Rare disease drug developer BioMarin Pharamceutical Inc. won regulatory approval Thursday of a drug that promises to change the course of a fatal childhood neurodegenerative disease.
San Rafael-based BioMarin’s Brineura is the first drug approved by the Food and Drug Administration to treat children with CLN2, a form of Batten disease. And patients will pay for the innovation — $702,000 a year, or $27,000 per carton every other week — though the company (NASDAQ: BMRN) said it will offer hefty discounts.
It took four years from BioMarin’s first clinical trial of the drug to FDA approval, a relatively fast timeline in an industry that says drug development generally takes 10-plus years and more than $1 billion.
As a side benefit of approval, BioMarin also will receive a rare pediatric disease priority review voucher, which it can use to cut months off the typical FDA 10-month review of another drug — or it can sell the voucher to another company. BioMarin sold a voucher in 2014 to Regeneron Pharmaceuticals Inc. for $67.5 million.
Available in June, Brineura is an enzyme replacement therapy that slows the rate of decline in most patients with the disease, which leads to seizures, loss of sight and speech and motor skills and, eventually, death by the time children are 8-12 years old.
There are 14 known variations of Batten disease, but CLN2 is believed to be one of the most prevalent subsets of the disease. Every year, about 20 children are born in the United States with the disease; there are about 500 U.S. children overall affected by the disease.
CLN2 patients can’t make a specific enzyme that collects neuronal garbage, so neurons can’t properly recycle in the brain.
BioMarin, led by CEO Jean-Jacques Bienaimé, said the cost of the drug is due to the “innovation factor,” the small number of patients and manufacturing costs.
“Treating children with CLN2 disease requires an extraordinary amount of collaboration between families, hospitals, advocates and physicians,” Bienaimé said in a press release. “We are grateful for the partnership of all those involved and look forward to continuing to work together to make Brineura accessible to children who may benefit.”