Biotechnology Events

Home

BioMarin Pharmaceuticals Inc.

.

PLEASE SCROLL DOWN TO THE RESULTS SECTION (below) TO SEE THE OUTCOME OF THIS EVENT.

.

BioMarin Posts Presentation From FDA Advisory Committee Meeting for Kyndrisa(TM) (drisapersen) for the Treatment of Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
SAN RAFAEL, Calif., Nov. 24, 2015 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today that the Company's presentation from the Peripheral and Central Nervous System Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA)'s meeting for KyndrisaTM (drisapersen) is now available. The Advisory Committee is meeting today to discuss the data submitted to support the New Drug Application (NDA) for Kyndrisa for the treatment of Duchenne muscular dystrophy amenable to exon 51 skipping.

Interested parties may access BioMarin's presentation from today's Advisory Committee meeting via the link below to the investor section of the BioMarin website.

http://investors.bmrn.com/common/download/download.cfm?companyid=ABEA-3W...
More
Source: press release, 11/24/15. http://investors.bmrn.com/releasedetail.cfm?ReleaseID=944310

.

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) Announced Today the Stock is Halted
SAN RAFAEL, Calif., Nov. 24, 2015 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced today that NASDAQ has halted trading of the company's stock. The Peripheral and Central Nervous System Drugs Advisory Committee (PCNS) of the U.S. Food and Drug Administration (FDA) is meeting today to review BioMarin's New Drug Application (NDA) for Kyndrisa, an investigational antisense oligonucleotide drug candidate for the treatment of patients with Duchenne muscular dystrophy amenable to exon 51 skipping.

The PCNS advisory meeting is scheduled for November 24 at 8:00 a.m. to 5:30 p.m. EST. The briefing materials and webcast information can be found on the FDA website at: http://www.fda.gov/AdvisoryCommittees/Calendar/ucm467180.htm

The Prescription Drug User Fee Act (PDUFA) action date for completion of FDA review of the Kyndrisa NDA is December 27, 2015.
MMore
Source: press release, 11/24/15. http://investors.bmrn.com/releasedetail.cfm?ReleaseID=944221

.

Briefing Information for the November 24, 2015 Meeting of the Peripheral and Central Nervous System (PCNS) Drugs Advisory Committee
More
Source: FDA.gov. http://www.fda.gov/AdvisoryCommittees/CommitteesMeetingMaterials/Drugs/P...

.

BioMarin Announces FDA Advisory Committee to Review Drisapersen for Treatment of Patients With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

Advisory Committee Meeting Scheduled for November 24, 2015

SAN RAFAEL, Calif., Oct. 15, 2015 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced that 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 drisapersen. The FDA is currently reviewing drisapersen for the treatment of patients with Duchenne muscular dystrophy amenable to exon 51 skipping. The advisory committee will review drisapersen data included in a new drug application (NDA) during a meeting on November 24, 2015.

"Duchenne muscular dystrophy is a deadly, progressive muscle disorder with limited treatment options that affects mostly boys and young men. We welcome the necessary regulatory review processes to move treatment for this community beyond supportive care to a therapy that addresses the underlying cause of the disease," said Camilla V. Simpson, Global Head of Regulatory Affairs, Pharmacovigilance. "We look forward to discussing the New Drug Application for drisapersen with the advisory committee with the goal of bringing this much-needed treatment one step closer to patients who have a specific type of Duchenne muscular dystrophy."

Drisapersen is an investigational antisense oligonucleotide drug candidate for the treatment of the largest subset of Duchenne muscular dystrophy patients amenable to single exon skipping. In the U.S., it is estimated there are approximately 2,000 patients who might benefit from treatment with drisapersen.

The Prescription Drug User Fee Act (PDUFA) action date for completion of FDA review of the drisapersen NDA 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. The FDA has also granted drisapersen Orphan and Fast Track status, as well as Breakthrough Therapy designation.
More
Source: press release, 10/15/15. http://investors.bmrn.com/releasedetail.cfm?ReleaseID=936743

.

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 FDA: Advisory Committee Meeting

Dates: 2015-11-24

Results:

.

FDA Advisory Committee Discusses Clinical Data Package for BioMarin's Kyndrisa(TM) (drisapersen) for the Treatment of Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
-- If Approved, Kyndrisa Would Become the First Disease-Modifying Therapy for Patients With Duchenne Muscular Dystrophy in the United States Amenable to Exon 51 Skipping --

SAN RAFAEL, Calif., Nov. 24, 2015 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today that the Peripheral and Central Nervous System Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA) met to discuss the data submitted to support the New Drug Application (NDA) for KyndrisaTM (drisapersen) for the treatment of Duchenne muscular dystrophy (Duchenne) amenable to exon 51 skipping. The committee reviewed the Kyndrisa data package, which includes three randomized, placebo controlled trials with more than 300 patients and more than 500 patient years of exposure. The committee discussed the overall strengths and weaknesses of the application but was not asked to vote on a recommendation for approval of Kyndrisa.

"I'd like to thank the patients, families and physicians who participated in Kyndrisa clinical trials and in today's panel discussion," said Jean-Jacques Bienaimé, chairman and chief executive officer of BioMarin. "We look forward to continuing our efforts to bring Kyndrisa to the Duchenne community. After today's meeting, our next step is to continue working with the FDA as they complete their regulatory review."

"The Committee's discussion today is part of the FDA's evaluation of Kyndrisa and the potential for a treatment option for the Duchenne community in the United States," said Debra Miller, co-founder and CEO of CureDuchenne. "We have long supported the research and development of Kyndrisa and eagerly anticipate the FDA's decision, which could make our hopes for therapy a reality."

In addition to discussing the overall strengths and weaknesses of the data supporting the efficacy of drisapersen and the acceptability of its safety profile, the FDA's advisory committee was asked to discuss the following issues and vote on the associated questions.

Discussion Issues

Voting Questions (Results)
1. Discuss the strength of efficacy evidence provided by Study 1 with particular consideration of the following issues and any other issues that you think may be important:
a. Discrepant results of the two dosing regimens despite similar exposure to drisapersen
b. Lack of statistically significant results on secondary endpoints

What overall impact do the issues discussed in question #1 have on the persuasiveness of Study 1?

a. Strengthen (1)
b. Weaken (9)
c. No effect (7)

2. Discuss the strength of efficacy evidence provided by Study 2 with particular consideration of the following issues and any other issues that you think may be important:
a. Lack of statistical significance of the primary outcome measure (p = 0.07 on ITT analysis, p = 0.23 on per protocol analysis)
b. 3 mg/kg group numerically inferior to placebo
c. 6 mg/kg group numerically inferior to placebo for most secondary endpoints

What overall impact do the issues discussed in question #2 have on the persuasiveness of Study 2?

a. Strengthen (0)
b. Weaken (5)
c. No effect (12)
3. Discuss the evidence provided by Study 3 with particular consideration of the following issues and any other issues that you think may be important:
a. Lack of statistical significance of the primary outcome measure (p = 0.42) in a well-powered Phase 3 study
b. Lack of nominally statistically significant results on all secondary endpoints

What is the impact of Study 3 results on the persuasiveness of findings in Study 1 and Study 2?

a. Strengthen (0)
b. Weaken (15)
c. No effect (2)
4. Drisapersen was designed to increase production of dystrophin. Discuss the evidence presented about dystrophin production, including the following:
a. Similar number of patients with skipped band of mRNA detected by PCR in the placebo group and drisapersen group
b. Similar number of patients with dystrophin increase from baseline in the placebo group and drisapersen group on immunofluorescence testing
c. Lack of notable increase in dystrophin with drisapersen treatment on western blot analysis (pre-treatment levels <1% and post-treatment levels <1%) What is the impact of the dystrophin results on the interpretation of the clinical results?

a. Strengthen (0)
b. Weaken (6)
c. No effect (10)
not voting (1)

The Committee's feedback will be considered by the FDA in its review of the NDA for Kyndrisa. The FDA is not bound by the Committee's guidance, but takes its advice into consideration when reviewing investigational medicines.

The FDA has set a target action date of December 27, 2015, under the Prescription Drug User Fee Act (PDUFA). An application for marketing approval of Kyndrisa is also pending in the European Union.
More
Source: press release, 11/24/15. http://investors.bmrn.com/releasedetail.cfm?ReleaseID=944400

.