Thanks to a Takeda approval Thursday, patients now have a drug to tackle a rare inherited blood clotting disorder.
The U.S. FDA has given a green light to Takeda’s Adzynma, the first recombinant protein product as a preventative or on demand enzyme replacement therapy in adults and kids with congenital thrombotic thrombocytopenic purpura (cTTP).
According to the FDA, cTTP is a very rare inherited blood clotting disorder caused by a disease-causing mutation in the ADAMTS13 gene, which makes an enzyme responsible for regulating blood clotting. Patients with a deficiency in this enzyme create blood clots in the small blood vessels throughout the body. The disease is estimated to affect fewer than 1,000 people in the U.S.
While symptoms of cTTP typically develop in infancy or early childhood, they can also crop up in adulthood and may manifest first during pregnancy, the FDA said. People with cTTP can suffer from severe bleeding episodes, strokes and damage to vital organs.
Until now, treatment has typically revolved around prophylactic plasma-based therapy, the regulator added.
Takeda’s Adzynma, for its part, is a recombinant form of the ADAMTS13 gene that helps patients by replacing low levels of the deficient enzyme. For protective enzyme replacement therapy, the drug is given to help reduce the risk of disease symptoms, though it can also be given on demand when patients are dealing with acute events.
Adzynma is administered through the vein once every other week in its protective form, and it’s given once daily for on-demand enzyme replacement therapy.
The FDA based its approval on a trial of 46 cTTP patients who either got six months of treatment with Adzynma or plasma-based therapies. Patients crossed over to the other drug after the study’s six-month mark, the FDA said.
Efficacy was determined based on incidence of thrombotic thrombocytopenic purpura (TTP) events and TPP manifestations, plus the incidence of the need for supplemental doses.
For on-demand enzyme replacement therapy, Takeda looked at the proportion of acute TTP events responding to its drug in both the prophylactic and on-demand cohorts throughout the study’s run. All acute and subacute TTP events were resolved after treatment with either Adzynma or plasma-based therapies, the FDA explained.
In terms of safety, the most common side effects associated with Adzynma were headache, diarrhea, migraine, abdominal pain, nausea, upper respiratory tract infection, dizziness and vomiting.
The green light for Adzynma marks Takeda’s second approval this week. The company has also received an FDA all-clear on its VEGFR inhibitor Fruzaqla, also known as fruquintinib, in adults with metastatic colorectal cancer as a third-line treatment.
Takeda pushed fruquintinib over the FDA finish line after acquiring its rights outside of China in January. The Tokyo company paid Hutchmed $400 million up front, with the potential for $730 million in milestone payments.