A newly published report by the HHS inspector general found that a substantial number of drugs given an accelerated approval by the FDA still haven’t proven whether they help patients.

The list price is slightly lower than another ALS therapy approved five years ago, but well above what one drug cost watchdog believes to be reasonable given its purported benefit.

The Swiss company said it would suffer irreparable harm if generics for the multiple sclerosis drug were allowed to enter the market before the higher court reviews the merits of its case.

The resubmission has been long awaited after BioMarin’s original application was unexpectedly rejected by the FDA two years ago. The company expects a decision around the middle of next year.

The biotech, which fell behind Sarepta in developing a Duchenne gene therapy, will merge with AavantiBio, a gene therapy startup run by a former Sarepta executive.

The “Evolution and Innovation in Oncology Therapeutics Development” podcast series explores the role of multi-biomarkers in precision oncology.

The drug, which will be sold as Relyvrio, showed modest benefits in function and survival in testing. It also became the latest test of the FDA's flexibility toward new therapies for neurological disorders.

The application comes a year earlier than previously had been expected, as company says drug reviewers are open to accelerated review based on data from early-stage trials.

Run by the hospital’s Healey Center, the platform trial is designed to evaluate several prospective treatments simultaneously and get answers on their potential more quickly.

Positive results from a late-stage clinical trial of the drug, called lecanemab, were a surprising success for a field used to setbacks. But doctors and researchers point out that the reported benefit appears relatively modest.

The biotech will part with its lead drug candidate, an anti-inflammatory small molecule, in Novo Nordisk’s bet on inflammasome treatments.

Expectations are now higher for amyloid-clearing drugs from Eli Lilly and Roche after lecanemab showed it can slow the disease’s progression in a large Phase 3 clinical trial.