BioMarin hits snag with latest rare disease drug
- The U.S. Food and Drug Administration has extended the Prescription Drug User Fee Act (PDUFA) goal date for Biomarin's Brineura (cerliponase alfa) by three months. The delay has pushed the approval date back to April 27, 2017.
- The regulatory action is in response to the FDA requesting further data from the company. Biomarin had to submit results that followed patients out to 81 weeks of treatment—the original Biologics License Application (BLA) only included data through 48 weeks. The FDA considers the new data a "major amendment" to the BLA and needs further time for review.
- The regulatory agency said that an Advisory Committee meeting would be required to review the drug and would be announced at a later date in the Federal Register. FDA uses the expert panels to weigh in on drugs that don't have a clear cut path to approval. While the agency often follows their recommendation, they are not required to.
In March 2016, BioMarin announced 48-week data from its Phase 1/2 pivotal study. This trial showed a reduction in clinical decline of motor and language function of around 80% less than the untreated population, and disease stabilization in 15 of the 23 patients. The company used this data as the basis of a BLA, with a Prescription Drug User Fee Act (PDUFA) date of 27 2017, and an MAA in Europe, projecting an approval date in Europe in the first half of 2017.
The FDA’s declaration of the 81-week data as a major amendment and the moving of the PDUFA data is a major setback in the progression towards the market. According to BioMarin, this data is consistent with the data already submitted, and the FDA has not communicated its rationale for declaring this submission a major amendment.
BioMarin has already had a challenging year. After $66 million and years of investment, it ditched its Duchenne muscular dystrophy (DMD) drug drisapersen in May 2016 after rejection by the FDA and clear indications from the EU that approval would not be forthcoming in Europe. The failure of its DMD program was a major hit to both the company and the community—forcing BioMarin to change gears and focus on other drugs in its pipeline.
Brineura is a recombinant form of the enzyme TPP1, and is used in replacement therapy in CLN2 disease, a form of Batten disease. The market for Brineura is just 1200-1600 children, allowing the company to garner an Orphan Drug designation for the treatment in both the U.S. and EU.
CLN2 disease is a rare and rapidly progressing, fatal neurodegenerative disease that affects around 1 in 200,000 children. The disorder, which is as a result of mutations in the TPP1/CLN2 gene, means that the child cannot produce the enzyme tripeptidyl peptidase 1 (TPP1). The child, who appears to develop normally at first, begins to show symptoms around the age of two to four, leading to language delay, seizures, loss of the ability to walk and talk, ending with death between 10 and 16 years of age.
- BioMarin Press Release
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