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.  

Four of the six products up for a marketing authorization opinion from the EMA’s advisory committee, the CHMP, this week are orphan drugs. One of them, omburtamab, was recently turned down by a US FDA advisory panel.

Entrada gets $250m cash and equity up front under four-year deal, which extends its runway and will enable it to focus on Duchenne muscular dystrophy. Vertex further diversifies its pipeline with a novel drug class.

The latest drug development news and highlights from the Pink Sheet’s US FDA Performance Tracker