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BioMarin Pharmaceuticals Inc.

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BioMarin Announces That FDA Has Advised it Will Not Take Action on the Kyndrisa™ (drisapersen) New Drug Application by the PDUFA Date
SAN RAFAEL, Calif., Dec. 18, 2015 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today that the U.S. Food and Drug Administration (FDA) has notified the Company that they had not yet completed their review process and would be unable to take an action by the Prescription Drug User Fee Act (PDUFA) action date for KyndrisaTM (drisapersen) of December 27, 2015, and anticipate taking action in early January 2016.
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Source: press release, 12/18/15. http://investors.bmrn.com/releasedetail.cfm?ReleaseID=947587

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Kyndrisa (drisapersen) for Duchenne muscular dystrophy: The Peripheral and Central Nervous System Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA) will review the New Drug Application (NDA) for Kyndrisa on November 24, 2015. The PDUFA date for Kyndrisa is December 27, 2015.
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Source: press release, 10/29/15. http://investors.bmrn.com/releasedetail.cfm?ReleaseID=939338

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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."
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Source: press release, 6/29/15. http://investors.bmrn.com/releasedetail.cfm?ReleaseID=919809

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BioMarin Completes Rolling NDA Submission to FDA for Drisapersen for Treatment of Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

SAN RAFAEL, Calif., April 27, 2015 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced completion of the rolling submission of a New Drug Application (NDA) to the United States Food and Drug Administration (FDA) for drisapersen, an investigational exon-skipping drug candidate for the treatment of the largest genetically defined subset of Duchenne muscular dystrophy (DMD). DMD is the most common fatal genetic disorder diagnosed in childhood, affecting approximately 1 in every 3,500 live male births with about 20,000 new cases diagnosed globally each year. 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. The company intends to also submit an application for registration in the European Union in summer 2015.

"We believe drisapersen may offer a meaningful benefit to boys living with DMD whose mutations are amenable to exon 51 skipping. The totality of data on drisapersen contains three randomized, placebo-controlled, efficacy trials and two long term extension studies, which include some boys treated for approximately 3.4 years," said Camilla V. Simpson, Global Head of Regulatory Affairs, Pharmacovigilance. "With this application, BioMarin continues in its long-standing tradition of developing important therapies for those who are most in need. The submission of the NDA represents a significant milestone for BioMarin, and we appreciate the strong, collaborative effort of many hard working employees, investigators, patients and their families. We look forward to working with the U.S. Regulatory Authorities to thoroughly understand the data generated for this heterogenous and critically ill patient population and hopefully to bring this treatment to patients expeditiously."

Drisapersen has been granted Orphan and Fast Track status, as well as Breakthrough Therapy designation by the FDA.

DMD is caused by a mutation in the gene that encodes for dystrophin, a protein that is important in connecting the cytoskeleton of muscle fibers to the extracellular matrix. Its deficiency in DMD leads to progressive muscle weakness, loss of ambulation in early adolescence, and typically death due to pulmonary or cardiac insufficiency in the late twenties. Because the Duchenne gene is found on the X-chromosome, it primarily affects boys; however, it occurs across all races and cultures. There is currently no approved therapy in the United States for DMD.

"This is a first for the Duchenne community, and we are filled with hope that there could be a treatment for Duchenne in the United States," said Debra Miller, co-founder and CEO of CureDuchenne. "CureDuchenne has been supporting the development of drisapersen for more than a decade, and we are delighted that BioMarin has reached this important stage. We salute the researchers who have been working so hard, and we share their determination to find a cure for Duchenne."
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Source: press release, 4/27/15. http://investors.bmrn.com/releasedetail.cfm?ReleaseID=908731

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Drisapersen for the treatment of Duchenne Muscular Dystrophy patients with the exon 51 mutation is currently under rolling review with the FDA. The NDA submission to the FDA is expected to be completed in April 2015 and the Marketing Authorization Application (MAA) with the European Medicines Agency (EMA) is expected to be submitted this summer.
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Source: press release, 2/25/15. http://investors.bmrn.com/releasedetail.cfm?ReleaseID=898344

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Compound/DeviceSpecialtyIndicationCompound ClassTarget
Drisapersen (PRO051)Medical GeneticsDuchenne Muscular DystrophyExon-skipping technologyExon-51

Mechanism of action: Drisapersen (PRO051) is an RNA-based product that induces exon 51 skipping in the dystrophin gene and is intended for approximately 13% of all Duchenne Muscular Dystrophy (DMD) patients, the largest known subpopulation of patients that includes those with deletions of exon 50, exon 52, exons 45-50, exons 48-50, and exons 49-50. It is highly sequence-specific, minimizing the risk for off-target effects.

Phase of Development: III

Event Type: Regulatory FDA: PDUFA DATE

Dates: 2016-01-01 - 2016-01-15

Results:

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FDA Issues Complete Response Letter for KyndrisaTM for Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
Marketing Application in Europe Remains Under Review

SAN RAFAEL, Calif., Jan. 14, 2016 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today that the U.S. Food and Drug Administration (FDA) issued a Complete Response letter to the Company's New Drug Application (NDA) for KyndrisaTM (drisapersen) for the treatment of Duchenne muscular dystrophy (Duchenne) amenable to exon 51 skipping.

The FDA issues Complete Response letters to indicate that the review cycle for an application is complete and that the application is not ready for approval in its present form. FDA has concluded that the standard of substantial evidence of effectiveness has not been met. BioMarin is reviewing the Complete Response Letter and will work with the FDA to determine the appropriate next steps regarding this application.

Duchenne affects approximately 1 in every 3,500-5,000 male children, making it the most common fatal genetic disorder diagnosed in childhood. There is currently no FDA-approved therapy designed specifically to treat Duchenne.

The ongoing Kyndrisa extension studies will continue, as will the ongoing clinical trials for other exon-skipping oligonucleotides, BMN 044, BMN 045 and BMN 053, while BioMarin is exploring next steps for this application. Patients currently receiving Kyndrisa, BMN 044, BMN 045 and BMN 053 will remain on therapy.

Kyndrisa Marketing Authorization Application Remains Under Regulatory Review in Europe

An application for marketing approval of Kyndrisa is also under review in the European Union. BioMarin anticipates that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) will provide an opinion for the company's Marketing Authorization Application (MAA) for Kyndrisa for the treatment of Duchenne muscular dystrophy (Duchenne) amenable to exon 51 skipping in the first half of 2016. If the CHMP opinion is positive, the MAA will be referred to the European Commission (EC). If the MAA is approved by the EC, BioMarin would receive marketing authorization for Kyndrisa in all EU Member States. The EC is expected to render a final decision for Kyndrisa in the second half of 2016.
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Source: press release, 1/14/16. http://investors.bmrn.com/releasedetail.cfm?ReleaseID=950309

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