BioMarin's Batten disease drug gets a yes at a cost of $702K
- BioMarin Pharmaceutical has snagged a thumb's up for Brineura (cerliponase alfa), its treatment for the ultra-rare, progressive and fatal form of Batten disease known as late infantile neuronal ceroid lipofuscinosis type 2 disease (CLN2). Promotion will start immediately, with the drug becoming available in June.
- BioMarin also gains a Rare Pediatric Disease Priority Review Voucher, which gives its holder access to priority review for drugs that wouldn't otherwise qualify, and which also can be sold to other companies —often for millions.
- BioMarin announced will price the ultra-rare disease drug at $27,000 per infusion, or approximately $702,000 annually. After mandatory government discounts and rebates and factoring in patient compliance, the company estimates the annual U.S. net price will be closer to $486,000 per year.
Brineura (cerliponase alfa) is the first Food and Drug Administration-approved treatment to slow the loss of walking ability in children aged three years and older who have the ultra-rare pediatric brain disorder CLN2. A form of Batten disease, CLN2 affects fewer than one in a million people in the U.S., with 20 children born each year with the disorder. The affected children lose their ability to walk and talk by the age of six, and often die before they reach 12.
This is wonderful news for the parents and families of children born with this devastating disease. However, these days, miracles come with big price tags. Brineura will cost almost half a million dollars a year after discounts and rebates, which BioMarin Pharmaceutical describes as "consistent with other treatments for similar ultra-rare disorders," backing up the price tag with explanations based around severity, clinical benefit, rarity, manufacturing and supply costs and commitment to ongoing safety and efficacy studies.
Credit Suisse analysts predict peak sales of $500 million.
To cushion the blow for the huge price tag, BioMarin is offering a no-cost genetic testing program called 'Behind the Seizure' to support early testing in children who have fits, has put in place physician education around CLN2 disease, and provides patient and family support and education through its BioMarin RareConnections program. The company will also provide a co-pay program for Brineura patients.
Ultra rare disease drug pricing has for many years been accepted as a necessary evil of developing drugs for very small patient populations. The market has typically born the cost of these drugs as a means of incentivizing the development for orphan diseases. Yet, backlash to pricing in recent years has started to bleed over to orphan diseases as well, with payers pushing back and refusing to cover certain drugs that are priced too high.
The Rare Pediatric Disease Priority Review Voucher, which came as part of the approval package, is also a valuable thing to have. BioMarin can choose to use this to boost one of its own programs, or may, more controversially, sell it on the open market. Last month, Gilead bought a priority review voucher from Sarepta Therapeutics for a princely $125 million, and vouchers have changed hands for as much as $350 million.
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