BioMarin Pharma's (BMRN) MAA for Brineura in CLN2 Disease Validated by EMA

September 15, 2016 8:42 AM EDT

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BioMarin Pharmaceutical Inc. (Nasdaq : BMRN) announced that the European Medicines Agency (EMA) validated the Marketing Authorization Application (MAA) for Brineura (cerliponase alfa) to treat children with CLN2 disease, a form of Batten disease. Validation of the MAA confirms that the submission is accepted and starts the formal review process by the EMA's Committee for Human Medicinal Products (CHMP).

Earlier this year, the CHMP accepted BioMarin's request for accelerated assessment of the MAA on the grounds that Brineura is of major public health interest with the potential to have a major impact on medical practice for CLN2 patients in Europe. Accelerated assessment has the potential to shorten the EMA's review procedure. However, applications which are initially granted accelerated assessment frequently revert to standard assessment timelines. Assuming a positive opinion from the CHMP and standard assessment timing, a decision from the European Commission is anticipated by the third quarter of 2017. The EMA previously granted Brineura Orphan Drug Designation.

“CLN2 disease is a rapidly progressing, fatal neurodegenerative disease with no approved treatments. Based on the positive results of the pivotal study, we are working to bring this meaningful therapeutic option to patients and families in Europe as soon as possible," said Hank Fuchs, MD, Chief Medical Officer of BioMarin. "We greatly appreciate the CLN2 community’s ongoing support and look forward to continuing to work with regulatory authorities in the coming months.”

Children with CLN2 disease typically begin to present symptoms between the ages of two and four, with the majority of affected children losing their ability to walk and talk by approximately six years of age. Initial symptoms can include language delay and seizures, followed by movement disorders, motor deterioration, dementia and blindness. During the later stages of the disease, feeding and tending to everyday needs become very difficult, and death often occurs between eight and 12 years of age.

Marketing Applications

On July 27, 2016, the U.S. Food and Drug Administration (FDA) accepted for review the submission of a Biologics License Application (BLA) for cerliponase alfa. The Prescription Drug User Fee Act (PDUFA) goal date for a decision is April 27, 2017. The FDA granted cerliponase alfa Priority Review status, which is designated to drugs that offer major advances in treatment or provide a treatment where no adequate therapy exists. Cerliponase alfa was previously granted Orphan Drug Designation and Breakthrough Therapy Designation by the FDA. The Agency has advised that they plan to hold an advisory committee meeting at a date to be confirmed per their usual practice of notification in the Federal Register.

Early Access Program

BioMarin has implemented an early access (compassionate use) program as planned to provide experimental drug for additional CLN2 patients prior to obtaining marketing approval. The program is limited in scope and number of participants, and is being conducted under a protocol. The program initially is being conducted at centers that have participated in the cerliponase alfa study. The program began in August 2016 in Hamburg, Germany and Columbus, OH, U.S.A. We continue to work on opening the other sites, while adhering to specific legal and regulatory procedures for each country. In order to assure fairness in inclusion, enrollment decisions will be made independent of BioMarin. In addition, the identities of participants are confidential to protect the privacy of the patients and families.

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