BioMarin's DMD Drug Kyndrisa Receives CRL from the FDA
BioMarin Pharmaceutical Inc. BMRN announced that it has received a Complete Response Letter (CRL) from the FDA for its New Drug Application (NDA) for Kyndrisa for the treatment of patients with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping.
The FDA stated that the standard of substantial evidence of effectiveness of Kyndrisa was not met. The company reported that it will work with the agency to determine the appropriate steps to address the application.
Meanwhile, the company will continue evaluating Kyndrisa in the ongoing extension studies for other exon-skipping oligonucleotides – BMN 044, BMN 045 and BMN 053.
We remind investors that a marketing application for Kyndrisa is currently under review in the EU for the same indication. An opinion from the Committee for Medicinal Products for Human Use (CHMP) is expected in the first half of 2016. BioMarin expects a final decision on the marketing authorization of the candidate in the second half of 2016.
As per information provided by the company, 1 in every 3,500–5,000 male children is estimated to be affected by DMD.
Our Take
A negative response from the FDA does not come as a surprise considering the unfavorable feedback from the FDA’s advisory panel regarding Kyndrisa in Nov 2015.
We remind investors that next week (Jan 22), Sarepta Therapeutics, Inc.’s SRPT eteplirsen will be reviewed by the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee for the same indication. A final response from the FDA is expected by Feb 26.
We expect investor focus to remain on the outcome of the upcoming advisory panel meeting of eteplirsen as well as further details on Kyndrisa.
BioMarin currently carries a Zacks Rank #4 (Sell). A couple of better-ranked stocks in the health care sector are Horizon Pharma plc HZNP and Ligand Pharmaceuticals Incorporated LGND, both sporting a Zacks Rank #1 (Strong Buy).
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