BioMarin Prices Orphan Drug at $702,000, Promises Big Discounts

(Bloomberg) -- BioMarin Pharmaceutical Inc. won approval from U.S. regulators for a treatment for an ultrarare pediatric brain disorder. The therapy will have an extraordinary price tag.

At $27,000 per biweekly infusion, the drug, called Brineura, will cost about $702,000 a year. BioMarin said Thursday that most patients who have the disorder, known as Batten disease, are on federal assistance programs like Medicaid, and that the price after mandatory government discounts will be $486,000.

Even with discounts, Brineura would be one of the most expensive drugs in the world. Treatments for rare genetic diseases, such as Horizon Pharma Plc’s Ravicti for urea cycle disorders, carry some of the highest prices in the industry. Ravicti has a list price of $556,000, according to the company.

In a clinical trial, patients getting BioMarin’s drug saw a far slower rate of decline than is typical for such patients. Previously, there was no available therapy for children with Batten disease, which leads most patients to die between the ages of eight and 12. An estimated 20 infants are born with the disease in the U.S. each year, according to the company.

Many drugmakers have come under fire in the past year for the prices of their medications, with politicians including President Donald Trump weighing in on the issue. The drug industry has argued that it needs to be adequately rewarded for the risks and costs that come with developing novel therapies, and have warned that without sufficient reimbursement, investors will stop supporting the industry.

To contact the reporter on this story: Caroline Chen in San Francisco at cchen509@bloomberg.net.

To contact the editors responsible for this story: Drew Armstrong at darmstrong17@bloomberg.net, Timothy Annett