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BioMarin scores a quick FDA OK for its latest rare disease entry — flagged at $702K WAC

Jean Jacques Bienaimé

The FDA has provided a widely expected green light for BioMarin’s Brineura (cerliponase alfa), setting up the rollout for one of the most expensive new drugs to hit the market.

BioMarin $BMRN attracted attention for its latest rare disease drug for its efficacy in treating Neuronal Ceroid Lipofuscinosis Type 2 disease (CLN2), a form of Batten disease. The disease progresses rapidly, with recurrent seizures and vision loss occurring as most victims are quickly left unable to walk by the age of six before eventually killing them. And just days ago the European Medicines Agency’s Committee for Medicinal Products for Human Use provided their thumbs up as well.

The drug was tested in only 22 patients in an abbreviated study, with a comparison on the natural history of several dozen others. And that’s a model that will help define more such studies in the future.

BioMarin CEO JJ Biename noted in a call with analysts Thursday evening  that the approval came “after just 3 years and 8 months in the clinic,” with maximum flexibility from the FDA for an ultra rare disease based on one small clinical study, with a historical comparison, and no expert panel review. And they won a priority review voucher, which could easily be worth $125 million or more, based on recent sales.

The drug is designed to slow loss of ambulation among patients, as the data indicate.

The cost: $27,000 every two weeks, or $702,000 list; an average $486,000 per year after Medicaid discounts are factored. That will be the second most expensive therapy available in the US, right after Horizon’s Ravicti, at $793,632 wholesale, according to a Reinsurance Group of America list based on WAC prices. Spinraza from Biogen is now being sold for $750,000 for the first year, which then drops to half that price.

Piper Jaffray analyst Joshua Schimmer has already noted that the company warned that its list price on this drug will fall on the high side, putting it on our latest list of the world’s 10 priciest drugs, which you can see here. That list is dominated by orphan drugs developed for small patient populations.

BioMarin’s stock, which had essentially baked this approval into the price, was up only slightly at the end of the day.

“The FDA is committed to approving new and innovative therapies for patients with rare diseases, particularly where there are no approved treatment options,” said Julie Beitz, director of the Office of Drug Evaluation III in the FDA’s Center for Drug Evaluation and Research. “Approving the first drug for the treatment of this form of Batten disease is an important advance for patients suffering with this condition.”


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