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 numbers may be down, but Europeans will in future be able to gain access to a number of highly innovative medicines, including several products for hard-to-treat cancers, a gene therapy for severe and moderately severe hemophilia B, and two vaccines against disease caused by respiratory syncytial virus.

The arrival of an indigenous CAR-T cell therapy, a shot at GLP-1s and Parkinson’s disease, strides into adjacencies and an “easy revenue stream” formula for products set to lose exclusivity are some of the striking developments seen playing out in India. Industry leaders, including Cipla’s CEO, share their views with Scrip on some of the key trends in the first instalment of this two-part roundup.