Takeda's Adzynma (ADAMTS13, recombinant-krhn) has been approved by the US Food and Drug Administration (FDA) as the first therapeutic option for congenital thrombotic thrombocytopenic purpura (cTTP), an ultra-rare inherited blood clotting disorder.

Estimated to affect fewer than 1,000 people in the US, cTTP is caused by a deficiency in the ADAMTS13 enzyme that regulates blood clotting.

Patients with cTTP can experience severe bleeding episodes, strokes and damage to vital organs, and mortality rates are high if left untreated.

"Without treatment, cTTP is ultimately fatal,” said Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research.

He continued: “[The] approval reflects important progress in the development of much-needed treatment options for patients affected by this life-threatening disorder.”

Until now, cTTP treatment has typically involved plasma therapy, which Takeda has previously described as "insufficient in restoring ADAMTS13, time-consuming and costly”.

Adzynma, which has been authorised for use as a preventative or on-demand treatment of adult and paediatric patients, is a purified recombinant form of the ADAMTS13 enzyme that works by providing a replacement for the low levels of the deficient enzyme.

The FDA’s decision was supported by results from a late-stage trial of cTTP patients who were randomised to receive six months of Adzynma or plasma-based therapies and then crossed over to the other treatment for another six.

No patient experienced an acute thrombotic thrombocytopenic purpura (TTP) event while receiving Adzynma as a preventative treatment, while there was one acute TTP event in a patient receiving plasma-based therapies, Takeda said.

Additionally, no subacute TTP events were reported in patients receiving Adzynma, compared to five subacute TTP events in four patients receiving plasma-based therapies.

Adzynma clinical trial investigator, Spero Cataland, 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 favourable efficacy and safety profile and reduced administration time and volume compared to current plasma-based therapies.”

https://www.pmlive.com/pharma_news/takedas_enzyme_replacement_therapy_approved_by_fda_for_rare_blood_clotting_disorder_1503781