FDA Posts Briefing Documents for Advisory Committee Meeting to Review Kyndrisa™ (drisapersen) for the Potential Treatment of Duchenne Muscular Dystrophy Amendable to Exon 51 Skipping

BioMarin (BMRN) PT Trimmed at Wedbush Following Briefing Docs; Sees Positive Read-Thru to SRPT, None to PTCT

Wedbush analyst Heather Behanna lowered her price target on BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) to $125.00 (from $137.00) following today's FDA briefing documents. The firm maintained a Neutral rating.

Behanna said the briefing docs for drisapersen efficacy were in line with their expectation. Meanwhile, the FDA has unleashed its view point on BioMarin's drisapersen for DMD including the focus on potential unblinding of studies due to injection site reactions. "Things that were new to us include analysis that boys in the 6 mg/kg continuous arm of the one successful 24-week Phase II study may have... More

Despite Harsh BioMarin (BMRN) Briefing Docs, the Jury is Still Out - Leerink Partners

Leerink Partners analyst Joseph Schwartz weighed in on BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) following today's FDA briefing docs, noting they were even more harsh then expected. That said, the analyst said the jury is still out.

Schwartz commented, "FDA briefing documents for... More

BioMarin Pharma (BMRN) Briefing Docs 'Ugly' - Baird (SRPT)

Baird analyst Brian Skorney weighed in on BioMarin Pharma (NASDAQ: BMRN) after today's Drisapersen briefing docs, calling them "ugly"

Skorney commented, "The FDA isn't even offering voting questions to the panelist and the review on risk benefit states: The benefit of drisapersen in exon... More

BioMarin Pharma (BMRN) Announces Consistent Drisapersen Efficacy Evidence Demonstrated Across Three Studies

BioMarin Pharma (NASDAQ: BMRN) announced data analysis for Kyndrisa demonstrating consistent evidence of efficacy in comparable patients across three randomized, placebo-controlled studies that were conducted contemporaneously. Kyndrisa is currently under review with U.S. and European health authorities for the treatment of patients with Duchenne muscular dystrophy who are amenable to exon 51 skipping. This analysis is included in materials prepared by BioMarin for the November 24, 2015 Peripheral and Central Nervous System Drugs Advisory Committee (PCNS) meeting and can be found on the FDA website at:

BioMarin Pharma (BMRN) Affirms Posting of Drisapersen NDA Briefing Docs

Following earlier reports, BioMarin Pharma (NASDAQ: BMRN) announced that the U.S. Food and Drug Administration (FDA) posted briefing materials... More