Diazoxide Choline Receives Orphan Drug Designation To Treat Glycogen Storage Disease Type 1a

Release Date: 08-Sep-2021

US FDA has granted Orphan Drug Designation to DCCR (diazoxide choline) Extended-Release tablets to treat Glycogen Storage Disease Type 1a (GSD 1a), or von Gierke disease. GSD 1a is a disease marked by the body’s inability to metabolize glycogen into glucose, leading to hypoglycemia, impaired growth, and high levels of fat in the blood, among other complications.


DCCR’s mechanism of action is an ATP-dependent potassium channel agonist, with the potential to regulate hypoglycemia and reduce fatty acid synthesis that could provide a meaningful treatment option for GSD 1a. There is no clinical development program going on for GSD 1a. DCCR previously had also received Orphan Drug Designation for the treatment of Prader-Willi syndrome.


The FDA's orphan-drug program gives special status to drugs and biologics for diseases and disorders that affect fewer than 200,000 people in the U.S and it also gives several benefits, like tax credits for qualified clinical trials, potential seven years of market exclusivity after approval and exemption from user fees.

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