Failure of the EMBARK confirmatory trial convinced US FDA reviewers the Duchenne muscular dystrophy gene therapy did not show clinical benefit, but biologics center director Peter Marks invoked totality of evidence to grant full approval.

With several new drugs progressing for the estimated 70,000 patients in China with DMD, a recent setback for Nippon Shinyaku's contender and progress for Chinese gene therapy developers are adding both uncertainty and likely competition in an untapped market with pressing medical need.

The success of Enhertu led Daiichi Sankyo to raise its annual forecast for the fiscal year ending 31 March, while Eisai is readying for Leqembi’s steady US growth. Meanwhile, Astellas, Chugai and Shionogi balanced growth for some products with declines for others.

Commissioner says more judges have recently stepped in and ‘acted like they were FDA.’ Concern is heightened as Supreme Court is to rule on whether to overturn or limit Chevron deference to agency decisions.