Batch of new drug EMA/CHMP recommendations for May 2021

Following its May 2021 meetings, the European Medicines Agency’s human medicines committee (CHMP) recommended eight even medicines, including one gene therapy and one generic, for approval. Final decisions on approval will be announced by the European Commission, which usually occurs within two or three months.

The Committee recommended granting a marketing authorization for Skysona (elivaldogene autotemcel), from US biotech bluebird bio (Nasdaq: BLUE), for the treatment of early cerebral adrenoleukodystrophy (CALD) for patients without a matched sibling hematopoietic stem cell donor. As Skysona is a gene therapy, the CHMP’s positive opinion is based on an assessment by the EMA’s Committee for Advanced Therapies (CAT).

The Committee adopted a positive opinion, recommending the granting of a marketing authorization under exceptional circumstances for Bylvay (odevixibat), from US firm Albireo Pharma (Nasdaq: ALBO), for the treatment of progressive familial intrahepatic cholestasis (PFIC) in patients aged six months or older, and could be the first non-surgical treatment for this rare disease if it gains final approval.