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A Drug Was Approved for a Fatal Childhood Disease–But Costs $700,000


[DIGEST: Bloomberg, Fortune, fda.gov, BizJournals, Biospace; FDA)

The FDA approved a new drug for a form of Batten disease, a rare inherited neurodegenerative condition which rarely allows children to live past their teens. But the drug retails at over $700,000 per year. This, and other drugs that treat rare diseases, are protected by what is known as the Orphan Drug Act. Originally created to encourage research and production of treatments for rare diseases, the Act has inadvertently produced monopolies that allow prices to skyrocket out of control, and corruption among drug companies as they try to push more product onto a tiny market.

FDA Approved Brineura

In April of 2017, the FDA approved BioMarin Pharmaceutical Inc.’s Brineura, the first drug treatment for ceroid lipofuscinosis type 2 (CLN2), a form of Batten disease. The symptoms, which typically begin between ages two and four, include language delay, epilepsy, loss of coordination and vision, and muscle twitches. Loss of motor skills later affects the patient’s ability to sit and walk. Children with CLN2 often require a wheelchair by late childhood, rarely surviving past their teens. Approximately 20 children each year are born with CLN2 in the United States, with about 500 total U.S children currently suffering from the disease.

CLN2 prohibits patients from being able to produce the specific enzyme that allows neurons to recycle in the brain properly. For most patients, Brineura provides enzyme replacement therapy to diminish the progression of the disease.

“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, M.D., 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.”

Credit: Source.

To that end, Brineura received Priority Review status, shaving four months off the typical ten month FDA review, and Breakthrough Therapy designations, allowing Biomarin to fast-track the FDA process. Bineura also received a Rare Pediatric Disease Priority Review Voucher to urge development of additional drugs for rare diseases; this can be transferred for a Priority Review for a different drug or sold to another drug company – the highest to date brought $350 million.

The FDA also granted Brineura Orphan Drug status, which provides financial incentives for development of drugs for rare diseases—including grants and tax incentives to defray costs of testing and producing drugs.  

Yet BioMarin has priced Brineura at $702,000 per year, or $27,000 in bi-weekly infusions, even though patients will pay much less than that after mandatory discounts rebates and

To read more, please continue to page 2.

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  • Amy McElroy is a contributing editor and writer for Rewire Me. She has written for print, radio, and online publications such as The Bold Italic, The Billfold, Noodle, Cosmopolitan, BlogHer, and others. Her website, amyjmcelroy.net, lists her editorial services. She’s on twitter at @amyjmcelroy. Amy balances her work at the computer by teaching yoga and fitness.

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