Azafaros B.V. today announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for AZ-3102, a novel oral small molecule, in GM2 gangliosidosis including both Sandhoff and Tay-Sachs diseases. The ODD for GM2 gangliosidosis has been granted based on efficacy demonstrated in a Sandhoff mouse model, including a clear effect on animal survival. These promising preclinical findings support Azafaros’ strategy to develop AZ-3102 as a potential disease-modifying treatment for GM1 and GM2 gangliosidoses and other neurogenetic, pediatric, metabolic lysosomal disorders.
Source:- Azafaros B.V.
The prevalence of Tay-Sachs disease is 1 case per 320 000 live births. The AB variant is extremely rare; only a few cases have been reported worldwide. Based on a few sources, the incidence of Sandhoff disease is approximately 1 out of every 180,000 live births worldwide. Among the diagnosed cases, 38%of patients had a history of developmental delay or regression, and ~23% had seizures. ~56% of patients had a dysmorphic face with protruding forehead, depressed nasal bridge, and hypertelorism. ~21 patients had blond hair.
Comprehensive insight on patient segmentation based on pheno-types (Tay-Sachs disease & Sandhoff disease) Age group, Sex, Types (Infantile, Juvenile & Late-onset), Symptoms (developmental regression, respiratory and urinary tract infections, central hypotonicity, spasticity, and visual disorders), and Genetic factors (HEXA, HEXB, and GM2A, etc.) has been provided into the epidemiology (Incidence and Prevalence) section of the GM2 Gangliosidoses and its treatment in the 8 MM countries, covering the United States, EU5 (Germany, Spain, France, Italy, UK), Japan, and China
Despite high unmet needs, there are few assets under development to treat GM gangliosidosis. Some of the key assets are IB-1001 from Intrabio, AXO-AAV-GM2 from Roivant Sciences and Axovant, CAM-GM201 from Sanofi, Tay-Sachs Program from Gene Therapy Research Institution Co. LTD, Linoleic acid ethyl ester homolog from Retrotope, Tay Sachs gene therapy from Amicus Therapeutics, and another Tay Sachs gene therapy from Recursion Pharmaceuticals. Enzyme replacement therapy & Gene therapy are being studied as another possible approach to treat lysosomal storage disorders. The launch of any of these therapies will positively impact the overall market from 2022 to 2025. Chaperone therapy is also being studied for Tay-Sachs disease. This type of therapy involves very small molecules that attach to newly-created hexosaminidase A enzymes, before the mutated enzymes are broken down, and guide them to the lysosome, where the enzymes can perform their normal function. Such a molecule can also cross the blood-brain barrier. This therapy is only in the initial stages of study, and more research is required for determining its long-term safety and effectiveness.
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