BioMarin's hemophilia gene therapy may have a chance at winning over Europe: analysts

Remember how bluebird bio retreated from Europe after failing to reach reimbursement deals for its gene therapies? Now, one team of analysts believes BioMarin’s hemophilia A gene therapy, Roctavian, may have big potential in the market if the company can overcome the reimbursement hurdle.

A survey of 35 hematologists in Germany, France, Italy and Spain suggests Roctavian may have a “significant opportunity” in the EU, SVB Securities analysts Joseph Schwartz and Joori Park, Ph.D., said in a note Tuesday.

The doctors estimated that Roctavian could attract about 26% of their adult patients with severe hemophilia A at peak, the survey shows. That’s almost the same level of penetration that Roche’s blockbuster antibody drug Hemlibra currently has within the group. For reference, Hemlibra reeled in 741 million Swiss francs sales from Europe last year.

Encouragingly, about half of the doctors have already started evaluating their patients to understand whether they are eligible for potential treatment with Roctavian, according to SVB’s survey.

Doctors’ positive attitude aside, BioMarin still faces the same coverage negotiation hurdle that tripped bluebird in 2021. Despite a European Commission approval in August, BioMarin had yet to treat a commercial patient as of April.

The company is initially targeting Germany. It has begun talking with Germany’s national insurance fund, GKV, to determine Roctavian’s price, Jean-Jacques Bienaime told investors during a call in April. He described their initial interactions as positive. 

In a statement to Fierce Pharma, BioMarin confirmed that it’s still working with GKV to determine a federal price for Roctavian in Germany, with results expected around September.

“[W]e believe the German healthcare system will recognize Roctavian’s value, based on the transformational efficacy observed in a majority of participants across our extensive development program,” BioMarin said. 

Progress is also being made in France and Italy, where BioMarin expects to wrap up negotiations by the end of the year.

BioMarin has made some progress in reaching EU doctors. Ninety-one percent of the SVB-surveyed hematologists have used the agent in clinical trials or are familiar with its clinical data. A third of the doctors said they had contacted BioMarin’s representatives, and that they were satisfied with the interactions. The survey was conducted in May.

Almost all doctors rated Roctavian’s clinical data as impressive, including on the gene therapy’s durability.

There are at least two problems, though. First, doctors viewed the need for steroids following Roctavian treatment as a potential obstacle to adoption. In the drug’s phase 3 trial, 79% of Roctavian patients received corticosteroids to cope with elevated liver enzyme levels after two years, the SVB team noted.

Doctors in Germany appeared to have the strongest concern over steroid use as all of them labeled it as either a moderate or significant impediment to patient adoption.

Second, some doctors were still figuring out the appropriate patients to put on the gene therapy. In Germany, where BioMarin intends to launch first, none of the surveyed doctors there has identified eligible patients.

This delay suggests that Roctavian’s launch might initially be slow in Germany, the SVB analysts said. But considering doctors in other countries have made progress on patient identification, and because of the positive opinions on Roctavian’s clinical profile, the SVB team anticipated the drug’s sales ramp could pick up.

Outside Europe, BioMarin is awaiting an FDA decision by June 30. The FDA in March extended the review timeline by three months to consider the additional three-year data analysis from an ongoing phase 3 trial.

Editor's Note: The story has been updated with a statement from BioMarin.

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