The US Food and Drug Administration (FDA) has expanded its approval of BioMarin Pharmaceutical’s supplemental biologics licence application (sBLA) for enzyme replacement therapy, BRINEURA (cerliponase alfa), to include children aged under three with neuronal ceroid lipofuscinosis type 2 (CLN2) disease.
The move broadens the therapy’s previous indication to reduce the loss of ambulation, which was initially limited to symptomatic children aged three years and above with late infantile CLN2 disease
The treatment is intended for children of all ages with CLN2, irrespective of symptom status.
The sBLA for BRINEURA was supported by data from a Phase II trial known as Study 190-203 which indicated that the treatment slows the decline in motor function and delays disease onset, even in children younger than three.
The open-label, multicentre trial analysed children aged one to six years at baseline over approximately three years.
Results from Study 190-203 demonstrated that intraventricular-administered BRINEURA effectively slowed the progression of CLN2 disease.
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The safety profile of BRINEURA in children under three was consistent with the known safety data of the medicine.
These findings also suggested that initiating treatment before the age of three could delay the onset of the disease.
BioMarin pharmaceutical worldwide research and development president Hank Fuchs stated: “The approval represents a significant step forward in enabling children to be treated with BRINEURA as early as possible, when we can have the greatest impact in altering the natural course of disease.
“We know that every day counts for families affected by serious genetic conditions such as CLN2 disease, which is characterised by a rapid onset of neurodegenerative symptoms. We have been working diligently since BRINEURA’s initial approval to support this expanded use in children of all ages, even before they begin to show symptoms.”
In June 2023, BioMarin’s Roctavian received FDA approval as a one-time therapy in adults with severe haemophilia A.
