According to a story from Globe Newswire, the biotechnology company Wave Life Sciences recently announced that its investigational product WVE-210201 has received both Rare Pediatric disease designation and Orphan Drug designation in order to treat Duchenne muscular dystrophy. Wave Life Sciences prioritizes the development of treatments for severe, genetically linked diseases.
About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy is a severe form of muscular dystrophy and it is also the most common type. Its effects begin around age four, and it is characterized by progressive muscle atrophy that typically occurs in the pelvic region and upper legs. It mostly affects boys. The disease is caused by a mutation that causes a lack of dystrophin, a muscle protein that is essential for maintaining muscle mass and composition. The symptoms of Duchenne muscular dystrophy include abnormal walking gait, poor coordination, muscle wasting, loss of walking ability, neurobehavioral disorders, and scoliosis. Treatment for the disorder includes some medications, which can help delay disease progression, and physical therapy. Many patients will eventually require breathing assistance. Average lifespan for patients is around 25 years, but with top of the line care, many patients are living into their 30s. To learn more about Duchenne muscular dystrophy, click here.
About Orphan Drug Designation
WVE-210201 originally received Orphan Drug designation in July. This designation is reserved for products that are intended to treat rare diseases, which is defined as any disease that affects less than 200,000 people in the US. The drug must also display advantages in effectiveness or safety compared to current treatments or must have the potential to fulfill a currently unmet medical need. The designation provides some benefits to the recipient company, such as the waiving of certain fees, tax breaks, and a seven year period of market exclusivity if the drug gets approved.
About Rare Pediatric Disease Designation
Rare Pediatric Disease designation can be given to products that intent to treat severe disease that begin to cause symptoms in people under the age of 18. A recipient of this designation is eligible for the Priority Review voucher if the drug is successfully approved. WVE-210201 received this designation in August.
The designation display the potential that WVE-210201 has displayed as a treatment for Duchenne muscular dystrophy. This drug utilizes an exon-skipping mechanism which allows a functional version of dystrophin to be produced.
