Soleno Therapeutics Inc. (NASDAQ: SLNO) stock gains by 42.65% in the current market, at last check. Soleno focuses on the research and marketing of new treatments for rare illnesses. DCCR Extended-Release pills, a once-daily oral tablet developed by the business, is now being tested in Phase 3 clinical research program for the treatment of Prader-Willi syndrome (PWS).
What is happening?
The US Food and Drug Administration has given Orphan Drug Designation to Soleno Therapeutics’ experimental drug, DCCR Extended-Release tablets, in order to treat the Glycogen Storage Disease Type 1a (GSD 1a), also known as von Gierke disease. DCCR was formerly designated as an orphan drug for the treatment of Prader-Willi syndrome.
Orphan Medication Designation is granted by the FDA’s Office of Orphan Drug Products to encourage drug candidates in development for underserved patient populations or uncommon illnesses affecting fewer than 200,000 persons in the United States. Orphan Drug Designation entitles a candidate to a variety of research benefits, including tax subsidies for qualified clinical studies, application cost waivers, and seven years of commercial exclusivity after FDA approval.
Anish Bhatnagar, CEO of Soleno Therapeutics stated:
Soleno has reached a key milestone with the approval of Orphan Drug Designation for its DCCR development in a new indication, GSD 1a. GSD 1a is a kind of glycogen storage disorder in which the body is unable to convert glycogen to glucose, resulting in hypoglycemia, excessive blood fat levels, and stunted development, among other consequences. SLNO thinks that DCCR’s mode of action as an ATP-dependent potassium channel agonist, with the ability to control hypoglycemia and decrease fatty acid production, might give a substantial therapy alternative for GSD 1a, a disorder for which no authorized medications are presently available.