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BioMarin nabs FDA approval for orphan drug Brineura in ultrarare pediatric disease

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After a three-month review delay at the FDA, BioMarin Pharmaceutical on Thursday won the agency’s backing for its orphan drug Brineura, now approved to treat the ultrarare CLN2 disease.

With the approval, BioMarin is prepping for a launch by early June, the California-based biotech said in a release. The drugmaker didn’t initially disclose pricing, although execs told analysts last week the med would fall in the “high end” of orphan drug prices, Piper Jaffray analyst Joshua Schimmer wrote in a note.

RELATED: BioMarin sees ‘high end’ pricing on Brineura ahead of FDA decision

U.S. regulators approved the med based on efficacy data in 22 patients and safety data in 24 patients. The FDA’s Julie Beitz, director of the Office of Drug Evaluation III, said in a statement that the drug represents an “important advance” for patients, who previously had no treatment options.

CLN2, a form of Batten disease, is a rapidly progressive fatal brain condition. Children born with the disease completely lose the ability to walk and talk at around 6 years of age, BioMarin says, and often die between the ages of 8 and 12.

Brineura’s approval requires BioMarin to conduct follow-up safety studies in patients under 2 years of age, plus a long-term study tracking real-world safety over a minimum of 10 years. Twenty children are born with the condition each year in the U.S., BioMarin says.

BioMarin also won a rare disease priority review voucher with its Brineura approval, a significant award that could be worth hundreds of millions of dollars in resale to another biopharma.

Less than a week ago, experts in the European Medicines Agency’s Committee for Medicinal Products for Human Use recommended the med, sending it to EU authorities for a final sign-off. EMA isn’t required to approve drugs recommended by CHMP, but it usually does.

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After the CHMP decision on Friday, BioMarin execs held a conference call to let analysts know they were in label discussions with U.S. authorities and that the drug would feature a super-high price tag. They also warned of slow growth because CLN2 disease is so rare.

But BioMarin has a plan to search “aggressively” for early-stage and minimally symptomatic patients to help with Brineura’s chances of commercial success.

The FDA previously delayed Brineura by three months, requesting BioMarin submit updated efficacy data. The company submitted that information back in September.

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