Santhera Receives FDA Fast Track Designation For Raxone®/Catena® To Treat Duchenne Muscular Dystrophy

Santhera Receives FDA Fast Track Designation For Raxone®/Catena® To Treat Duchenne Muscular Dystrophy

santheraSanthera Pharmaceuticals recently announced it received Fast Track designation from the United Stated Food and Drug Administration (FDA)  for its Raxone®/Catena® (idebenone) to address treatment for Duchenne Muscular Dystrophy (DMD). Through the FDA’s Fast Track program, both the development and review of the drug are facilitated, since the has the potential to treat a severe condition, fill unmet medical gaps and treat patients as soon as possible. Santhera also announced that the Phase III trial in DMD (DELOS) met its primary endpoint and proved that Raxone/Catena could delay respiratory function loss common in DMD cases.

Thomas Meier, Santhera’s CEO, commented in a press release: “We are very pleased that the FDA has granted Fast Track designation for Raxone/Catena, which further underscores the unmet medical need for effective treatments for patients with DMD. On the basis of the positive data from our Phase III trial with Raxone/Catena in DMD, we have started to prepare a New Drug Application and plan to meet with the FDA in the coming weeks to discuss our NDA dossier in a pre-NDA meeting.”
Raxone/Catena, is a substrate for the enzyme NAD℗H:quinone oxidoreductase (NQO1), which makes it capable of supplementing cellular energy levels.

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The findings were the result of the DELOS clinical trial, a double-blind, randomized, placebo-controlled, Phase III trial that treated 64 patients (American and European) suffering with DMD with the therapy are that were not receiving concomitant corticosteroids. Patients were between 10 and 18 years old and received for 52 weeks Raxone/Catena tablets (900 mg/day) or matching placebo.

Santhera Pharmaceuticals is a Swiss-based pharmaceutical firm currently developing and commercializing innovative products to address treatment of neuromuscular and orphan mitochondrial diseases. The hope is that the data presented during Dr. Buyse’s anticipated presentation will spur continued development of Idebenone as a viable treatment for DMD.

DMD is a devastating type of muscle degeneration. It is an X-linked genetic disease and its incidence is about 1 in 3,500 live born males worldwide. The disease is characterized by the loss of dystrophin, which ultimately leads to progressive muscle weakness, early morbidity and mortality due to heart failure.

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