Wowsers! FDA Approves BioMarin's Brineura, the First Drug for Batten Disease

Published: May 02, 2017

Wowsers! FDA Approves BioMarin's Brineura, the First Drug for Batten Disease April 28, 2017
By Alex Keown, BioSpace.com Breaking News Staff

San Rafael, Calif.-based BioMarin Pharmaceutical announced that the U.S. Food and Drug Administration (FDA) approved Brineura (cerliponase alfa) for rare disease late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), which is also called tripeptidyl peptidase 1 (TPP1) deficiency. CLN2 is a type of Batten disease.

Although this is not particularly surprising for a rare disease, the drug, called Brineura, is coming with a sky-high price tag of $27,000 per biweekly infusion, or $702,000 per year. The company indicates that most patients with Batten disease are on federal assistance programs such as Medicaid. After mandatory government discounts, the price will likely be $486,000.

Which would still make it one of the most expensive drugs in the world.

According to the Batten Disease Support and Research Association, Batten disease is one of about 50 diseases known as lysosomal storage disorders (LSD). Genetic mutations impair cells’ ability to get rid of wastes. In Batten disease, there is a build-up of proteins and fats (lipids). This results in damaged cells that can result in seizures, visual impairment and blindness, personality and behavior changes, dementia, and loss of motor skills and the ability to walk, talk and communicate.

CLN2 is believed to be one of the most common subsets of Batten disease, of which there are 14 variations. In the U.S., about 20 children are born annually with the disease, with about 500 children in the U.S. affected. Most children with Batten disease die between the ages of 8 and 12 years.

In clinical trials, Brineura, which is an enzyme replacement therapy, slowed the loss of ambulation in pediatric patients three years of age and older with CLN2. The company indicates the drug is the first enzyme replacement therapy to be directly administered to the brain, using intraventricular administration.

The company has also partnered with a commercial laboratory to offer a no-cost genetic testing program called “Behind the Seizure,” as well as tools and resources to educate doctors on the disease. It has also created BioMarin RareConnections, a resource for patients and families, a free personalized support service that includes information about financial assistance programs.

“We thank the FDA for recognizing Brineura’s potential to alter the course of CLN2 disease and its urgency in delivering this treatment to children as quickly and safely as possible,” said Jean-Jacques Bienaime, chairman and chief executive officer of BioMarin, in a statement. “Brineura was approved in under four years from starting the first clinical trial to today, a significant achievement for a condition that progresses so rapidly. Treating children with CLN2 disease requires an extraordinary amount of collaboration between families, hospitals, advocates and physicians. 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.”

In addition to the approval, BioMarin received a Rare Pediatric Disease Priority Review Voucher. This allows a future priority review, generally knocking about four months off the review process from the usual 10. BioMarin can use it for a future drug approval or sell it to another company. In 2014, for example, BioMarin sold a voucher to Regeneron Pharmaceuticals for $67.5 million. In February, Sarepta Therapeutics sold a priority review voucher to Gilead Sciences for $125 million. The most expensive to date was sold by United Therapeutics to AbbVie for $350 million in August 2015.

Last week, the European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use (CHMP) provided a positive opinion regarding Brineura. A decision by the European Commission (EC) is expected by the second quarter of this year.

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