Children with a rare, devastating brain disease may soon have their first treatment option in BioMarin’s Brineura, up for an FDA decision later this week. And it's likely to instantly join the ranks of the world’s most expensive meds if approved.
Experts from the European Medicines Agency’s Committee for Medicinal Products for Human Use on Friday endorsed the drug to treat Neuronal Ceroid Lipofuscinosis Type 2 disease, a form of Batten disease. The EMA usually green-lights drugs that have CHMP recommendations, but it isn’t required to do so.
In the U.S., Brineura is set for a decision date on Thursday. On a call late last week with analysts, execs at BioMarin said the company is in label discussions with the U.S. agency, according to a note from Piper Jaffray analyst Joshua Schimmer.
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But BioMarin is warning that the med will be near the top of the world’s costliest orphan drugs, and that growth will be slow because it treats an “extremely rare” disease, according to Schimmer. In an effort to boost the drug’s prospects, BioMarin plans to search “ aggressively” for early-stage and minimally symptomatic patients, the analyst wrote.
The disease, CLN2, renders the majority of patients unable to walk and talk by age six. It’s a “rapidly progressing, fatal” brain disease, according to the drugmaker.
European health authorities are expected to make a decision on the med during the current quarter.
If the drugmaker does have trouble making a commercial success out of the super-pricey med, it won’t be the first company in that situation. UniQure last week said it’ll pull the plug on its $1-million-plus Glybera after a current marketing license runs out in October, citing “extremely limited” use.
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Glybera, the world’s first gene therapy to win a marketing approval, didn’t generate enough sales to justify the company’s substantial regulatory, commercial and manufacturing costs for the drug.
The FDA previously delayed Brineura’s review action date by three months, requesting updated efficacy data, which BioMarin submitted in September.