by The cerebrotendinous xanthomatosis market deals with products related to treating the rare metabolic disorder cerebrotendinous xanthomatosis (CTX). CTX is characterized by tendon xanthomas, cataracts, neurological problems, and increased cholesterol levels caused due to a deficiency of the CYP27A1 gene enzyme. Effective pharmaceutical therapies for managing symptoms and slowing disease progression include chenodeoxycholic acid, which helps eliminate toxic bile alcohols from the body.
The global cerebrotendinous xanthomatosis market size is estimated to be valued at US$150 million in 2031 and is expected to register a CAGR of 7% over the forecast period 2021-2031.
CTX remains under-diagnosed due to its rarity and non-specific symptoms. However, increased awareness initiatives by advocacy groups have resulted in more accurate diagnoses. Pharmaceutical firms have also stepped up efforts in orphan drug development. For instance, clinical trials are ongoing to study combination therapies and new drug delivery methods. The availability of standardized diagnostic protocols and management guidelines issued by medical experts are also boosting market growth prospects.
Key Takeaways
Key players operating in the cerebrotendinous xanthomatosis market are Avion Pharmaceuticals, LLC, CEREPUR, and RETROPHIN, INC.
Growing public knowledge about rare diseases and facilitating access to screening and treatment have augmented demand for CTX therapies. Product sales increased by 15% between 2018-2020 driven by improved patient identification in Europe and North America.
Pharmaceutical manufacturers are expanding their global footprint through partnerships with hospitals and labs in Asia Pacific and Latin America. This allows for early intervention, prevention of long-term disabilities, and better management of the condition.
Market Key Trends
A major driver for the cerebrotendinous xanthomatosis market is rising genetic testing adoption. Next-generation sequencing methodologies enable mutation analysis and pre-symptomatic diagnosis. This helps physicians initiate treatment at preliminary disease stages and halt the pathological process. From 2021, genetic tests for CTX have become more affordable and accessible through collaborations between biotech companies and managed care providers. Widespread genetic screening programs are expected to quadruple the diagnosed patient numbers in the next decade.
Porter’s Analysis
Threat of new entrants: Low, as there is high cost of R&D and barriers to develop drugs for rare genetic disorders.
Bargaining power of buyers: Moderate, as there are limited treatment options for buyers to choose from.
Bargaining power of suppliers: High, as there are few companies developing drugs for this rare disease.
Threat of new substitutes: Low, as there are no close substitute treatment available.
Competitive rivalry: Intense, as leading players aim to gain majority market share.
Geographical Regions
North America region currently holds majority of the global Cerebrotendinous Xanthomatosis market in terms of value, as the awareness regarding rare diseases is high and healthcare infrastructure is robust in the US and Canada.
Asia Pacific region is poised to witness fastest growth during the forecast period, attributed to rising healthcare expenditure, rapidly growing economies, and increasing awareness about rare diseases in countries like China, India and Japan.
*Note:
1. Source: Coherent Market Insights, Public sources, Desk research
2. We have leveraged AI tools to mine information and compile it
Money Singh is a seasoned content writer with over four years of experience in the market research sector. Her expertise spans various industries, including food and beverages, biotechnology, chemical and materials, defense and aerospace, consumer goods, etc.
