The U.S. Food and Drug Administration (FDA) will review a Biologics License Application (BLA) for cerliponase alfa, BioMarin Pharmaceutical Inc. announced yesterday.
The therapy is being developed for
treatment of CLN2 disease, a fatal neurological disease that affects children. It is a form of Batten disease.
“CLN2 disease is a rapidly progressing, fatal neurodegenerative disease with no approved treatments. The FDA recognized the potential of cerliponase alfa to help address this devastating condition, and we look forward to working closely with the agency over the coming months," BioMarin Chief Medical Officer Dr. Hank Fuchs said. "We thank the community for its continued support, as well as the patients and families who dedicated their time to the clinical development of cerliponase alfa.”
In addition to the BLA review, the FDA has given cerliponase alfa Priority Review status as well as Orphan Drug Designation and Breakthrough Therapy Designation. A Marketing Authorization Application (MAA) has also been submitted to the European Medicines Agency (EMA).
The symptoms of CLN2 disease appear between ages 2 and 4. Symptoms progress from language delays or seizures to devastating motor and cognitive deterioration and blindness. Most children succumb to the disease between ages 8 and 12.
"This is an historic milestone for the Batten disease community," Batten Disease Support and Research Association Executive Director Margie Frazier said. "For the first time since it was originally described more than a century ago, there is a potential treatment for our children with CLN2 disease. We appreciate BioMarin’s continued commitment to pursue a therapy for this devastating disease and to advancing it quickly through the development process."
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