The US FDA’s waiving of an advisory committee is another promising signal that Sarepta's Duchenne gene therapy SRP-9001 could be approved on 29 May and set it up to become a bigger earner than Novartis’s Zolgensma.

Digital maturity of clinical operations at several big pharma firms varies significantly with the gap between best-in-class and the industry average narrowest for site selection tools, recruitment capabilities, and automation, a study has indicated. Digital patient experience tools figure among the least mature capabilities.

Alongside CSL/Behring/uniQure’s Hemgenix (etranacogene dezaparvovec), the European Medicines Agency has given the green light to new drugs for liver cancer, NSCLC, Pompe disease and multiple sclerosis. But it turned down omburtamab for treating the rare cancer, neuroblastoma.  

The FDA and Sarepta have had a controversial history with the accelerated approvals of the firm’s earlier drugs for Duchenne muscular dystrophy – but the company’s investigational gene therapy, SRP-9001, now under a priority review, could be a genuine breakthrough.