The FDA has recently granted the Rare Pediatric Disease (RPD) designation to AMO-02, a treatment for congenital myotonic dystrophy. As there is currently an unmet medical need for this condition, this designation is a big step in the right direction towards viable treatment options for patients.
About Congenital Myotonic Dystrophy
Congenital myotonic dystrophy is a form of muscular dystrophy that is characterized by the progressive weakening and atrophy of the muscles. There are two forms of this condition: type 1 and type 2, with the latter often resulting in less severe symptoms and affecting different muscles. Type 1 is the result of a mutated DMPK gene, while type 2 occurs due to a mutated ZNF9 gene. Common symptoms of both forms include an abnormal heart rhythm, weakness of the cardiac muscles, problems with breathing, weakness in the voluntary muscles, cataracts, learning disabilities, an inability to relax the muscles, issues swallowing, gallstones, constipation, abnormal uterus muscles, and issues with speech, vision, and hearing. There is currently no cure or treatment specific to myotonic dystrophy; treatment is symptomatic.
About the Rare Pediatric Disease Designation
The RPD designation is reserved for therapies that aim to treat a severe or life-threatening disease that primarily impacts people below the age of 18 and less than 200,000 people in the US. When a company is granted this designation they are also given an incentive: a priority review voucher. This voucher can be used by the company that earned it or sold.
To continue the development of AMO-02, there will be a double-blind, randomized, and placebo-controlled trial. Researchers plan to enroll 56 patients across eleven centers throughout the world, where they will be evaluated on muscle function and measures of congenital myotonic dystrophy features.
Hopefully the data from this trial will be positive and support the submission of a marketing authorization application in the future.
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