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BioMarin's (BMRN) Kyndrisa Review Period Extended by FDA

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BioMarin Pharmaceutical Inc.BMRN announced that the FDA has not yet completed its review process for the marketing application for Kyndrisa. Subsequently, the regulatory agency expects to render a final decision in early Jan 2016 instead of the previous date of Dec 27, 2015. The company is seeking approval for Kyndrisa for the treatment of patients with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping.

We remind investors that last month, BioMarin announced that the FDA's Peripheral and Central Nervous System Drugs Advisory Committee's vote on Kyndrisa was not favorable.

The committee reviewed the Kyndrisa data package, which includes three randomized studies, and discussed the overall strengths and weaknesses of the regulatory application. However, the panel was not asked to vote on a recommendation for the approval of Kyndrisa. Out of the 17 panel members, 15 were of the opinion that the lack of statistical significance in the primary outcome measure and nominally statistically significant results on all secondary endpoints in the phase III study weakened the persuasiveness of findings from Study 1 and Study 2. The remaining two voted that it had no effect.

Only one panelist voted that the discussions on Study 1 and Study 2 strengthened Biomarin's case. Regarding the impact of dystrophin results on the interpretation of trial data, all panel members were of the opinion that it weakened or had no impact, except one who did not vote.

Kyndrisa has Orphan drug and Fast Track status, Breakthrough Therapy and Rare Pediatric Disease designation in the U.S. The company is also submitting a regulatory application for Kyndrisa in the EU.

We note that Sarepta Therapeutics, Inc. SRPT is also looking to bring its DMD candidate, eteplirsen, to market. The candidate will be reviewed by the FDA's advisory panel on Jan 22, 2016. A response from the FDA is expected by Feb 26, 2016.

BioMarin carries a Zacks Rank #3 (Hold). A couple of better-ranked stocks in the health care sector are Achillion Pharmaceuticals, Inc. ACHN and Corcept Therapeutics Incorporated CORT , both sporting a Zacks Rank #1 (Strong Buy).

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BIOMARIN PHARMA (BMRN): Free Stock Analysis Report

CORCEPT THERAPT (CORT): Free Stock Analysis Report

ACHILLION PHARM (ACHN): Free Stock Analysis Report

SAREPTA THERAP (SRPT): Free Stock Analysis Report

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The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.


The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.

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