Takeda Pharma Gets US FDA Approval for Rare Blood Disorder Therapy

Reuters

9:29 JST, November 10, 2023

Nov 9 (Reuters) – The U.S. Food and Drug Administration on Thursday approved Takeda Pharmaceutical’s 4502.T therapy to treat a rare genetic blood disorder in adult and pediatric patients.

Takeda’s Adzynma becomes the first treatment available to patients with congenital thrombotic thrombocytopenic purpura (TTP).

Congenital TTP is a life-threatening, rare genetic disorder with debilitating chronic symptoms. It leads to abnormal clotting in the small blood vessels throughout the body and is associated with anemia and low platelet levels.

The FDA said that most common side effects associated with the therapy include headache, diarrhea, migraine, abdominal pain, nausea, upper respiratory tract infection, dizziness and vomiting.

The health regulator’s decision is based on the late-stage study of the drug, which showed it was able to reduce incidence of Thrombocytopenia, the most common symptom of the disease associated with low level of platelets, by 60% compared to standard of care.

Takeda expects Adzynma will be commercially available in December 2023, and pricing will be disclosed at that time, the president of its US business unit, Julie Kim, told Reuters.

The treatment, chemically known as TAK-755, helps in replacing the deficient protein ADAMTS13 that helps in blood clotting at the site of injury.

Currently plasma treatments, spleen surgeries and off-label use of Roche’s ROG.S blood cancer drug Rituxan are used to slow down or treat the condition.