FDA Approves New Treatment for Rare Blood Clotting Disorder

Director Marks announced the therapy for the life-threatening disorder.

The U.S. Food and Drug Administration recently approved Adzynma for patients with congenital thrombotic thrombocytopenic purpura (cTTP). The genetically engineered protein is authorized for use as a preventive and as an enzyme replacement therapy for this rare, life-threatening blood-clotting disorder.

“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 FDA’s Center for Biologics Evaluation and Research Director Peter Marks. “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.”

As the Lord Leads, Pray with Us…

  • For FDA researchers and scientists as they test and review therapies and treatments for various diseases and disorders.
  • For Commissioner Califf to seek God’s direction as he heads the Food and Drug Administration.
  • That people who are suffering from cTTP would have their lives improved through the new medication.

Sources: Food and Drug Administration


Back to top