Clinical-stage biopharmaceutical company Soleno Therapeutics, Inc. (NASDAQ: SLNO) recently announced that its drug DCCR (diazoxide choline) Extended-Release tablets has received Orphan Drug Designation from U.S. Food and Drug Administration (FDA). The drug is to be used for the treatment of von Gierke disease, scientifically called Glycogen Storage Disease Type 1a or GSD 1a. Earlier, FDA has granted Orphan Drug Designation to the same drug for Prader-Willi syndrome treatment.
Soleno Therapeutics achieving a “significant milestone” with this
Getting orphan drug designation for the additional indication is the company’s new milestone, as stated by its CEO, Anish Bhatnagar. As explained, GSD 1a is a glycogen storage disease that indicates that the human body is incapable of metabolizing glycogen into glucose. This, in turn, results in hypoglycemia, or layman terms, a high level of fat in the blood. The disease causes impaired growth apart from several other complications.
DCCR works by regulating hypoglycemia, which in turn reduces fatty acid synthesis. This is pitches as the potential treatment of GSD 1a, where no other approved therapies are available until now.
GSD 1a to benefit from orphan drug designation status
The FDA provides orphan drug designation to this treatment to support drug candidates in treatments with no visible development for rare diseases or underserved patient populations. This can be fewer than 200,000 people in the U.S. With Orphan drug designation, GSD 1a has qualified for different development incentives, tax credits, waiver application fee, along with market exclusivity for the next seven years, once FDA approves the drug.
DCCR is the lead candidate of the company and is a once-daily oral tablet projected as Extended-Release tablets. DCCR, at present, is in the evaluation stage. It is in Phase-3 clinical development program mainly for Prader-Willi syndrome treatment. GSD 1a is not in any clinical development program as of now. The orphan drug designation by FDA will help the company is taking it up further. The new status for GSD 1a gives a new landmark to Soleno Therapeutics.