BioMarin Files for Approval of its Duchenne Muscular Dystrophy Drug in Europe

BioMarin Pharmaceutical Inc. has filed for European approval of its investigational drug for a rare, genetic disorder that causes progressive muscle wasting.

The company said that it has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for drisapersen, an investigational antisense oligonucleotide drug candidate for the treatment of the largest subset of Duchenne muscular dystrophy (DMD) amenable to single exon skipping. If approved by the EMA, drisapersen will be the first exon-skipping therapy for patients with DMD.

BioMarin acquired drisapersen through its $840 million purchase of Prosensa last November. The drug induces the skipping of dystrophin exon 51, potentially providing therapeutic benefit to DMD patients for whom skipping of exon 51 restores the proper dystrophin reading frame.  This represents approximately 13 percent of DMD patients.

BioMarin submitted a rolling application to the US Food and Drug Administration (FDA) in April.

“The submission of this application to the EMA represents an important achievement in BioMarin’s efforts to bring a meaningful therapeutic option to patients and families around the world with Duchenne muscular dystrophy. We were able to reach this point because of the extraordinary effort of the employees at BioMarin, the investigators for the clinical trials and most important, the boys and their families who participated in the clinical trials,” said Camilla V. Simpson, Global Head of Regulatory Affairs, Pharmacovigilance. “BioMarin has a track record of efficiently developing therapies in rare genetic diseases, and we are pleased that we have submitted this MAA ahead of the expected timeline. We look forward to working with the EMA in the coming months with the goal of bringing this therapy to patients in need.”

DMD is a rare, debilitating childhood neuromuscular disease, caused by mutations in the dystrophin gene. These mutations result in the absence or defect of the dystrophin protein, which is important in connecting the cytoskeleton of muscle fibers to the extracellular matrix. As a result, DMD patients suffer from progressive loss of muscle strength, often causing patients to be wheelchair-bound before the age of 12 years. Additionally, respiratory and cardiac muscle can be affected by the disease and most patients die in early childhood due to respiratory and cardiac failure. DMD is the most common fatal genetic disorder diagnosed in childhood, affecting approximately 1 in every 3,500 live male births with roughly 20,000 new cases diagnosed globally each year. In Europe, there are an estimated 23,000 boys with DMD, of which approximately 3,000 would be candidates for drisapersen.

“We applaud BioMarin’s commitment to develop a therapy for Duchenne muscular dystrophy,” said Elizabeth Vroom, Chair of United Parent Projects Muscular Dystrophy (UPPMD). “The community has been supporting research and development of treatments, and we are pleased that drisapersen has been submitted for EMA review. We are hopeful that this therapy will lead not only to an approved therapy, but will further scientific advances and the development of other treatments for boys with Duchenne.”

Source: BioMarin Pharmaceutical Inc.

Last updated: 6/10/15; 10:50am EST

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