The US Food and Drug Administration (FDA) has granted approval for Takeda’s Adzynma (recombinant-krhn ADAMTS13 ) for congenital thrombotic thrombocytopenic purpura (cTTP), a rare blood clotting disorder.

Adzynma is indicated for use as a prophylactic and on-demand therapy in adult and paediatric cTTP patients.

It is the first and only recombinant A disintegrin and metalloproteinase with thrombospondin motifs 13 protein to receive approval for this indication.

This protein treatment works by replacing the deficient ADAMTS13 enzyme.

The latest development is based on findings from an open-label, controlled, randomised, crossover Phase III clinical trial and continuation study.

The Phase III trial evaluated the safety, efficacy, tolerability and pharmacokinetics of intravenous doses of Adzynma versus plasma-based therapies in cTTP patients.

No subject had an acute TTP event in the Adzynma prophylactic treatment arm, while one who received plasma-based therapies did so.

In the Adzynma group, the mean annualised event rate of thrombocytopenia events was two, versus 4.44 in those receiving plasma-based therapies.

The pharmacokinetic data showed that patients who received a single dose of Adzynma attained a four to five-fold rise in ADAMTS13 activity versus plasma-based therapies.

Adzynma also showed a favourable safety profile compared to plasma-based therapies.

Takeda US business unit president and country head Julie Kim stated: “People living with cTTP face serious, life-threatening health challenges and until today were without any approved treatment specifically indicated for their disease.

“We are proud to further support the rare disease community by delivering Adzynma as the first FDA-approved therapeutic option for people with cTTP.”

The company also received US FDA approval for Fruzaqla (fruquintinib) to treat metastatic colorectal cancer (mCRC) patients who had received treatment previously.