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Kyndrisa Approval Status

  • FDA approved: No
  • Brand name: Kyndrisa
  • Generic name: drisapersen
  • Company: BioMarin Pharmaceutical Inc.
  • Treatment for: Duchenne Muscular Dystrophy

Kyndrisa (drisapersen) is an investigational antisense oligonucleotide in development for the treatment of Duchenne muscular dystrophy amenable to exon 51 skipping.

In January 2016, BioMarin Pharmaceutical Inc. announced the receipt of a Complete Response Letter from the U.S. Food and Drug Administration (FDA) advising the company that the New Drug Application for Kyndrisa was not ready for approval in its present form. The FDA concluded that the standard of substantial evidence of effectiveness had not been met. BioMarin will review the Complete Response Letter and work with the FDA to determine the appropriate steps regarding future of the application.

Development Status and FDA Approval Process for Kyndrisa

DateArticle
Jan 14, 2016FDA Issues Complete Response Letter for Kyndrisa for Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
Dec 18, 2015BioMarin Announces That FDA Has Advised it Will Not Take Action on the Kyndrisa (drisapersen) NDA by the PDUFA Date
Oct 15, 2015BioMarin Announces FDA Advisory Committee to Review Drisapersen for Treatment of Patients With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
Aug 19, 2015BioMarin Receives Rare Pediatric Disease Designation From FDA for Drisapersen for the Potential Treatment of Duchenne Muscular Dystrophy
Jun 29, 2015BioMarin Announces FDA Accepts Drisapersen NDA for Treatment of Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
Apr 27, 2015BioMarin Completes Rolling NDA Submission to FDA for Drisapersen for Duchenne Muscular Dystrophy
Oct 10, 2014Prosensa Begins NDA Submission for Drisapersen to treat Duchenne Muscular Dystrophy
Jun  3, 2014Prosensa Announces Regulatory Path Forward for Drisapersen as a Potential Treatment for DMD

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