Takeda announced at the end of last month (November 2023) that it had been approved by the US Food and Drug Administration (FDA) on November 9, 2023.
cTTP is a very rare, complex condition that can present as an acute life-threatening disorder requiring prompt diagnosis, early referral, and effective immediate and ongoing management in a center with comprehensive provision and a multi-discipline approach.
Adzynma is the first and only recombinant ADAMTS13 enzyme replacement therapy for people with cTTP designed to address the root cause of the condition, an ADAMTS13 enzyme deficiency.
On-demand treatment for cTTP
Adults and children in the US with cTTP can now access Adzynma for prophylactic and on-demand treatment.
“For nearly a century, people impacted by this debilitating, ultra-rare condition have been without a treatment option specifically indicated for their disease,” said Cheryl Schwartz, senior vice president of, Rare Disease Business Unit at Takeda.
“Patients have waited too long, and so it is with great pride that we announce the availability of the first and only FDA-approved treatment for patients with cTTP to directly address their ADAMTS13 enzyme deficiency.”
Significant milestone for cTTP community
The ultra-rare, chronic blood clotting disorder caused by a deficiency in the ADAMTS13 enzyme is associated with acute events and debilitating chronic symptoms or thrombotic thrombocytopenic purpura (TTP) manifestations. These can include thrombocytopenia, microangiopathic hemolytic anemia, headache, and abdominal pain. When left untreated, acute TTP events have a mortality rate of more than 90%.
“The availability of this treatment marks a significant milestone for the cTTP community,” said James Wynn, president and co-founder, The Ree Wynn Foundation.
“It is an honor to stand with the community as we recognize this turning point for the treatment of cTTP, and ultimately, work to improve outcomes for patients.”
cTTP – idiopathic and secondary forms
The treatment was previously granted Orphan Drug Designation (ODD) by the US FDA for the treatment and prevention of TTP, including its acquired idiopathic and secondary forms, as well as Fast Track and Rare Pediatric Disease Designation.
The FDA granted Takeda a Rare Pediatric Disease Priority Review Voucher for the approval of Adyznmz. It has also been granted ODD by the European Medicines Agency (EMA) and Japan’s Ministry of Health, Labour and Welfare (MHLW) for the treatment of TTP.
In its research, Takeda found that TTP has an estimated prevalence of 2-6 cases per million. The inherited form of the disease, cTTP, accounts for the same as or more than 5% of TTP patients. It develops due to deficiency in ADAMTS13, a von Willebrand factor (VWF) cleaving protease, which results in the accumulation of ultra-large VWF multimers in the blood.
Uncontrolled platelet manifestations
The accumulation of ultra-large VWF multimers leads to uncontrolled platelet aggregation and adhesion. This can lead to abnormal clotting in the small blood vessels of the body and is associated with microangiopathic hemolytic anemia and low platelet levels known as thrombocytopenia.
Takeda says cTTP has both acute and chronic manifestations including stroke and cardiovascular disease and can also cause ongoing widespread organ damage and other co-morbidities resulting from an ADAMTS13-deficient state if left untreated.
Takeda says it is focused on ‘creating better health for people and a brighter future for the world’.
The company said: “We aim to discover and deliver life-transforming treatments in our core therapeutic and business areas, including gastrointestinal and inflammation, rare diseases, plasma-derived therapies, oncology, neuroscience and vaccines.”
