Back To Back FDA Approval For Takeda Within Two Days, Secures Green Signal For Rare Blood Disorder Therapy

The FDA approved Takeda Pharmaceutical Co Ltd's TAK Adzynma, a recombinant (genetically engineered) protein product indicated for prophylactic (preventive) or on-demand enzyme replacement therapy (ERT) in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP).

cTTP is an ultra-rare, chronic blood clotting disorder caused by a deficiency in the ADAMTS13 enzyme.

The FDA approval of Adzynma was supported by data from the Phase 3 trial in cTTP and data from the continuation trial. 

The efficacy of Adzynma was evaluated in 46 patients who were randomized to receive six months of treatment with either Adzynma or plasma-based therapies (Period 1), then crossed over to the other treatment for six months (Period 2). 

The efficacy was demonstrated based on the incidence of thrombotic thrombocytopenic purpura (TTP) events and TTP manifestations and the incidence of the need for supplemental doses. 

No patient experienced an acute TTP event while receiving Adzynma prophylactic treatment (n=37), while there was one acute TTP event in a patient receiving plasma-based therapies (n=38).

The mean annualized event rate of thrombocytopenia manifestations was 2.0 (4.706) for patients receiving Adzynma (9/37 patients experienced a manifestation) compared to 4.44 (6.312) in patients receiving plasma-based therapies (19/38 patients experienced a manifestation).

Wednesday, the FDA approved Takeda's Fruzaqla (fruquintinib), an oral targeted therapy for adults with metastatic colorectal cancer (mCRC) who have been previously treated.

The approval was received under Priority Review more than 20 days before the scheduled PDUFA date of November 30, 2023.

Price Action: TAK shares are up 1.32% at $13.77 premarket on the last check Friday.