Significant progress was made last week for those suffering from Batten Disease (CLN2), a form of lysosomal storage disorder characterized as a rare, rapidly progressing, fatal, neurodegenerative genetic disease in which the majority of affected children lose their ability to walk and talk by the age of six. Both the FDA and the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) have approved BrineuraTM, developed by BioMarin Pharmaceuticals Inc., to treat the motor-language symptoms associated with CLN2.
Children with this disease lack a sufficient amount of the enzyme tripeptidyl-peptidase 1, which aids in breaking down specific proteins. The accumulation of these proteins damages tissues, leading to the deterioration of the brain and retina. An enzyme replacement therapy, BrineuraTM is designed to slow the progression of the disease.
Families affected by the disease are hopeful.
“Today at Noah’s Hope – Hope4Bridget we are celebrating this innovative and transformative therapy for CLN2 Batten children like Noah, Laine and Bridget. Thank you does not begin to express how thankful we are to all who have supported these efforts since 2009 when our children were diagnosed,” commented Tracy VanHoutan. The VanHoutan family lost their son Noah in March of 2016.
“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 advancement for patients suffering with this condition.”
In September 2016, BioMarin reported 81-week data that evidenced the motor-language scores of patients receiving this treatment, had substantially reduced disease progression relative to declines observed historically in untreated children. This data was consistent with earlier 48-week data which reported that patients who received the treatment experienced a reduction in average clinical declines in language and motor functions of roughly 80 percent.
The FDA will require further evaluation of the safety of BrineuraTM in patients under two years of age, and a long-term safety study that will assess patients treated for a minimum of ten years. BrineuraTM has also been granted Orphan Drug designation, and a “Rare Pediatric Disease Priority Review Voucher” intended to promote new drug development for the prevention and treatment of rare diseases.
The marketing authorization for BrineuraTM granted by CHMP also requires an ongoing evaluative study for children under two years old and the creation of a patient registry to monitor the long-term safety of the treatment. To follow will be an adoption of a decision by the European Commission on an EU-wide marketing authorization that includes consideration for potential use by each Member State.