Galactosemia | Executive Insights | US | 2020
Galactosemia is a group of inherited metabolic disorders characterized by the body’s inability to produce a sufficient amount of enzymes to process galactose. The mostly commonly insufficient enzymes are GALT, GALK, and GALE. Patients are identified at birth through newborn screening (NBS) and are immediately put on a restricted diet, which is the only available treatment. However, dietary restriction does not always prevent long-term complications. Galactosemia is characterized by a varied phenotypic spectrum, ranging from normal development to severe complications, such as jaundice, cataracts, failure to thrive, bacterial sepsis, learning difficulties, neurological impairment, primary ovarian insufficiency (POI), and liver and kidney disease. Some therapies with novel mechanisms of action are in early clinical development and, if efficacious, stand to capitalize on the immense unmet need for effective therapies in this space.
QUESTIONS ANSWERED
- What are the key unmet needs and opportunities in the galactosemia therapy market?
- What is the incidence of galactosemia in the United States? How are the total diagnosed cases segmented by subtype?
- How are galactosemia patients currently treated in the United States?
- What is the expected impact of new therapies launching for galactosemia?
- How does each current and future player influence the market, and how will this scenario change in the future?
- What are the key drivers and limiters of the galactosemia therapy market?
GEOGRAPHY
United States
PRIMARY RESEARCH
3 KOL interviews in May 2020
KEY COMPANIES COVERED
Applied Therapeutics, Moderna Therapeutics
KEY DRUGS COVERED
AT-007, GALT mRNA, salubrinal, GALK1 inhibitors
PRODUCT DESCRIPTION
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.
