FDA okays first drug for Batten disease

Constance Williams  Published 2017.04.28  15:49  Updated 2017.04.28 16:03


Biomarin Pharmaceutical won the go-ahead from the U.S. Food and Drug Administration Thursday for Brineura (cerliponase alfa), the first treatment for children who suffer from the Batten disease.

The Batten disease causes mental impairment, seizures, and progressive loss of sight, speech and motor skills.

Brineura is an enzyme replacement therapy to slow down the loss of walking ability in symptomatic pediatric patients three years of age and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase-1 (TPP1) deficiency.

The graphic shows pigmentary lipid in the brain causes the Batten disease in infants.

CLN2 disease is one of a group of disorders known as neuronal ceroid lipofuscinoses (NCLs), often referred to as Batten disease. Children with the disease are unable to produce enough TPP1 enzymes, which plays a role in breaking down certain proteins inside the cells. The build-up of protein deposits in the cells, including nerve cells, damages tissues and lead to degeneration of the brain and retina.

Individuals with this condition often require wheelchairs by late childhood and usually do not survive past their teens. Batten disease is relatively rare, occurring in an estimated two to four of every 100,000 live births in the United States.

“The FDA is committed to approving new and innovative therapies for patients with rare diseases, especially when there are no approved treatment options,” said Julie Beitz, a director at FDA’s Center for Drug Evaluation and Research. “Approving the first drug for the treatment of this form of Batten disease is a significant advance for patients suffering from this condition.”

The efficacy and safety of Brineura was proven in a non-randomized, single-arm dose escalation clinical study in 22 pediatric patients with the CLN2 disease, and was compared to 42 untreated pediatric patients with CLN2 disease from an independent historical control group and had the motor of language symptoms.

Judged by age, genotype, and standard walking ability, Brineura-treated patients, showed fewer declines in walking ability compared to untreated patients in the independent historical control group.

The recommended dose in pediatric patients is 300 mg overseen once every other week by intraventricular and electrolyte infusion, which last approximately 4.5 hours.

Common hostile reactions in patients who are treated with Brineura are slow heart rate, fever, vomiting, seizures, headache, irritability, increased CSF white blood cell count, device-related infection, hypersensitivity, decrease or increase in CSF protein and jitteriness.

Brineura is recommended not to be administered to patients if they show signs of device-related infections such as swelling, bulging of the scalp around or above the intraventricular access device, and extravasation of fluid. Health care providers should routinely test patient CSF samples to detect device infections and monitor vital signs before the infusion starts.

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