By Chris Wack

Takeda said Friday that the European Medicines Agency's Committee for Medicinal Products for Human Use has recommended the approval, under exceptional circumstances, of recombinant ADAMTS13 for the treatment of ADAMTS13 deficiency in children and adult patients with congenital thrombotic thrombocytopenic purpura.

The European Commission will consider the CHMP positive opinion when determining the potential marketing authorization for rADAMTS13 throughout the European Union.

If approved, Takeda said, rADAMTS13 will be the first and only enzyme replacement therapy in the EU for the treatment of cTTP.

Congenital thrombotic thrombocytopenic purpura is an ultra-rare, chronic blood clotting disorder caused by a deficiency in the ADAMTS13 enzyme.

The committee's positive opinion was supported by the totality of evidence including the interim analysis of efficacy, pharmacokinetic, safety and tolerability data from a Phase 3 trial.

Write to Chris Wack at

(END) Dow Jones Newswires

05-31-24 1026ET