BioMarin withdraws Kyndrisa's marketing authorization for Europe
Kyndrisa, an experimental drug, is designed to treat Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping.
The MAA was submitted to the European Medicines Agency (EMA) to receive approval for marketing the product in Europe. At the meeting, the Committee for Medicinal Products for Human Use (CHMP) made it clear that it planned to release a negative opinion of the drug.
In light of the discussions with the CHMP, as well as other meetings with the U.S. Food and Drug Administration (FDA), BioMarin wants to end regulatory and clinical development of the drug. The agencies also want to discontinue three additional first-generation follow-on products: BMN 045, BMN 044 and BMN 053.
"The withdrawal of the MAA and discontinuation of our current experimental drugs for Duchenne is a difficult but necessary decision at this time," Jean-Jacques Bienaimé, BioMarin chairman and CEO, said. "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."
Organizations in this story
BioMarin Pharmaceutical Inc 770 Lindaro Street San Rafael, CA - 94901