BioMarin Pharmaceutical Inc. will halt development of Duchenne muscular dystrophy (DMD) drug, a press release read.
The drug in question is Kyndrisa, the company's leading proposed therapy for the debilitating and fatal disease DMD. Reports of negative reviews from the European Medicines Agency (EMA) surfaced after the drug was rejected by U.S. regulators early this year. Specifically, the drug was rejected by the U.S. Food and Drug Administration after it failed to meet efficacy requirements.
Before facing possible rejection from the European agency, BioMarin decided that it was necessary to discontinue developing Kyndrisa and three other related drugs - BMN 044, BMN 045 and BMN 053, which are all in Phase II studies. Kyndrisa (drisapersen) is an experimental drug proposed for the treatment of DMD and which is responsive to exon 51 skipping.
Exon skipping is the inability of exons or gene sections to join together and prohibit the protein assembly needed for dystrophin protein to work. The process of exon skipping encourages the cellular machinery via antisense nucleotides to mask and skip the exons so they do not affect protein production.
In 2014, BioMarin invested $680 million in cash by buying up Prosensa and its drisapersen in hopes of developing therapeutic drugs for DMD and have them go through regulatory approval. However, as EMA expressed during the 2016 Committee for Medicinal Products for Human Use (CHMP) meeting that it would likely reject the drug, BioMarin is now withdrawing its Market Authorization Application (MAA). But the company said it is not giving up the fight against Duchenne, as it is going to focus on developing next generation oligonucleotides.
"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," said Jean-Jacques Bienaimé, BioMarin's Chairman and CEO.
Meanwhile, Sarepta's eteplirsen NDA, also a DMD treatment, was recently rejected by the FDA. BioMarin is currently in patent wars with Sarepta for the use of antisense oligonucleotide.
