June 23 Quick Takes: FDA unveils neurogenerative disease action plan

 FDA released an action plan for rare neurodegenerative diseases including amyotrophic lateral sclerosis (ALS). The five-year plan, developed in accordance with the Accelerating Access to Critical Therapies for ALS Act (ACT for ALS), calls for establishing a task force, creating a public-private partnership, developing “disease specific science strategies” and “leveraging” FDA’s current regulatory science initiatives. The blueprint includes an ALS science strategy with a focus on improving characterization of disease pathogenesis and natural history, facilitating patient access to new therapies and improving clinical trial infrastructure and “agility to enable early selection of promising therapeutic candidates for further development, optimize clinical trial design, improve access to the trials, streamline clinical trial operations, and reduce the time and cost of drug development.”

Sarepta Therapeutics Inc. (NASDAQ:SRPT) said it would propose changes to its monitoring of the 5051-201 trial following a serious adverse event of hypomagnesemia that led FDA to place a hold on Duchenne muscular dystrophy therapy vesleteplirsen (SRP-5051). Vesleteplirsen is a peptide-conjugated phosphorodiamidate morpholino oligomer designed to bind to exon 51 of dystrophin pre-mRNA, excluding this exon during mRNA processing, and leading to production of an internally shortened functional dystrophin protein. ...