European regulators accept FibroGen's anemia drug for review; Passage Bio's lead gene therapy gets more love from the FDA
→ It’s approved in China and Japan, the FDA plans to make its decision by December and now the EMA has accepted FibroGen‘s anemia drug, roxadustat, for review. The development triggered a milestone payment of $130 million by partner Astellas to FibroGen. The drug is engineered to stimulate the production of red blood cells by mimicking the effect of high altitude in humans.
→ Passage Bio, which works closely with Jim Wilson’s Gene Therapy Program at the University of Pennsylvania, has secured the FDA‘s rare pediatric disease designation for its lead experimental therapy, PBGM01, broadly for the treatment of a lysosomal storage disease called GM1 gangliosidosis. The therapy has also been granted orphan drug status by the agency. A Phase I/II study in infantile GM1 patients is expected to kick off in the fourth quarter, Passage Bio said.
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