BioMarin nets conditional approval for valrox in Europe, marking the first hemophilia gene therapy
As BioMarin prepares to head back to the FDA with its hemophilia A gene therapy, the EU is sailing forward at full speed ahead.
The European Commission granted conditional approval to the company’s valoctocogene roxaparvovec — more colloquially known as valrox — Wednesday afternoon, making it the first hemophilia A gene therapy approved anywhere. Valrox, to be branded on the continent as Roctavian, will treat adults with severe hemophilia A who need regular treatments to prevent bleeding episodes.
There’s limited info so far about the timeline for confirmatory data. BioMarin said Wednesday that it will provide follow-up data from earlier studies, as well as new results from a confirmatory trial, for which enrollment has completed. But the company did not say when it expects to report such data in a press release.
In an email to Endpoints News, a BioMarin spokesperson did not directly answer questions about when the company would report confirmatory data. But they offered clues hinting at when the results might come. Of note, the spokesperson says the confirmatory data required include:
- 5 years of data from the Phase 3 study, and BioMarin will reach year three this November 2022.
- 2 years of data from the steroid study, and year one data is expected in 1Q23.
Wednesday’s approval follows a positive CHMP opinion in June, with the EMA saying that the therapy provided some benefit for patients more than two years down the road, as well as for a few patients at least five years post-treatment. But regulators hedged at the time, noting they don’t know for sure how long the effects will last.
The confirmatory data are ostensibly expected to help answer that key question. For now, though, BioMarin is touting trial results showing the therapy reduced bleeding episodes by 95% over four years.
Valrox was initially handed down a CRL back in August 2020 after the FDA requested more follow-up data. The move came as a surprise both to the company and to analysts, as regulators sought an additional two years of results showing whether or not the therapy was durable.
With the European approval now in hand, BioMarin says it expects to re-file its BLA by the end of next month. That timeline was pushed back three months in May after the company said the FDA wanted more information, though crucially did not ask for any more clinical trials.
Though submissions following a CRL typically take six months to re-review, the company also said Wednesday it would not be surprised if the FDA takes longer than the standard half-year period.
Durability has proven a key sticking point at the agency in recent months, with seemingly every gene therapy developer positioning such data more prominently to better their approval chances. And as safety issues continue to loom over the field, regulators appear to be scrutinizing such applications closer and closer.
In Europe, regulators plan to monitor patients who take valrox for 15 years to ensure everything remains hunky-dory. The therapy uses an AAV vector to deliver the gene for factor VIII and is expected to carry the gene into the liver cells, helping the blood to clot more easily to prevent or reduce bleeding episodes.
Editor’s note: This article has been updated to include comment from a BioMarin spokesperson.