According to a story from Business Wire, the gene therapy company Myonexus Therapeutics announced that the U.S. FDA has given Rare Pediatric Disease Designation for its gene therapy candidate MYO-101. The therapy is in development for the treatment of rare limb girdle muscular dystrophy type 2E. This announcement comes after MYO-101 was previously granted Orphan Drug Designation earlier in the year, in February.
Limb girdle muscular dystrophy (LGMD) is a rare subtype of muscular dystrophy that is distinguished by which muscles are first affected. This type often affects the hips and shoulders first. There is a significant variance in severity and progression among patients; symptoms include hypertrophy, breathing problems, palpitations, lower back discomfort, and weakness of the shoulder and facial muscles. Onset of symptoms typically begins between 10 and 30 years old; generally, the earlier limb girdle muscular dystrophy symptoms appear, the more severe the case becomes. Patients must take extra care to monitor their heart and respiratory health. Physical therapy can help patients maintain muscle strength in some cases. Currently, there is no medical treatment currently available that is designed specifically to target limb girdle muscular dystrophy. To learn more about limb girdle muscular dystrophy, click here.
Type 2E is one of the most severe types of LGMD, often appearing before age ten. It can also result in rapid deterioration of walking ability, and unlike many forms of LGMD, can shorten the lifespan considerably, with most patients dying before age 30. Preliminary data for MYO-101 has been promising so far, and Myonexus plans to begin trials for the therapy soon.
The Rare Pediatric Disease designation is only reserved for drugs that are in development for treating a disease that primarily affects people under the age of 18. In addition, the disease must be considered rare, which, under U.S. Law, is any disease that affects 200,000 or less in the country. About half of rare diseases are known to affect children. In The new drug must also demonstrate the capability to fulfill an unmet medical need or offer a substantial improvement in quality of life for patients. A company that receives the designation is eligible to receive a priority review voucher, which allows a drug to be approved in six months instead of the standard of ten months.
Hopefully, MYO-101 will continue to display positive results in the trials to come.