US FDA Approves New Drug For Very Rare Childhood Disease

There is a group of birth defect disorders known collectively as Batten disease. It is a rare condition that, mostly, affects the nervous system; initial symptoms include language delay, epilepsy, and ataxia. Secondary symptoms include myoclonus and vision loss.

Children who inherit this condition typically need a wheelchair by the time they reach late childhood and, unfortunately, do not survive past their teenage years. Although the condition is severe, the condition is extremely rare, reported in only about 1 in one million US residents.

Well, this week, the United States Food and Drug Administration have approved a new drug to treat ceroid lipofuscinosis type 2 (CLN2), which is one of the disorders named in the Batten disease group. This medication will be sold under the label Brineura (cerliponase alfa) to treat this singular form of Batten disease. The treatment is the very first to ever be approved to treat the slow-to-walk ability in pediatric patients 3 years of age or older who show symptoms of Batten disease.

Julie Beitz, MD, is the director of Office of Drug Evaluation at the US Food and Drug Administration’s Center for Drug Evaluation and Research. She notes, “The FDA is committed to approving new and innovative therapies for patients with rare diseases, particularly where there are no approved treatment options. Approving the first drug for the treatment of this form of Batten disease is an important advance for patients suffering with this condition.”
According to the FDA’s statement, the recommended dose for this medication will be 300 mg for those patients age 3 and older, to be administered once every other week, with an electrolyte infusion to follow.

The most common adverse reactions for this include fever, abnormal ECG readings (bradycardia, hypersensitivity, changes in CSF protein levels, seizures, headache, pleocytosis, vomiting, hematoma, and irritability.
In addition, BioMarin RareConnections, a resource available to patients and families, provides a variety of personalized support services at no cost to patients, including education on CLN2 disease and Brineura, and coordination of additional services, such as information about financial assistance programs.

Nationwide Children’s Hospital attending pediatric neurologist—and principal Brineura study investigator—Emily de Los Reyes, MD, comments, “CNL2 is a devastating diagnosis that robs families of life with their children much too young. Recent announcement gives my patients and their families hope.”

Furthermore, Batten Disease Support and Research Association executive director, Margie Frazier, PhD, LISW-S, notes, “The approval of Brineura is an extraordinary medical breakthrough for the CLN2 Batten community who have been waiting for this moment for more than a century when the condition was first described. We appreciate BioMarin’s commitment and partnership to the CLN2 Batten community and investing the resources needed to bring this pivotal treatment to families.”

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