The Fabry Disease drugs in development market research report provides comprehensive information on the therapeutics under development for Fabry Disease, complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA), and molecule type. GlobalData’s report assesses key aspects of the companies and drugs in development for Fabry Disease. Buy the report here.

The report also covers the descriptive pharmacological action of the therapeutics and the latest news and press releases. Additionally, the report provides an overview of the key players involved in therapeutic development for Fabry Disease and features dormant and discontinued products.

GlobalData tracks 40 drugs in development for Fabry Disease by 33 companies/universities/institutes. The top development phase for Fabry Disease is preclinical with 17 drugs in that stage. The Fabry Disease pipeline has 37 drugs in development by companies and three by universities/ institutes. Some of the companies in the Fabry Disease pipeline products market are: Yuhan, Takeda Pharmaceutical and M6P Therapeutics.

The key targets in the Fabry Disease pipeline products market include Alpha Galactosidase A (Alpha D-Galactosidase A or Alpha D Galactoside Galactohydrolase or Melibiase or Agalsidase or GLA or EC 3.2.1.22), Ceramide Glucosyltransferase (Glucosylceramide Synthase or GLCT 1 or UDP Glucose:N Acylsphingosine D Glucosyltransferase or UDP Glucose Ceramide Glucosyltransferase or UGCG or EC 2.4.1.80), and Coagulation Factor IX (Christmas Factor or Plasma Thromboplastin Component or F9 or EC 3.4.21.22).

The key mechanisms of action in the Fabry Disease pipeline product include Alpha Galactosidase A (Alpha D Galactosidase A or Alpha D Galactoside Galactohydrolase or Melibiase or Agalsidase or GLA or EC 3.2.1.22) Replacement with 14 drugs in Phase III. The Fabry Disease pipeline products include five routes of administration with the top ROA being Intravenous and eight key molecule types in the Fabry Disease pipeline products market including Gene Therapy, and Recombinant Enzyme.

Fabry Disease overview

Fabry disease is an X-linked lysosomal disorder that results in abnormal deposits of globotriaosylceramide in blood vessel walls throughout the body. It is caused by mutations in the GLA gene. The GLA gene controls the production of a particular enzyme called alpha-galactosidase A, which is responsible for breaking down globotriaosylceramide. Symptoms include skin rash, cramps, gas, diarrhea, heart enlargement, angina, dizziness, headache, nausea, and heat intolerance. Treatment includes enzyme replacement therapy (ERT) and pain management.

For a complete picture of Fabry Disease’s pipeline drug market, buy the report here.

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GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

GlobalData’s pipeline drugs offers detailed profiles of pharmaceutical drugs in all stages of pre-clinical and clinical development, from discovery through to pre-registration. Coverage is limited to novel human medicinal drugs and biosimilars seeking market approval proprietary and is one of two primary repositories of pharmaceutical drug information offered by GlobalData through its Pharmaceutical Intelligence Center.