BioMarin’s EU nod another lift for budding gene therapy sector

The European Medicines Agency (EMA) endorsement of BioMarin’s hemophilia A gene therapy comes days after the US recommended two bluebird bio gene therapy products.

The Agency’s Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for Roctavian (valoctocogene roxaparvovec), laying the ground for the approval of the first gene therapy for the treatment of hemophilia A.

Roctavian is based on an adeno-associated virus (AAV) containing the gene for factor VIII, which, once administered as a one-off therapy, carries the gene into the liver cells, enabling them to produce the missing factor VIII.

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BioMarin, which originally licensed the candidate from University College London (UCL) and St. Jude Children’s research Hospital in February 2013, said a final approval decision is expected from the European Commission in Q3 2022.

The firm submitted Roctavian to the US Food and Drug Administration (FDA) for approval in 2019 but announced in August 2020 that the Agency issued a complete response letter (CRL) asking for more data on the durability of the product. BioMarin’s resubmission in the US has been subject to several delays.

“Today’s announcement bodes well for the company that could potentially have Roctavian approved in Q3 22 in the EU, assuming a positive outcome,” SVB Securities analyst Joseph Schwartz said in a note.

“As for the US, we expect BioMarin to submit their BLA [Biologics License Application] in September. Assuming a 6-month review process, BioMarin could also be approved in ~March 2023.”

Burgeoning gene therapy sector

The news comes less than two weeks after FDA Advisory Committees (AdComs) voted overwhelmingly to endorse two gene therapies in development by bluebird bio: elivaldogene autotemcel (eli-cel) for cerebral adrenoleukodystrophy (CALD) and betibeglogene autotemcel (beti-cel) for beta-thalassemia.

It thus represents another major boost to the gene therapy space; a sector that has yet to live up to the optimism at the end of the last decade.

Since AveXis’s Zolgensma (onasemnogene abeparvovec) won approval in 2019, the sector has appeared to have been in somewhat of a tailspin, with a series of adverse events associated with numerous AAV-based candidates. For further details of the problems affecting the gene therapy sector, download the Insider Gene Therapy 2022 Ebook here.