The UK Fabry Disease Therapeutics market size stood at around USD xx billion in 2020 and is projected to reach USD xx billion by 2028, exhibiting a CAGR of xx% during the forecast period.
Fabry disease (also known as Anderson-Fabry disease) is a rare genetic disorder that is caused by the deficiency of defective gene i.e., alpha-galactosidase-A gene (GLA gene) which leads to deficiency of an alpha-galactosidase enzyme. Alpha-galactosidase A (AGA) enzyme hydrolyses the terminal a -galactosyl moieties from glycolipids and glycoproteins. This disease is caused by an abnormal build-up of a specific fatty matter called globotriaosylceramide in multiple tissues of the body including the eyes, skin, kidneys, gastrointestinal systems, brain, heart, and central nervous systems.
People probably carry from 5 to 10 genes with mutations in each of their cells. It is estimated that nearly 6% of the UK population (around a 3.5million people) will be affected by a rare disease at some point in their lives. It has been estimated that Fabry disease affects between 1:40,000 people, in males, it is around 1:17,000. Fabry is seen across all ethnic groups. As per the publication developed for the Fabry International Network by MPS Commercial, UK, the average age of 44.5 years of Fabry affected patients has been reported while 81% of women and 61% of men has been found the classical Fabry disease.
Market Growth Drivers
The growing prevalence of Fabry disease and its consequences on healthcare expenditure has augmented the demand for special treatments, positively impacting the Fabry disease treatment market growth during the forecast period. The increasing focus on research activities and the development of novel therapeutic drugs has uplifted the Fabry disease treatment market value.
Moreover, favorable government policies aiming at creating awareness pertaining to the Fabry disease treatment are leading to increased adoption of effective treatments of Fabry disease during the forecast period.
However, there are several factors hindering the efficient and effective clinical trials of drug development of Fabry disease including low patient numbers, limited understanding of pathology and progression, and lack of established endpoints. Therefore, UK regulatory bodies are engaged in implementing regulatory standards for drug approval for Fabry disease to ensure patient safety and efficacy of drugs.