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BioMarin Pharmaceuticals Inc.

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BioMarin Announces Withdrawal of Market Authorization Application for Kyndrisa™ (drisapersen) in Europe

SAN RAFAEL, Calif., May 31, 2016 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today that it has withdrawn its Kyndrisa™ (drisapersen) Marketing Authorization Application (MAA) from the European Medicines Agency (EMA) following discussions at the May 2016 Committee for Medicinal Products for Human Use (CHMP) meeting. Those discussions clearly indicated that the CHMP intended to issue a negative opinion. Kyndrisa is an experimental drug for the treatment of Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping.

Based on discussions at the CHMP meeting and the Food and Drug Administration Complete Response Letter in January, BioMarin intends to discontinue clinical and regulatory development of Kyndrisa as well as the three other first-generation follow-on products, BMN 044, BMN 045 and BMN 053, currently in Phase 2 studies for distinct forms of Duchenne muscular dystrophy. Notwithstanding this outcome for Kyndrisa in Europe, the Company continues to expect to achieve non-GAAP break-even or better in 2017.

BioMarin plans to work with physicians, patient groups, and regulatory authorities to develop a transition plan for those patients currently being treated with Kyndrisa, BMN 044, BMN 045 and BMN 053. The Company will continue to explore the development of next generation oligonucleotides for the treatment of Duchenne muscular dystrophy.

"The withdrawal of the MAA and discontinuation of our current experimental drugs for Duchenne is a difficult but necessary decision at this time," said Jean-Jacques Bienaimé, BioMarin chairman and chief executive officer. Mr. Bienaimé added, "We want to extend our sincere gratitude to all of the families and caregivers who supported our efforts over the last year to bring Kyndrisa to patients with Duchenne. Our plan now is to invest in research of next generation oligonucleotides with the goal of making a safe and effective treatment available for boys with this devastating disorder."
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Source: press release, 5/31/16. http://investors.bmrn.com/releasedetail.cfm?ReleaseID=973536

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Jean-Jacques Bienaime, Chief Executive Officer, gave guidance for the CHMP opinion for Kyndrisa. He stated, "But we remain hopeful that there is a near-term fast-forward for approval in EU and beyond where more than 80% of global patients reside. We look forward to staying on track with the European Regulatory submission of the Marketing Authorization Application for Kyndrisa and to the current timeline, we anticipate a CHMP opinion in late Q2, which could lead to a potential approval in Q3."
Source: Q4 2015 earnings conference call, 2/25/16.

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Kyndrisa (drisapersen) for Duchenne muscular dystrophy: The Committee for Medicinal Products (CHMP), the arm of the European Medicines Agency that is currently reviewing the Marketing Authorization Application for Kyndrisa, is expected to provide an opinion on the application in the second quarter of 2016. If the CHMP provides a positive opinion, Kyndrisa could potentially be approved in the E.U. in the second half of 2016.
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Source: press release, 2/25/16. http://investors.bmrn.com/releasedetail.cfm?ReleaseID=957260

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Jean-Jacques Bienaime, Chief Executive Officer, commented on the timing of the CHMP opinion for Kyndrisa. He stated, "So anyway over the next six months, we have numerous development and regulatory opportunities to look forward to, including our advisory committee meeting on November 24 for Kyndrisa, and our PDUFA date of December 27, followed by an anticipated CHMP opinion in the first half of 2016 in Europe, on our marketing authorization application with the EMA, and the potential approval decision in the second half of next year."
Source: Q3 2015 earnings conference call, 10/29/15.

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BioMarin Announces EMA Validates MAA for Drisapersen for Treatment of Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

SAN RAFAEL, Calif., June 25, 2015 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced the European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for drisapersen for the treatment of Duchenne Muscular Dystrophy amenable to exon 51 skipping. Validation of the MAA confirms that the submission is complete and starts the EMA's standard review process. Day 120 questions will be received on 22 October 2015, leading to a potential CHMP opinion in the first half of 2016 and a European Commission Decision by the third quarter of 2016.

Drisapersen is an investigational antisense oligonucleotide drug candidate for the treatment of the largest subset of Duchenne muscular dystrophy (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.

"Reaching this important regulatory milestone demonstrates BioMarin's unwavering commitment to provide a first-in-class therapy for patients who have a specific type of Duchenne muscular dystrophy and have few, if any, treatment options," said Camilla V. Simpson, Global Head of Regulatory Affairs, Pharmacovigilance. "We are making significant strides on behalf of patients with Duchenne muscular dystrophy. Our hope is to offer a meaningful treatment option around the world."

DMD is the most common fatal genetic disorder diagnosed in childhood, affecting approximately 1 in every 3,500 live male births. In Europe, it is estimated there are 23,000 boys and young men with Duchenne Muscular Dystrophy, and approximately 3,000 of those would be candidates for drisapersen. In BioMarin's commercial territories, approximately 85 percent of Duchenne patients are located outside of the United States, including Western Europe, Middle East, Eastern Europe, Latin America and Japan. Western Europe has the largest patient population among those areas, exceeding the United States by around 30 percent. It is estimated that DMD affects approximately 90,000 patients in BioMarin's commercial territories.

"We are thrilled that BioMarin has reached this point in the EMA review process because it represents a potentially important medical development for boys with Duchenne muscular dystrophy," said Elizabeth Vroom, Chair of United Parent Projects Muscular Dystrophy (UPPMD). "We are looking forward to the day when boys with Duchenne muscular dystrophy have a number of treatment options that will change the course of this devastating disease."
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Source: press release, 6/25/15. http://investors.bmrn.com/releasedetail.cfm?ReleaseID=919556

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BioMarin Submits Drisapersen MAA to EMA for the Treatment of Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

SAN RAFAEL, Calif., June 8, 2015 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today the submission of 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. 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. In Europe, it is estimated there are 23,000 boys with Duchenne Muscular Dystrophy, and approximately 3,000 of those would be candidates for drisapersen. In BioMarin's commercial territories, approximately 85 percent of Duchenne patients are located outside of the United States, including Western Europe, Middle East, Eastern Europe, Latin America and Japan. Western Europe has the largest patient population among those areas, exceeding the United States by around 30 percent.

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 recently submitted a New Drug Application (NDA) to the United States Food and Drug Administration (FDA) for drisapersen in April 2015.

"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."

"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."
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Source: press release, 6/08/15. http://investors.bmrn.com/releasedetail.cfm?ReleaseID=916857

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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: Filed

Event Type: Regulatory EMA: CHMP Opinion (Estimate)

Dates: 2016-06-24

Results: Discontinued