The Food and Drug Administration's (FDA) recent approval of Sarepta Therapeutics' Vyondys 53 for Duchenne muscular dystrophy has unexpectedly brought Biogen's experimental Alzheimer's disease treatment, aducanumab, back into the spotlight. This development follows the FDA's surprising reversal on Vyondys 53, which was initially rejected due to safety concerns but later approved after an appeal to the Office of New Drugs.
Vyondys 53 targets a small group of young boys unable to produce a protein essential for muscle growth and function. Its approval, despite minimal evidence of efficacy and lingering safety concerns, has led to speculation about the FDA's potential leniency towards Biogen's aducanumab. Biogen's application for aducanumab is based on a Phase 3 study that, while declared positive, has been met with skepticism due to its statistical analysis and the contradictory results of a similarly designed trial.
Analysts have noted the FDA's neurology division, under Dr. Billy Dunn, has a reputation for being stringent. However, the approval of Vyondys 53 after its review was moved to the Office of New Drugs suggests a possible shift in the FDA's approach. This has led to questions about whether aducanumab could benefit from a similar flexibility, despite the lack of a precedent like that set by Sarepta's earlier drug, Exondys 51.
While some analysts see the Vyondys 53 approval as a positive sign for aducanumab, others caution against drawing direct parallels. They highlight the differences in patient populations and the absence of a precedent for aducanumab's approval based on mixed data. The debate continues as Biogen prepares to submit aducanumab for FDA review, with the Alzheimer's community eagerly awaiting the outcome.
