European regulators push Biogen, CRISPR drugs toward approval

European regulators push Biogen, CRISPR drugs toward approval

  17 Dec 2023

The committee that recommends whether drugs should be approved in Europe has thrown its support behind the first medicine based on CRISPR gene editing technology, issuing on Friday a positive opinion of the treatment, known as Casgvey.

The committee’s decision positions Casgevy, a blood disease therapy developed by Vertex Pharmaceuticals and CRISPR Therapeutics, to likely win approval from Europe’s drug regulator in a matter of months. That regulator, the European Medicines Agency, is expected to make a decision in February.

In issuing its positive recommendation, the committee is following similar decisions from regulators in the U.K. and U.S., both of which have cleared Casgevy for use in recent weeks. The drug would similarly be available for transfusion-dependent beta thalassemia or severe sickle cell disease patients who are at least 12 years of age and can’t get a stem cell transplant.

As with the U.K., Casgevy’s potential clearance in Europe would be “conditional,” meaning it will only be upheld if the drug’s benefits are confirmed in further testing. To satisfy European regulators, Vertex and CRISPR must submit results from ongoing pivotal trials by August 2026, as well as results from a lengthy follow-up study, among other data. Treated patients will be followed for up to 15 years to evaluate the long-term effects and safety of Casgevy. Vertex and CRISPR also have to submit results from a study based on data from a patient registry.

Separately, the committee recommended approval of Skyclarys, a treatment for the rare condition Friedreich’s ataxia that’s important to Biogen’s future.

Biogen acquired the medicine through its $7.3 billion purchase of Reata Pharmaceuticals, believing the treatment could help offset the shrinking sales from its core business. But Skyclarys’ fate in Europe was uncertain at the time: the drug was approved in the U.S. in March, but only after years of back-and-forth between Reata and the Food and Drug Administration.

A clearance in Europe is deemed by Wall Street analysts as crucial for justifying the price paid for Reata and cementing its chance of becoming a blockbuster drug. Yet analysts and investors have been skeptical of its chances, as regulators there have sometimes been more cautious in evaluating neurological disease drugs. They recently rejected another brain disease medicine, Amylyx Pharmaceuticals’ ALS treatment Relyvrio, for instance.

“While this in and of itself is not necessarily hugely needle-moving for the company, we believe it is important from an optics standpoint,” as it helps “reaffirm” the business development capabilities of Biogen’s management and the returns the company will get from the Reata deal, wrote RBC Capital Markets analyst Brian Abrahams.

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