CIFFREO Study Fails Primary End Point, Gene Therapy for Duchenne Shows Promise, Deflazacort Generic Approved by FDA

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Welcome to this special edition of Neurology News Network. I'm Marco Meglio.

According to a new announcement, fordadistrogene movaparvovec (previously known as PF-06939926), Pfizer’s investigational mini-dystrophin gene therapy, did not meet its primary end point of improvement in motor function among ambulatory patients with Duchenne muscular dystrophy (DMD) in the phase 3 CIFFREO study (NCT04281485).1 The company noted that it will continue to closely monitor all participants enrolled in CIFFREO, and will evaluate appropriate next steps for this program. In the trial, the gene therapy failed to achieve the primary end point, which investigators assessed by the change from baseline in the North Star Ambulatory Assessment at 1 year following treatment. The findings also did not show a significant difference between patients treated with the therapy and placebo in the secondary end points, which included 10-meter run/walk velocity and time-to-rise from floor velocity. The company noted that the overall safety profile of the treatment was manageable, with mostly reports of mild to moderate adverse events (AEs), and that treatment-related serious AEs generally responded to clinical management.

Data from an early-stage trial showed that treatment with GNT0004 (Genethon), an investigational adeno-associated virus (AAV8) vector-based gene therapy, was safe and well tolerated among patients with Duchenne muscular dystrophy (DMD), with encouraging results on microdystrophin expression and functional improvement. Based on the recommendation from an independent expert committee, Genethon is preparing the pivotal part of the clinical trial in the EU. The trial is unique in that it combines phases 1, 2, and 3 with a dose-escalation phase, followed by a pivotal phase at the selected dose. To date, 5 ambulant boys aged between 6 and 10 have been treated with GNT0004, with 2 receiving only the first dose and 3 receiving the second as well.

According to a new announcement, the FDA has approved a new generic version of deflazacort (Emflaza; PTC Therapeutics), a treatment indicated for patients 5 years of age and older with Duchenne muscular dystrophy (DMD). The new generic, which allows for greater access and improved cost-effectiveness, is from Cranbury Pharmaceuticals, a wholly-owned subsidiary of Tris Pharma. Deflazacort oral suspension, a corticosteroid indicated for DMD, has been on the market since 2017 under the name Emflaza and manufactured by PTC Therapeutics. It has several noted warnings and precautions, including alterations in endocrine function, immunosuppression and increased risk of infection, alterations in cardiovascular/renal function, gastrointestinal perforation, behavioral and mood disturbances, and effects on bones, among others.

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