Sarepta Therapeutics has faced another setback with the FDA placing a clinical hold on all its investigational trials for limb girdle muscular dystrophy (LGMD). The biopharma had also lost its platform technology designation due to “unreasonable and significant risk of illness or injury” associated with one of its gene therapies.
The FDA cited three deaths potentially related to Sarepta’s products, including a recent death in a 51-year-old man participating in a phase 1 trial for LGMD type 2D/R3. The patient died from acute liver failure after receiving the investigational gene therapy SRP-9004.
Sarepta had been conducting global trials for its experimental gene therapies against LGMD, but will now be discontinuing most of these programs except for one called SRP-9003.
The biopharma plans to explore strategic alternatives, such as partnering with other companies, for the discontinued programs. The FDA has not responded to requests for details on the clinical holds.
Sarepta’s CEO Douglas Ingram downplayed the significance of a recent death related to one of its LGMD assets, saying it was “neither material nor central to the topics at hand.”
The development comes as Sarepta continues to navigate its pipeline restructuring efforts aimed at saving $400 million annually.
Source: https://www.fiercebiotech.com/biotech/sarepta-ldmd-trials-all-hit-fda-hold-amid-newly-surfaced-safety-concerns