Takeda's enzyme replacement therapy receives FDA approval for life-threatening blood disorder

By Liza Laws

- Last updated on GMT

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© Getty Images

Related tags Rare disease Patient recruitment Patient centricity Clinical trial Takeda Pharmaceutical industry Japan

The first and only enzyme replacement therapy for the treatment of a very rare and complex blood disorder has been approved by the US Food and Drug Administration.

Takeda’s Adzynma (ADAMTS13, recombinant-krhn) for the treatment of congenital thrombotic thrombocytopenic purpura (cTTP) was given the green light on November 9. This is good news for any affected by the life-threatening disorder that needs prompt diagnosis, early referral, and effective, immediate management.

Takeda has been working with the rare hematology community for more than 70 years and is Japan’s biggest pharmaceutical company. The recombinant protein is designed to address an unmet medical need in people with cTTP by replacing the deficient ADAMTS13 enzyme.

Recombinant proteins are proteins encoded by recombinant DNA that have been cloned in an expression vector that supports the expression of the gene and translation of messenger RNA.

Life-threatening health challenge

“People living with cTTP face serious, life-threatening health challenges, and until today, were without any approved treatment specifically indicated for their disease,” said Julie Kim, president of US Business Unit and US country head at Takeda.

“As we strive to help patients with limited or no treatment options, developing innovative treatments in rare diseases is an inspiring challenge and one we have taken on for 70-plus years as a leader in hematology. Today, we are proud to further support the rare disease community by delivering Adzynma as the first FDA-approved therapeutic option for people with cTTP.”

Takeda explains that cTTP is associated with acute events and debilitating chronic symptoms or thrombotic thrombocytopenic purpura (TTP) manifestations, which can include thrombocytopenia, microangiopathic hemolytic anemia, headache, and abdominal pain. When left untreated, acute TTP events have a mortality rate of more than 90%.

Spero Cataland, is a professor of internal medicine at the Wexner Medical Center at The Ohio State University, co-director at the US Thrombotic Microangiopathy Alliance (USTMA), and Adzynma clinical trial investigator.

Patients living with rare disease

He said: “In recent decades, significant progress has been made to better understand the link between ADAMTS13 deficiency and cTTP, ultimately leading to this moment where we finally have an FDA-approved treatment option for patients living with this rare disease.”  

“Adzynma provides patients with a treatment option that replaces their deficient ADAMTS13 enzyme and offers a favorable efficacy and safety profile and reduced administration time and volume compared to current plasma-based therapies. Today marks a significant achievement, providing new possibilities for the cTTP patient community.”

The company said the FDA approval of Adzynma was supported by the totality of the evidence provided by the analysis of efficacy, pharmacokinetic, safety, and tolerability data from the first randomized, controlled, open-label, crossover phase 3 trial in cTTP as well as by data from the continuation trial.

The treatment was previously granted Orphan Drug Designation (ODD) by the 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. It also granted Takeda a Rare Pediatric Disease Voucher for the approval of Adzynma. 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. 

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