Clinigen Group plc announced it has signed an exclusive agreement with Nippon Shinyaku Co., Ltd. ('Nippon Shinyaku') to implement a Managed Access Program for VILTEPSO® (viltolarsen) for patients with Duchenne muscular dystrophy (DMD) who are amenable to exon 53 skipping therapy. DMD is a progressive muscle disease that primarily occurs in boys due to a genetic mutation in the dystrophin gene. This prevents the production of normal dystrophin resulting in reduced muscle strength/function that progressively continues to decline.

There are many types of genetic mutations that can cause DMD. Viltolarsen skips exon 53; and therefore has the potential to rescue the dystrophin protein for 8-10% of DMD patients. Following finalization, the Managed Access Program will enable early access to VILTEPSO® for eligible patients in countries where it is not commercially available.

VILTEPSO® is currently only approved in the US and Japan. Under the terms of the agreement, Clinigen will manage key elements of the program including regulatory oversight, logistics and access management.