The ADZYNMA clinical development programme included a pivotal, randomised, controlled Phase 3 study, the first of its kind in cTTP patients.

ADZYNMA is an enzyme replacement therapy (ERT) indicated for the treatment of ADAMTS13 deficiency in children and adult patients with congenital thrombotic thrombocytopenic purpura (cTTP).

ADZYNMA is an enzyme replacement therapy (ERT) indicated for the treatment of ADAMTS13 deficiency in children and adult patients with congenital thrombotic thrombocytopenic purpura (cTTP).

ADZYNMA prophylaxis Primary endpoint: ADZYNMA prophylaxis for a median of ~ 13.3 months was associated with zero acute TTP events & fewer sub-acute TTP events compared to treatment with plasma-based therapies.

Patients with cTTP experience disease- and treatment-related complications and burdens that have a negative impact on their HRQoL.

Prophylaxis is a recommended approach for some patients with congenital thrombotic thrombocytopenic purpura (cTTP)

The clinical presentation of cTTP ranges from life-threatening, acute, overt TTP events to milder TTP manifestations, including thrombocytopenia, haemolytic anaemia, abdominal pain, headaches and neurological symptoms.