Tag Archives: eu orphan drug regulation

FDA orphan drug designation for ProTmune given to Fate Therapeutics

Fate therapeutics receives Orphan drug designation for prevention of graft-versus-host disease in patients undergoing allogeneic hematopoietic cell transplantation and also includes blood cancer and genetic disorders by Food and Drug Association (FDA). The company is committed to the development of programmed cellular immune therapies for cancer and immune disorders. The company boasts of the drug’s unique therapeutic potential to address life-threatening complications and improve the curative potential of allogeneic HCT.

Graft-versus-host disease (GvHD) is a severe immunological complication that arises when newly-transplanted donor immune cells attack the patient’s tissues and organs, resulting in a potentially fatal immune system reaction. Regardless of repetitive prevention practices, 50% of patients suffer from GvHD and Inspite of systematical treatment provided to patients only half of them responds positively.

ProTmune™ is an investigational programmed cellular immunotherapy undergoing clinical development for the prevention of acute GvHD and cytomegalovirus (CMV) infection in patients undergoing allogeneic HCT. The cell therapy is produced by modulating a donor-sourced, mobilized peripheral blood graft ex vivo with two small molecules (FT1050 and FT4145) to enhance the biological properties and therapeutic function of the graft’s immune cells. The programmed mobilized peripheral blood graft is administered to a patient as a one-time intravenous infusion.

Rare diseases are also known as Orphan diseases. These diseases are generally categorized as chronic, degenerative, and life-threatening. The rare diseases normally include certain cancers, metabolic conditions, diseases of the nervous system and musculoskeletal disorders, which are extremely life-threatening. As per the official definition, rare diseases are classified as those that affect fewer than 200,000 in the United States and less than 5 in 10,000 in the EU. These two regions form a dominant portion of the global market for orphan drugs.

For Free Sample of Global Orphan Drug Clinical Pipeline Reports Contact: avinash@kuickresearch.com
Or Visit:  Global Orphan Drug Market Future Outlook 2020