Drisapersen (also known as Kyndrisa, PRO051 and GSK2402968[1]) is an experimental drug that was under development by BioMarin, after acquisition of Prosensa,[2] for the treatment of Duchenne muscular dystrophy.
As there are 79 exons in the longest splice form of the dystrophin transcript, many different oligonucleotides are needed to address the range of mutations present in the population of people with DMD.
A long-term open-label extension study (DEMAND IV) suggests that giving the drug at an earlier age and treating the boys for longer may delay progression of the disease.
[5] The Prescription Drug User Fee Act (PDUFA) goal dates for these are December 27, 2015 for drisapersen and February 26, 2016 for eteplirsen.
In May 2016, BioMarin announced they intended to discontinue clinical and regulatory development of drisapersen as well as related first generation compounds currently in Phase 2 studies for distinct forms of Duchenne muscular dystrophy.