Keyword: Sarepta Therapeutics
Sarepta’s chief Doug Ingram has come out against accusations that the FDA is biased against the company after its recent drug rejection.
President Donald Trump is set to pick oncologist Stephen Hahn, currently at MD Anderson Cancer Center in Houston, as the next FDA commissioner.
Sarepta will negotiate a warning for golodirsen rather than carry out more studies. But the implications of the rejection range beyond golodirsen.
The FDA rejected the New Drug Application for golodirsen, the follow-up to Exondys 51, Sarepta’s first treatment for Duchenne muscular dystrophy.
Landos Biopharma picked up $60 million to propel its lead program into phase 2 for ulcerative colitis and Crohn's disease.
Lysogene is licensing its Sanfilippo syndrome gene therapy to Sarepta for $15 million upfront and up to $125 million in potential milestones.
Two months after Sarepta was hit with a clinical hold by the FDA, the rare disease biotech has seen this lifted after sorting out a quality blip.
Another gene therapy alliance shows that Sarepta won’t allow an issue with its lead gene therapy candidate to dampen its enthusiasm for the field.
Despite posting promising early data, Summit Therapeutics' Duchenne muscular dystrophy drug missed its primary and secondary endpoints in a phase 2 trial.
The data, taken from three patients, showed a 38% increase in a muscle protein that boys with DMD usually lack.