With 2nd chance at FDA approval, BioMarin preps for hemophilia gene therapy launch in US

With the recent FDA approval for Sanofi and Sobi’s Altuviiio, the hemophilia A field is heating up. But a subsequent approval for BioMarin's gene therapy Roctavian could bring even more change.

If approved by the FDA's decision deadline of March 31, BioMarin's Roctavian would be the first hemophilia A gene therapy in the U.S. The drug would provide a one-time treatment option for patients who otherwise have to take routine prophylactic treatments and often require transfusions.

Roctavian works by delivering a gene that helps the body produce a protein called factor VIII, which helps the blood clot. For patients with hemophilia, blood doesn't clot properly because of a lack of the protein, raising the risk of serious complications.

During BioMarin’s recent fourth-quarter earnings conference call, company executives delved into their Roctavian outlook. Even ahead of the potential approval, the company is seeing interest from patients in the U.S. 

So far, around 300 U.S. patients have “engaged with BioMarin directly” to learn more about the treatment, CEO Jean-Jacques Bienaimé said on the call. The company will follow up with those patients directly upon the launch, Executive Vice President and Chief Commercial Officer Jeff Ajer added.

The treatment won an approval in Europe last August. So far, BioMarin has secured a major payer agreement in Germany, the first European country where Roctavian will launch, at a price of 1.5 million euros per treatment. A federal agreement on reimbursement is being negotiated.

In the U.S., some uncertainty remains about the exact timing of the launch.

While the drug is currently slated for an FDA decision on March 31, there's a chance the agency might delay its review, BioMarin's president of R&D, Hank Fuchs, M.D., explained on the call.

That's because BioMarin recently submitted three-year phase 3 data on the drug as requested by the FDA. If the agency sees that as a "major amendment" to the application, it could trigger a three-month delay, Fuchs said.

That wouldn't be the first regulatory delay for the medicine. After rejecting the gene therapy in 2020, the FDA requested more details and analyses last June, pushing BioMarin’s resubmission back to September 2022.

Meanwhile, as part of the latest review, the FDA in December conduced an inspection of the company’s manufacturing facility in California, coming back with comments and observations about the production setup. BioMarin believes all of the FDA's findings are "addressable," Fuchs said. 

Before March 31, the FDA will also inspect some of the clinical study sites.

On the subject of reimbursement in the U.S., the company is currently fine-tuning a warranty contract to offer to payers. Under such an agreement, the company would stand behind the drug's efficacy and repay some of the costs if the drug doesn't work as expected.

Gene therapy prices are quite high, and Roctavian will be no different. However, the Institute for Clinical and Economic Review (ICER) has said BioMarin's gene therapy would be cost effective at a price of $2.5 million. That’s because regular prophylaxis adds up over a patient’s lifetime. For instance, ICER estimated the annual cost of managing the disease with Roche’s Hemlibra is $640,000 per year.

Elsewhere in the hemophilia field, CSL and uniQure's hemophilia B gene therapy last November won an FDA approval. That drug sports a $3.5 million list price.

Once payer talks are sorted and the FDA approves Roctavian, the drug will be ready for launch. BioMarin has homed in on a “relatively small number” of the largest and most capable hemophilia treatment centers to dole out the therapy, Ajer said. The company has manufactured enough supply to cover both the U.S. and European markets.

BioMarin projects 2023 revenues for the therapy to come in between $100 million and $200 million based on the "current uncertainty" of U.S. approval timing.