FDA official says Sarepta 'NOT a good model for other development programs'
In an October 18 slide presentation prepared for the NORD Summit Meeting, John Jenkins, director of the FDA's Office of New Drugs states: "A poorly planned and executed development program for a rare disease misuses valuable patient resources and serves to delay obtaining the knowledge required to understand the benefits and risks of a drug to support regulatory review and approval... Path taken by Sarepta NOT a good model for other development programs." Sarepta (SRPT) recently received approval for Exondys 51, its Duchenne muscular dystrophy treatment, despite criticism that it had not proven a true clinical benefit. In the wake of the medicine's controversial approval, market observers and analysts have debated whether drug candidates from PTC Therapeutics (PTCT) and BioMarin (BMRN) could benefit from the FDA's seemingly loosened approval standards and apparent desire to see Duchenne treatments on the market. Reference Link SRPT PTCT BMRN