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BioMarin Announces FDA Accepts Drisapersen NDA for Treatment of Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

FDA Grants Priority Review Status

FDA PDUFA Date is December 27, 2015

SAN RAFAEL, Calif., June 29, 2015 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced the U.S. Food and Drug Administration (FDA) has accepted for review the submission of a New Drug Application (NDA) for drisapersen for the treatment of Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping, and the Prescription Drug User Fee Act (PDUFA) goal date for a decision is December 27, 2015. The FDA has granted drisapersen Priority Review status, which is designated to drugs that offer major advances in treatment, or provide a treatment where no adequate therapy exists.

In the FDA's filing communication, the Agency informed the company that it is currently planning to hold an advisory committee meeting to discuss the application. No date has been set for this meeting. Drisapersen previously has been granted Orphan and Fast Track status, as well as Breakthrough Therapy designations by the FDA. The U.S. filing is based on three randomized placebo-controlled trials and two long-term open-label studies of more than 300 patients in which some boys have been treated for more than three years.

"We are dedicated to bringing a meaningful therapy specifically for patients with a particular form of Duchenne to patients all over the world. We are thrilled that BioMarin has reached this important step in the United States, which comes on the heels of the recent validation of our European filing for drisapersen," said Camilla V. Simpson, Global Head of Regulatory Affairs, Pharmacovigilance at BioMarin. "Obtaining Priority Review status is validation of BioMarin's commitment to urgently move treatment beyond supportive care and to address the underlying cause of the disease. We are thankful to the boys and their families who participated in our clinical trials, which have allowed us to achieve this important milestone for Duchenne patients."

Drisapersen is an investigational antisense oligonucleotide drug candidate for the treatment of the largest subset of DMD amenable to single exon skipping. Drisapersen induces the skipping of dystrophin exon 51, potentially providing a therapeutic benefit to DMD patients for whom skipping of exon 51 restores the proper dystrophin reading frame, corresponding to approximately 13% of DMD patients. In the U.S., it is estimated there are approximately 2,000 patients who would be candidates for drisapersen.

"Since PPMD's founding more than two decades ago, we have been focused on improving the treatment, quality of life and long-term outlook for boys with Duchenne muscular dystrophy," said Pat Furlong, President and Founder of Parent Project Muscular Dystrophy. "The completion of this regulatory milestone brings the community one step closer to what could be the first specific drug therapy to treat Duchenne in the United States. We are hopeful that this is the beginning of a new era of many medical advancements that will change the course of this devastating disease."

About Duchenne Muscular Dystrophy

DMD is the most common fatal genetic disorder diagnosed in childhood, affecting approximately 1 in every 3,500 live male births. Duchenne muscular dystrophy is a severely debilitating childhood neuromuscular disease caused by mutations in the dystrophin gene. This results 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, patients suffer from progressive loss of muscle function, often rendering them wheelchair-bound before the age of 12 years. Respiratory and cardiac muscle can also be affected by the disease and most patients die in early adulthood due to respiratory and cardiac failure. Because the dystrophin gene is located on the X-chromosome, DMD primarily affects boys.

About Drisapersen and Exon Skipping

In Duchenne muscular dystrophy, mutations in the dystrophin gene lead to the absence of dystrophin protein, resulting in the most severe form of dystrophin deficient muscular dystrophy. Drisapersen is an antisense oligonucleotide that induces exon skipping to provide a molecular patch for dystrophin transcripts produced by certain mutated dystrophin genes. Exons are the parts of a gene that contain the instructions for generating a protein. In applicable cases, skipping an exon near the mutation allows for the production of a truncated but functional dystrophin protein.

For more details, please visit the link http://www.biomarin.com/products/pipeline/drisapersen-for-duchenne-muscular-dystrophy/