Look back at pharma news in the week to August 24

On the regulatory front last week, there was a major setback for Sarepta Therapeutics on Monday, after the US Food and Drug Administration issued a complete response letter (CRL) for its Duchenne muscular dystrophy candidate Vyondys 53 (golodirsen). On a positive note, there was FDA approval for the TB Alliance’s pretomanid treatment-resistant tuberculosis (TB). Research news included that the NEPTUNE trial of AstraZeneca’s Imfinzi and tremelimumab had once again failed in lung cancer.

The Sarepta fairy tale takes a worrying turn

Strike three for Sarepta, which Monday evening revealed the FDA had declined to approve its Vyondys 53, the company’s next-to-market Duchenne muscular dystrophy (DMD) therapy. Coming in the wake of news of a substantial delay to its gene therapy project, SRP-9001, which was then hit by a safety scare a couple of days later, it is little wonder that August has proved a torrid time for the group’s stock, commented Amy Brown writing on Vantage, the editorial arm of the Evaluate group.