The U.S. Food and Drug Administration (FDA) has approved a recombinant enzyme replacement therapy for people with congenital thrombotic thrombocytopenic purpura (cTTP), an ultra-rare blood disorder. Adzynma, which is manufactured by Takeda Pharmaceuticals U.S.A., is a protein-based therapy indicated for prophylactic (preventive) or on demand use in adult and pediatric patients with cTTP.

cTTP is a subtype of thrombotic thrombocytopenic purpura, a chronic, and debilitating blood clotting disorder linked to mutations in the ADAMTS13 gene. It is characterized by a subsequent deficiency in the ADAMTS13 enzyme which leads to abnormal clotting in the small blood vessels of the body. Signs and symptoms typically arise in infancy/early childhood and include low platelet levels (thrombocytopenia), neurological dysfunction, and hemolytic anemia, a condition in which red blood cells are destroyed faster than they can be generated.

The condition has been linked to stroke and cardiovascular disease. Affected individuals will often experience a significantly decreased quality of life, along with a shortened lifespan, as compared to the general population. Left untreated, the condition can be lethal.

The safety and efficacy of Adzynma for both prophylactic and on-demand use, were demonstrated via a global clinical study, which compared it to existing human plasma-based treatments in people with cTTP.  

“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, MD, PhD, 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.”

Read the full FDA release.

Source: FDA news release dated November 9, 2023