MedCity News November 12, 2023
Takeda Pharmaceutical enzyme replacement drug Adzynma is the first FDA-approved therapy for a rare blood disorder affecting fewer than 1,000 Americans. Our regulatory recap also includes the much-anticipated approval of an Eli Lilly weight loss drug and a crackdown on what the FTC says are improperly listed pharma product patents.
A Takeda Pharmaceutical drug is now the first FDA-approved therapy for a rare, inherited enzyme deficiency that leads to blood clotting problems and a cascade of complications, some of them potentially fatal.
The disease, congenital thrombotic thrombocytopenic purpura (cTTP), stems from a mutation in the gene that codes for ADAMTS13, an enzyme that regulates blood clotting. Without enough of this enzyme, blood clots form in small blood vessels throughout the...
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