Acadia becomes the latest to fall foul of the US FDA's apparently increasingly strict stance.
Since 2010 14% of US accelerated approvals have been granted to Merck & Co’s Keytruda.
Early data could give clues about whether gene therapies from Sio, Passage Bio or Lysogene will come out on top in this rare inherited disease.
The US regulator will provide verdicts on cancer projects from TG and Athenex, while Sarepta aims for its third Duchenne approval.
Sarepta’s first ever controlled study, of a Duchenne muscular dystrophy gene therapy, brings the markets down to earth.
Early 2021 will see rare diseases dominate for biotech, with Sarepta revealing key data on its gene therapy play.