Adzynma is a recombinant protein designed to replace the deficient ADAMTS13 enzyme to treat patients with the rare hereditary blood clotting disorder congenital thrombotic thrombocytopenic purpura
Takeda has launched Adzynma (ADAMTS13, recombinant-krhn) to treat adults and children with congenital thrombotic thrombocytopenic purpura (cTTP). The FDA granted approval to Adzynma in early November 2023 as a prophylactic or on-demand enzyme replacement therapy.
CTTP is rare hereditary blood clotting disorder stemming from a disease-causing mutation in the ADAMTS13 gene. This gene plays a crucial role in producing the ADAMTS13 enzyme, which is responsible for regulating blood clotting.
In people with cTTP, a deficiency in this enzyme leads to the formation of blood clots in small vessels throughout the body. This life-threatening condition, affecting fewer than one thousand people in the United States, typically begins in infancy or early childhood, with some cases emerging in adulthood or during pregnancy.
Adzynma is a purified recombinant form of the ADAMTS13 enzyme, designed to replace the insufficient levels of the deficient enzyme in cTTP patients. Administered as an injection, Adzynma serves both as a prophylactic ERT, to prevent disease symptoms, and as on-demand ERT, for acute events. The prophylactic regimen involves bi-weekly intravenous administrations, while on-demand treatment requires daily doses.
“For nearly a century, people impacted by this debilitating, ultra-rare condition have been without a treatment option specifically indicated for their disease,” Cheryl Schwartz, senior vice president, Rare Disease Business Unit at Takeda, said in a press release.
Adzynma will have a list price of $3.28 per IU, or about $245,000 annually for a 72 kg patient receiving prophylactic treatment every other week, according to a Takeda spokesperson. The exact cost, however, will vary depending on patient weight and whether the patient is receiving prophylactic or on-demand treatment. Adzynma will be available through a network of specialty pharmacies and distributors.
Regardless of insurance status or benefit design, ensuring patients have access to the medicines they need is a top priority for Takeda, the spokesperson said. The company provides patient support programs that can assist eligible patients – both those with and without insurance – in accessing medicines.
The FDA approval was supported by the totality of the evidence provided by the analysis of efficacy, pharmacokinetic, safety and tolerability data from a phase 3 trial that enrolled 37 patients who received the enzyme therapy, as well as by data from the continuation trial. In the phase 3 trial, no patient treated with Adzynma experienced an acute TTP event.
The most common adverse reactions were headache, diarrhea, migraine, abdominal pain, nausea, upper respiratory tract infection, dizziness and vomiting.