Release Date: 22-Sep-2021
US FDA has granted orphan drug designation to Ganaxolone for treatment in TSC. Ganaxolone is a helpful allosteric modulator of GABAA receptors, is an investigational product being established in intravenous and oral formulations intended to make the most of therapeutic reach to adult and pediatric patient populations in both acute and chronic care settings. Ganaxolone exhibits anti-anxiety and anti-seizure activity through its effects on synaptic and extrasynaptic GABAA receptors.
TSC is a main cause of genetic epilepsy, often occurring in the first year of life as either focal seizures or infantile spasms. Currently, there are limited approved treatments for TSC. Tuberous sclerosis complex (TSC) is a rare genetic disorder that affects several organs and causes non-malignant tumors in the skin, brain, kidney, lungs, eyes, heart. The disorder is caused by inherited mutations in either the TSC1 gene or the TSC2 gene. TSC occurs with a frequency of 1:6,000 and a mutation is found in 85% of patients.
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.