Zolgensma (onasemnogene abeparvovec) is indicated for the treatment of spinal muscular atrophy (SMA). Thrombotic microangiopathy (TMA) has been reported in spinal muscular atrophy (SMA) patients treated with onasemnogene abeparvovec, particularly in the first weeks following the treatment. Patients should be monitored for signs, symptoms and laboratory findings suggestive of TMA.

Summary:

• Thrombotic microangiopathy (TMA) has been reported in spinal muscular atrophy (SMA) patients treated with onasemnogene abeparvovec, particularly in the first weeks following the treatment.
• TMA is an acute and life-threatening condition characterised by thrombocytopenia, haemolytic anaemia and acute kidney injury.
• Creatinine and complete blood count (including haemoglobin and platelet count) testing is now required before administration of onasemnogene abeparvovec in addition to the currently recommended baseline laboratory testing.
• Platelet counts should be closely monitored in the week following infusion and on a regular basis afterwards. In case of thrombocytopenia, further evaluation including diagnostic testing for haemolytic anaemia and renal dysfunction should be undertaken.
• If patients exhibit signs, symptoms or laboratory findings suggestive of TMA, direct specialist and multidisciplinary advice should be sought and TMA should be immediately managed as clinically indicated.
• Caregivers should be informed about signs and symptoms of TMA (e.g. bruises, seizures, oliguria) and should be advised to seek urgent medical care if such symptoms occur.