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.
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