USFDA gives nod to world’s first enzyme replacement therapy for patients with rare blood clotting disorder

financialexpress.com:

The U.S. Food and Drug Administration (USFDA) on Thursday approved Adzynma, the first recombinant (genetically engineered) protein product indicated for on-demand enzyme replacement therapy (ERT) in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP), a rare and life-threatening blood clotting disorder. The FDA granted approval of Adzynma to Takeda Pharmaceuticals U.S.A. Inc.

“The FDA remains deeply committed in our efforts to help facilitate the development and approval of safe and effective therapies for patients with rare diseases,” said Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research. “Without treatment, cTTP is ultimately fatal. Today’s approval reflects important progress in the development of much-needed treatment options for patients affected by this life-threatening disorder.”

According to USFDA, The very rare, inherited blood clotting disorder called cTTP is caused by a disease-causing mutation in the ADAMTS13 gene, which is responsible for making an enzyme, also named ADAMTS13, that regulates blood clotting. A deficiency in this enzyme causes blood clots to form in the small blood vessels throughout the body. It is estimated that cTTP affects fewer than one thousand people in the United States.

Symptoms of this rare disorder typically develop in infancy or early childhood, but in some cases may develop in adulthood and may first manifest during pregnancy.

Individuals with cTTP may experience severe bleeding episodes, strokes and damage to vital organs. If left untreated, the disease can be fatal.

The US regulator maintains that the treatment for cTTP typically involves prophylactic plasma-based therapy for individuals with chronic