Sarepta says US FDA said ‘no,’ but agency repeatedly urged the company to pursue the approach.
Sarepta Therapeutics Inc. and FDA appeared at odds over whether a placebo-controlled trial should have, or could have, been conducted as part of the later development stages of Exondys 51 in Duchenne muscular dystrophy.
A Sarepta official said recently that the company, patients and physicians concluded that a Phase III placebo-controlled trial would not...
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The European Medicines Agency’s human medicines committee, the CHMP, has recommended 14 new medicines for pan-EU approval this month, including five orphan medicines.
The European Medicines Agency says that Eli Lilly's Alzheimer's disease drug should be approved for use in the EU, now that it has re-examined the negative opinion it previously adopted.
The European Medicines Agency recommended against pan-EU marketing authorization of Roche/Sarpeta’s gene therapy Elevidys for the treatment of Duchenne muscular dystrophy as the US reported another death in an Elevidys patient.
George Tidmarsh has extensive experience in the biopharmaceutical industry and led several drugs to FDA approval.
The FDA will take a hard line on trial design and site selection to ensure applicability to the US, Oncology Center of Excellence Director Richard Pazdur said during a meeting on GSK's Blenrep.
Eli Lilly and PharmaMar were set to make their case before the European Medicines Agency this week as part of re-examination procedures for their respective drugs Kisunla and Aplidin.
Replimune's RP1 oncolytic immunotherapy became the third regenerative medicine to receive an FDA complete response letter this month as the agency appears to re-visit previous agreements about accelerated approval.
