Wave Life Sciences has announced that the Drug Regulator in the USA – the FDA - has granted both orphan drug designation and rare paediatric disease designation for WVE-210201 – a potential exon skipping drug. Orphan drug status has already been granted in Europe.

The US Orphan Drug Act was designed to encourage companies to develop new drugs to treat, diagnose or prevent rare conditions. Being granted Orphan Drug status means that a potential therapy shows promise in the rare disease field and gives the company increased access to regulatory assistance and provides economic benefits should the potential therapy gain marketing approval.

WVE-210201 is a potential exon skipping drug that is designed to skip exon 51 of the dystrophin gene (which could treat 13% of people living with Duchenne). It is currently being tested in a global placebo-controlled, phase 1 clinical trial. The trial, which will test the safety and effectiveness, will include approximately 40 people living with Duchenne who are amenable to exon 51 skipping and between 5 and 18 years of age. Participants completing the trial will have the option to enrol in an extension study in which everybody will receive WVE-210201.

Our team is motivated by a sense of urgency and compassion for the patients, families and caregivers affected by Duchenne muscular dystrophy and other serious, life-threatening conditions with high areas of unmet need. We are very pleased to receive these two important designations from the FDA and believe they further reinforce the potential of WVE-210201 to help boys suffering from DMD. -Michael Panzara, MD, MPH, Wave Life Sciences.

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