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FDA Approves Takeda's Adzynma as First Treatment for Ultra-Rare Blood Clotting Disorder cTTP

• The FDA has approved Takeda's Adzynma (ADAMTS13, recombinant-krhn) as the first therapeutic option for congenital thrombotic thrombocytopenic purpura (cTTP), an ultra-rare blood clotting disorder affecting fewer than 1,000 people in the US.

• Clinical trials demonstrated Adzynma's efficacy with no patients experiencing acute TTP events while on prophylactic treatment, compared to one event in the plasma-based therapy group, offering a significant advancement over current plasma-based treatments.

• Adzynma works by replacing the deficient ADAMTS13 enzyme that regulates blood clotting, providing both prophylactic and on-demand treatment options for adult and pediatric patients with cTTP, a condition that has a >90% mortality rate when left untreated.

Takeda Pharmaceutical has achieved a significant breakthrough in rare disease treatment with the U.S. Food and Drug Administration's approval of Adzynma (ADAMTS13, recombinant-krhn) for congenital thrombotic thrombocytopenic purpura (cTTP). This approval marks the first therapeutic option specifically designed for this ultra-rare inherited blood clotting disorder.
cTTP affects fewer than 1,000 people in the United States and is caused by a deficiency in the ADAMTS13 enzyme, which plays a crucial role in regulating blood clotting. Without treatment, the condition is ultimately fatal, with mortality rates exceeding 90% for acute events.

First Recombinant Enzyme Replacement Therapy for cTTP

Adzynma is a purified recombinant form of the ADAMTS13 enzyme that directly addresses the underlying cause of cTTP by providing a replacement for the deficient enzyme. The FDA has authorized its use as both a preventative and on-demand treatment for adult and pediatric patients.
"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 Takeda's U.S. Business Unit. "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."

Clinical Evidence of Superior Efficacy

The FDA's approval was supported by data from a late-stage randomized, controlled, open-label, crossover Phase 3 trial. In this study, patients were randomized to receive either Adzynma or plasma-based therapies for six months before crossing over to the alternative treatment for another six months.
Key findings from the trial demonstrated Adzynma's clinical benefits:
  • No patient experienced an acute thrombotic thrombocytopenic purpura (TTP) event while receiving Adzynma as a preventative treatment (n=37), compared to one acute TTP event in a patient receiving plasma-based therapies (n=38).
  • No subacute TTP events were reported in patients receiving Adzynma during the controlled comparison periods, compared to five subacute TTP events in four patients receiving plasma-based therapies.
  • The mean annualized rate of thrombocytopenia manifestations was lower with Adzynma (2.0) compared to plasma-based therapies (4.44).
In pharmacokinetic assessments, patients receiving 40 IU/kg of Adzynma intravenously achieved a four-to-five-fold increase in ADAMTS13 activity following a single infusion compared to plasma-based therapies.

Addressing an Unmet Medical Need

Until now, cTTP treatment has typically involved plasma therapy, which Takeda has described as "insufficient in restoring ADAMTS13, time-consuming and costly."
Dr. Peter Marks, director of the FDA's Center for Biologics Evaluation and Research, emphasized the importance of this approval: "Without treatment, cTTP is ultimately fatal. [The] approval reflects important progress in the development of much-needed treatment options for patients affected by this life-threatening disorder."
cTTP is associated with severe bleeding episodes, strokes, and damage to vital organs. The condition manifests with both acute events and debilitating chronic symptoms, including thrombocytopenia, microangiopathic hemolytic anemia, headache, and abdominal pain.

Safety Profile and Common Side Effects

Adzynma demonstrated a favorable safety profile compared to plasma-based therapies. The most common adverse reactions (incidence >5%) were headache, diarrhea, migraine, abdominal pain, nausea, upper respiratory tract infection, dizziness, and vomiting. Importantly, no patients receiving Adzynma developed neutralizing antibodies during the clinical trials.

Expert Perspective

Dr. Spero Cataland, professor of internal medicine at the Wexner Medical Center at The Ohio State University and ADZYNMA clinical trial investigator, highlighted the significance of this development: "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."
He added, "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."

Regulatory Designations

Prior to approval, Adzynma received multiple special designations from regulatory authorities, including Orphan Drug Designation, Fast Track, and Rare Pediatric Disease Designation from the U.S. FDA. The European Medicines Agency and Japan's Ministry of Health, Labour and Welfare have also granted Orphan Drug Designation for the treatment of TTP.
This approval represents a continuation of Takeda's 70-plus year legacy in hematology innovation and reinforces the company's commitment to addressing unmet needs in rare diseases.
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