US regulators have approved BioMarin Pharmaceutical’s Brineura as the first treatment for a rare inherited form of Batten disease.
Brineura (cerliponase alfa) is the first FDA-approved treatment to slow loss of walking ability in symptomatic paediatric patients with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase-1 (TPP1) deficiency.
CLN2 disease is one of a group of disorders known as neuronal ceroid lipofuscinoses (NCLs), collectively referred to as Batten disease.
Initial symptoms usually include language delay, recurrent seizures and difficulty coordinating movements, and affected children also develop muscle twitches (myoclonus) and vision loss. Patients with this condition often require the use of a wheelchair by late childhood and typically do not survive past their teens.
Brineura is an enzyme replacement therapy that boosts levels of TPP1, the enzyme deficient in patients with CLN2 disease. Clinical trials showed that patients treated with the therapy demonstrated fewer declines in walking ability compared to those untreated.
The FDA granted this application Priority Review and Breakthrough Therapy designations, and Brineura also carries Orphan Drug status, which provides incentives to assist and encourage the development of drugs for rare diseases.