Fabry Disease Treatment

Fabry Disease Treatment

Fabry disease is a rare genetic disorder caused by the deficiency of an enzyme called alpha-galactosidase A. This deficiency leads to the accumulation of certain fatty substances in the body, affecting various organs and systems. Symptoms often start in childhood or adolescence and can include pain, skin lesions, gastrointestinal issues, and problems with the heart, kidneys, and nervous system. Early diagnosis and management are crucial to help alleviate symptoms and prevent complications. Fabry disease treatment is gaining prominence in the market owing to the increasing prevalence of fabry disease, rising cases of obesity, and rising demand for oral drugs. For instance, according to the National Institute of Health, classic fabry disease mutations are seen in approximately 1 in 30,000 males, and atypical presentations are associated with about 1 in 15,000 males and 1 in 23,000 females. Several factors, including a surge in the increasing awareness regarding inherited disorders, an increasing geriatric population, and increased investments from the government, public, and private sectors in healthcare departments are driving the growth of the market during the forecast period. According to the World Health Organization (WHO), the share of people aged above 60 will rise to 1.4 billion by 2030 while this share will increase to 2.1 billion by 2050 which would be more than double the share of the geriatric population in the year 2020 i.e., 1 billion.

The Fabry Disease Treatment Market is expected to grow at a steady rate of around 7.1% owing to the increased prevalence of obesity which plays an important role in the progression of fabry disease. For instance, as per the World Obesity Atlas 2022, estimations are such that 1 billion people worldwide, comprising one in five women and one in seven men will be suffering from obesity by the year 2030. Many new therapies are being introduced for treating these inherited disorders. Several other factors, such as rising genetic disorders among people are also driving this market of fabry disease treatment at a steady rate. For instance, according to the World Health Organization, genetic disorders and congenital abnormalities occur in about 2-5% of all live births.

• Based on the mode of administration, the market is bifurcated into oral and intravenous segments. The oral segment held the maximum share in the global market of fabry disease treatment in 2022. This segment dominated the market because of the rising demand for oral drugs among patients. Tablets are reported to be the most consumed solid dosage forms among all medication forms, because of their highly effective composition, and ease of consumption. The oral mode of drug administration is much more convenient and easy on pockets for consumers, boosting this segment’s growth. Hence, the oral segment held a significant share of the market in 2022 due to the above reasons.
• Based on treatment, the market is categorized into substrate reduction therapy, enzyme replacement therapy, chaperone treatment, and others. The enzyme replacement therapy segment is expected to hold a significant share of the market in the forecast period owing to the efficient results of the therapy. Fabrazyme restores levels of alpha-galactosidase A. This allows the body to break down lipids and relieve the symptoms of fabry disease. For instance, according to Fabry Disease News in August 2023, enzyme replacement therapy has proved to be extremely helpful to fabry patients. Apart from this, the surge in the usage of rapid diagnostic tests is also impelling the growth of this segment. Hence, the enzyme replacement therapy category is expected to witness a higher CAGR during the forecast period amongst applications.
• For a better understanding of the market adoption of fabry disease treatment, the market is analyzed based on its worldwide presence in countries such as North America (U.S., Canada, and Rest of North America), Europe (Germany, U.K., France, Spain, Italy, Rest of Europe), Asia-Pacific (China, Japan, India, Rest of Asia-Pacific), Rest of World. North America dominated the global fabry disease treatment market in 2022 due to the increasing genetic disorders, increased fabry disease cases, and rising investments in developing effective therapeutics. For instance, according to the National Fabry Disease Foundation, the number of individuals carrying the Fabry disease gene in the U.S. at over 11,000 according to currently published statistics of 2023. Government organizations are initiating awareness programs to prevent these inherited neurological disorders and provide efficient care to patients. Thus, North America held a significant share of the market in the year 2022.
• Some of the major players operating in the market include Pfizer Inc.; Merck KGaA; Sanofi; Biocare Medical, LLC; Lilly; Teva Pharmaceutical Industries Ltd.; MITSUBISHI CHEMICAL GROUP CORPORATION; Bristol-Myers Squibb Company; GSK plc.; Bayer AG.