Friday, June 02, 2023
Vanda Pharmaceuticals, a company listed on Nasdaq under the ticker symbol VNDA, has announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to VCA-894A for the treatment of Charcot-Marie-Tooth disease, specifically axonal type 2S (CMT2S). This rare form of Charcot-Marie-Tooth disease has no approved treatment options available. CMT is an inherited peripheral neuropathy, and its prevalence is estimated to be 1 in 2,500 individuals. The clinical features of CMT can vary depending on the specific genetic variants. On the other hand, CMT2S is even rarer, with a prevalence estimated to be less than 1 in 1,000,000 worldwide. The disease typically manifests in early childhood and is characterized by progressive muscle weakness and atrophy in the distal regions of the body, particularly the upper and lower limbs. This leads to decreased reflexes and sensory impairment, often resulting in the need for wheelchair assistance as the disease progresses. VCA-894A is an antisense oligonucleotide (ASO) that specifically targets a cryptic splice site variant within the IGHMBP2 gene. Mutations in this gene play a significant role in the development of CMT2S, leading to the loss of alpha-motor neurons and the deterioration of the peripheral nervous system. ASOs have shown promise in modulating gene expression and providing personalized treatments for rare diseases. The Orphan Drug Designation granted by the FDA recognizes investigational therapies that address rare medical conditions and offers certain benefits to drug developers during the development process.