Developing CAR-Ts against solid tumors that are both safe and effective is proving a huge technical challenge, but San Diego biotech Poseida believes it has made important progress.

Close to 90% of the FDA’s 2021 first half novel approvals were cleared in the US before Europe, while the EU’s EMA acted first on only 15% of its first half marketing authorizations for new active substances. Exclusive Pink Sheet analysis explores use of expedited review schemes, special pathways and other comparative trends.

Brazil has established an accelerated pathway for approval of drugs for ultra-rare diseases, encouraging development activity – but the definition of qualifying drugs is narrow. Expansion to include more treatments could boost clinical trials activity and companies’ overall development presence.

Novartis aims to double the addressable market for its spinal muscular atrophy gene therapy – and catch up to competitors – with the intrathecal version known as OAV-101.