FDA Approves First Recombinant Treatment for Rare Blood Clotting Disorder

On Nov. 9, 2023, FDA announced the approval of Adzynma, the first genetically engineered protein product for preventative or on-demand enzyme replacement therapy (ERT) for adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP). cTTP, a rare and life-threatening blood clotting disorder, is an inherited disorder caused by a mutation in the ADAMTS13 gene, which is responsible for making an enzyme of the same name that regulates blood clotting. cTTP affects an estimated fewer than 1000 people in the United States and can be fatal if left untreated. Treatment has typically involved prophylactic plasma-based therapy for those with chronic disease, to replenish the ADAMTS13 enzyme.

Adzynma is a purified recombinant form of the ADAMTS13 enzyme, and it provides a replacement for low levels of the enzyme in patients with cTTP. For prophylactic (preventative) ERT, Adzynma is given to reduce the risk of disease symptoms. It can also be given as an on-demand ERT for treatment when a patient is experiencing sudden and severe symptoms. Adzynma is administered intravenously once every other week for prophylactic ERT, and once daily for on-demand ERT.

“Without treatment, cTTP is ultimately fatal,” said Peter Marks, director of FDA’s Center for Biologics Evaluation and Research, in a FDA press release. “Today’s approval reflects important progress in the development of much-needed treatment options for patients affected by this life-threatening disorder.”

FDA’s approval was based on a global study evaluating prophylactic and on-demand ERT with Adzynma compared to plasma-based therapies in patients with cTTP. The application for Adzynma was awarded a Rare Pediatric Disease Priority Review Voucher, and was granted Priority Review, Fast Track, and Orphan designations. FDA granted approval of Adzynma to Takeda Pharmaceuticals U.S.A. Inc.

Source: FDA