Spinocerebellar ataxia (SCA) is a heterogeneous group of rare neurodegenerative progressive disorders that primarily affects the cerebellum, and, in some cases, the spinal cord, resulting in the loss of balance and movement coordination. SCA subtypes 1, 2, and 3 are among the most common subtypes. Given the rarity of the condition and the symptomatic similarity to other progressive neurological disorders, reaching a definitive diagnosis of SCA is often a lengthy process. No therapies are approved to treat SCA; treatment consists entirely of symptomatic therapies used off-label, often together with supportive nonpharmacological approaches (e.g., physical therapy). As a result, the burden on patients and caregivers is high. Thus, a critical need exists for treatment options—both disease-modifying and symptomatic therapies—that can address SCA symptoms better than current off-label options. Given the debilitating nature of the disease and the lack of approved therapies, the market is primed to accept any safe, even modestly effective treatment at premium pricing.
Executive Insights provides indication-specific market intelligence with world-class epidemiology, keen insight into current treatment paradigms, biotherapeutic pipelines, key clinical unmet needs, and competitive landscapes, supported by primary and secondary research. This solution provides succinct insights to any biopharma function or business professional looking to quickly grasp a new indication of interest.
GEOGRAPHY: United States, EU5, Portugal, China
PRIMARY RESEARCH: 8 KOL interviews in June-July 2021
KEY COMPANIES COVERED: Biohaven Pharmaceuticals, Steminent, Sclnow Biotechnology, Seelos Therapeutics
KEY DRUGS COVERED: Troriluzole, Stemchymal, SCLnow -19#, trehalose