Sarepta cited two safety issues when announcing a complete response letter for its exon 53 skipping DMD drug golodirsen, but the US FDA's decision may be a result of the Exondys 51 approval from 2016, analysts suggest.
The US Food and Drug Administration's rejection of Sarepta Therapeutics Inc.' Duchenne muscular dystrophy (DMD) treatment golodirsen came as a shock to many, raising questions about whether the fallout from the Exondys 51 (eteplirsen) review factored into the agency's decision.
Sarepta announced 19 August that it received a complete response letter (CRL) for golodirsen, which is designed to treat DMD patients who have genetic mutations that are amenable to exon 53 skipping
Patients from Asia saw a differential treatment effect versus non-Asian regions in the Phase III multiregional study, with an adverse survival trend seen in the latter group, the FDA said about the lymphoma drug.
Brensocatib, Insmed's investigational treatment for non-cystic fibrosis bronchiectasis, is among the latest products that have been filed for review by the European Medicines Agency for potential EU marketing approval.
Advisory committee for Capricor’s deriamocel planned, regenerative medicine advanced therapy designations proliferate for osteoarthritis, and the ranks of dual RMAT/breakthrough therapy designation holders grow.
