/risk preferences of the disease community to better inform the company's new drug application. Santhera stepped forward immediately to work with PPMD on just such a study assessing the benefit/risk preferences for idebenone (Raxone®/Catena®).

According to PPMD's Founding President and CEO, Pat Furlong, "Since entering the Duchenne space, Santhera has proven their dedication to this community and to finding therapies to treat this disease. They are committed to provide thorough and reliable data and information in the submission to the FDA when they reach that stage. We are happy to collaborate with Santhera's team to ensure the patient's voice is included in the benefit/risk equation. The FDA has told us they want to know what the patients want and thanks to this study we will be able to tell them."

"Following the successful outcome of our Phase 3 trial, we are excited about this collaboration with PPMD to determine patients' and caregivers' views of the benefit/risk balance for Catena^®/Raxone^®," emphasized Thomas Meier, CEO of Santhera. "PPMD has become a powerful and reliable partner of industry in facilitating the development of promising treatment options. With this study of the benefit/risk preferences of the disease community, which is the first of its kind, we will proactively meet regulators' expectations in the content of our NDA filing."

Over the last few years, PPMD has implemented a detailed advocacy agenda that included publication of Putting Patients First, a white paper submitted to the FDA outlining recommendations to speed responsible access to new therapies for Duchenne and other rare, serious and life-threatening neurologic disorders; publication of our benefit/risk study that quantified caregiver preferences regarding emerging therapies, which was shared with the FDA; the above-mentioned draft guidance submitted to the FDA; and now this collaboration with Santhera to best prepare their submission of new drug applications to the Agency.

To learn more about PPMD and our ongoing advocacy efforts, visit PPMD's website.

About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy is the most common fatal genetic disorder diagnosed in childhood, affecting approximately one in every 3,500-5,000 live male births (about 20,000 new cases worldwide each year). Because the Duchenne gene is found on the X-chromosome, it primarily affects boys; however, it occurs across all races and cultures.

Duchenne results in progressive loss of strength and is caused by a mutation in the gene that encodes for dystrophin. Because dystrophin is absent, the muscle cells are easily damaged. The progressive muscle weakness leads to serious medical problems, particularly issues relating to the heart and lungs. Young men with Duchenne typically live into their late twenties.

Duchenne can be passed from parent to child, but approximately 35 percent of cases occur because of a random spontaneous mutation. In other words, it can affect anyone. Although there are medical treatments that may help slow its progression, there is currently no cure for Duchenne.

About Parent Project Muscular Dystrophy

Duchenne is a fatal genetic disorder that slowly robs young men of their muscle strength. Parent Project Muscular Dystrophy (PPMD) is the largest most comprehensive nonprofit organization in the United States focused on finding a cure for Duchenne muscular dystrophy—our mission is to end Duchenne.

We invest deeply in treatments for this generation of young men affected by Duchenne and in research that will benefit future generations. We advocate in Washington, DC, and have secured hundreds of millions of dollars in funding. We demand optimal care, and we strengthen, unite and educate the global Duchenne community.

Everything we do—and everything we have done since our founding in 1994—helps boys with Duchenne live longer, stronger lives. We will not rest until every young man has a treatment to end Duchenne. Go to www.ParentProjectMD.org for more information or to learn how you can support our efforts and help families affected by Duchenne.

About Santhera Pharmaceuticals

Santhera Pharmaceuticals (SIX: SANN) is a Swiss specialty pharmaceutical company focused on the development and commercialization of innovative pharmaceutical products for the treatment of orphan mitochondrial and neuromuscular diseases. Santhera develops Catena®/Raxone® as treatment for patients with Leber's Hereditary Optic Neuropathy (LHON), Duchenne Muscular Dystrophy (DMD) and Primary Progressive Multiple Sclerosis (PPMS) and omigapil for Congenital Muscular Dystrophies (CMD), all areas of high unmet medical need for which no therapies are currently available. For further information, please visit the Company's website: www.santhera.com.

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