BioMarin Pharmaceutical, having secured a long-awaited approval from the Food and Drug Administration, says its focus has now turned to launching its new hemophilia gene therapy in the U.S. market.
The FDA on Thursday approved the therapy, known as Roctavian, for certain people with hemophilia A, the more common form of the rare bleeding disorder. While there are other effective treatments already available, the hope surrounding Roctavian has been that, at least for some patients, it could be a one-time fix for their disease.
For BioMarin, the launch will have a significant affect on its financial future. The company has already brought seven other drugs to market — a rare achievement in the biotechnology industry — yet it hasn’t been consistently profitable. Wall Street analysts believe Roctavian could change that, and have marked it as a potential blockbuster product.
BioMarin’s chief commercial officer, Jeff Ajer, told investors during a conference call Thursday afternoon that the first priority of the launch will be getting the country’s largest hemophilia treatment centers to use Roctavian. The FDA approved the therapy for certain people living with severe hemophilia A, a group that totals around 2,500 in the U.S. by BioMarin’s estimates.
Like other approved gene therapies, Roctavian is expensive. BioMarin set its list price at $2.9 million, which Ajer said translates to net revenue of approximately $1.9 million. The company expects to record between $50 million and $150 million in Roctavian revenue this year. BioMarin is also tying the drug’s price to patient outcomes, promising a warranty that will reimburse insurers up to 100% if patients don't respond, and a pro-rated rebate for the first four years if the response wanes.
But unlike most available gene therapies, Roctavian treats a disease with a variety of available options. BioMarin argues that its therapy would likely save the healthcare system money over time. Many hemophilia A patients are currently treated multiple times a week with “replacement Factor,” medicines that supply them with the key blood-clotting protein they’re missing. These treatments are costly too, at about $800,000 per year for the typical patient, according to BioMarin.
Newer, longer-lasting drugs aren’t cheap either. Roche’s Hemlibra, a popular option because it can be given as infrequently as once every four weeks, carries a list price of around half a million dollars.
“We acknowledge that a hemophilia A gene therapy agent is unlike other gene therapies that have been developed for disorders with little to no treatment options,” wrote Joseph Schwartz, an analyst at SVB Securities, in a note to clients ahead of Roctavian’s approval. “However, we believe that [BioMarin’s] perseverance, time spent connecting with the medical community over a longer than anticipated time to market, and extended long-term data could ultimately help the launch.”
Schwartz added that his team commissioned a survey of U.S. physicians and found “considerable interest” in Roctavian. The SVB team has forecasted around $2.2 billion in peak annual sales.
“The product profile of Roctavian presents tremendous value to patients, including bleed control, good safety profile and freedom from the burden of chronic therapy,” Ajer said.
BioMarin recently presented three-year data from the main study that led to Roctavian’s approval. They showed that while the therapy’s effects seem to wane somewhat, patients on the therapy still experienced an 83% reduction in the number of bleeding episodes requiring treatment. The use of replacement Factor was also down 96% compared to when participants entered the study.
Notably, the efficacy data included in the FDA-approved label for Roctavian says the therapy reduced the average of bleeds per year from 5.4 to 2.6, reflecting a 52% decline. Hank Fuchs, head of BioMarin’s research and development, explained on the Thursday call that the FDA used a different statistical method to calculate bleeding rates, hoping to separate Roctavian's impact from other possible confounding factors like the use of replacement Factor.
Some analysts suspected this discrepency was at least partially to blame for BioMarin’s stock price dipping after the announcement of Roctavian’s approval. Shares were trading around $89 apiece late Friday morning, down more than 4% from market’s open Thursday.
“While at face this sent the stock lower, Roctavian's primary selling point — that the majority of patients remain off prophylaxis years after treatment — remains the same,” wrote Stifel analyst Paul Matteis in a note to clients.
Matteis also wrote that, to insurers, what ultimately matters most is how long Roctavian allows hemophilia A patients to live freely without needing Hemlibra or replacement Factor. There, the data continue to be encouraging.
BioMarin anticipates the first doses of commercial Roctavian will be available in two months. Ajer said the time from when a patient first expresses interest in the therapy to when they get it will be affected by their eligibility, location, insurer, and how often they can visit hemophilia treatment centers. But in general, these steps could take between two to five months.