BioMarin Pharmaceutical Inc. has announced the withdrawal of its Marketing Authorization Application (MAA) from the European Medicines Agency (EMA) for Kyndrisa (drisapersen), a drug developed for Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping.
The decision follows deliberations at the May 2016 Committee for Medicinal Products for Human Use (CHMP) meeting, which resulted in an indication that the CHMP would issue a negative opinion on the application.
Kyndrisa is an investigational antisense oligonucleotide developed for DMD, an x-linked genetic disorder affecting mostly young boys, who can start to show signs of muscle weakness as early as 2 to 5 years old. From then, DMD progressively weakens the skeletal or voluntary muscles in the arms, legs, and trunk, and can result in patients being wheelchair-bound between the ages of 7 and 13.
The drug induces exon skipping to provide a molecular patch for dystrophin transcripts produced by mutated dystrophin genes. Exons are parts of a gene that hold the instructions for producing proteins. In some cases, skipping an exon enables the reset of functional dystrophin protein.
“The withdrawal of the MAA and discontinuation of our current experimental drugs for Duchenne is a difficult but necessary decision at this time,” said BioMarin Chairman and CEO Jean-Jaques Bienaimé in a press release. “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.”
After what was discussed at the decisive CHMP meeting, in addition to the U.S. Food and Drug Administration (FDA) Complete Response Letter in January, BioMarin will discontinue clinical and regulatory development of Kyndrisa, as well as three other follow-on products (BMN 044, BMN 045, and BMN 053) currently in Phase 2 clinical studies for different forms of DMD.
Despite this outcome for Kyndrisa in the European Union, BioMarin continues to expect non-GAAP to break even or better by 2017.
BioMarin will continue to explore the development of next-generation oligonucleotides for the treatment of DMD. Now, the company will collaborate with medical physicians, patient groups, and regulatory authorities to develop a transition plan for those who are already receiving treatment with Kyndrisa or any of the three first-generation follow-on products.