Rare Disease Spotlight: MPS & DMD — a BioCentury podcast

An inflection point for MPS, another controversy for DMD

A biomarker breakthrough could shave years off the development of therapies for a group of devastating, progressive ultrarare diseases. On the latest BioCentury This Week podcast, BioCentury’s editors discuss FDA’s agreement to allow biopharma companies to use a biomarker as the basis for accelerated approval of neuronal mucopolysaccharidoses therapies, which marks an important inflection point, both for MPS and rare diseases in general.

The editors also assess FDA’s decision to grant full approval to and expand the label of Elevidys delandistrogene moxeparvovec, a gene therapy from Sarepta Therapeutics Inc. (NASDAQ:SRPT) for Duchenne muscular dystrophy. The decision, which establishes the product’s microdystrophin expression levels as the benchmark to beat for the next gene therapies, ignites another controversy for FDA in DMD therapies, argues Washington Editor Steve Usdin, revealing a poorly articulated, idiosyncratic decision-making process at the agency.

Finally, they discuss how human genetics is promising to remove some of the risk from neurology drug development and preview BioCentury’s new conference, Grand Rounds, which takes place in September in Nashville, at the interface of academia and industry.

This episode of BioCentury This Week was sponsored by Nxera Pharma Co. Ltd. For information on opportunities to sponsor The BioCentury Show and the BioCentury This Week podcast, please email conferences@biocentury.com.