BioMarin Announces Withdrawal of Market Authorization

BioMarin Announces Withdrawal of Market Authorization Application for Kyndrisa™ (drisapersen) in Europe

SAN RAFAEL, Calif., May 31, 2016 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today that it has withdrawn its Kyndrisa™ (drisapersen) Marketing Authorization Application (MAA) from the European Medicines Agency (EMA) following discussions at the May 2016 Committee for Medicinal Products for Human Use (CHMP) meeting.  Those discussions clearly indicated that the CHMP intended to issue a negative opinion.  Kyndrisa is an experimental drug for the treatment of Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. 

Based on discussions at the CHMP meeting and the Food and Drug Administration Complete Response Letter in January, BioMarin intends to discontinue clinical and regulatory development of Kyndrisa as well as the three other first-generation follow-on products, BMN 044, BMN 045 and BMN 053, currently in Phase 2 studies for distinct forms of Duchenne muscular dystrophy.  Notwithstanding this outcome for Kyndrisa in Europe, the Company continues to expect to achieve non-GAAP break-even or better in 2017.  

BioMarin plans to work with physicians, patient groups, and regulatory authorities to develop a transition plan for those patients currently being treated with Kyndrisa, BMN 044, BMN 045 and BMN 053.  The Company will continue to explore the development of next generation oligonucleotides for the treatment of Duchenne muscular dystrophy. 

"The withdrawal of the MAA and discontinuation of our current experimental drugs for Duchenne is a difficult but necessary decision at this time," said Jean-Jacques Bienaimé, BioMarin chairman and chief executive officer.  Mr. Bienaimé added, "We want to extend our sincere gratitude to all of the families and caregivers who supported our efforts over the last year to bring Kyndrisa to patients with Duchenne.  Our plan now is to invest in research of next generation oligonucleotides with the goal of making a safe and effective treatment available for boys with this devastating disorder."

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It's really sad to know that the hard work of boys and families went in vain. I don't know when will our boys get a treatment option. Years and years passing by, billions and billions are spent but no treatment so far!

This is a sad moment for us all parents. The disease itself is challenging and the drug approving authorities dont make it any easier. Hoping that the other companies continue to put in efforts and help us cure duchenne

I don't believe it's in vain. The DMD community learned alot about the trial process, what the FDA expects (reasonable or not) and that researchers need to fill in some  gaps in the natural history data. But, it is very disappointing. The hope for exon skipping is not dead.  We still have Sarepta and Wave Life Sciences (with a next generation exon skipping drug)

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