A leading pharmaceutical giant recently reported two deaths due to liver failure in the phase 3 trial of an FDA-approved spinal muscular atrophy (SMA) drug known as onasemnogene abeparovec . The drug, which is currently administered intravenously, is currently under phase 3 trial to evaluate its safety and efficacy via an adeno-associated virus (AAV) vector-based intrathecal delivery method. SMA is an autosomal recessive neuromuscular disorder caused due to mutations in the SMN1 and SMN2 genes. Onasemnogene abeparovec The drug was approved by the FDA in May 2019 and was granted EU approval in 2020.
The drug has shown significant benefits among SMA patients when administered early. Corticosteroids are often prescribed along with this drug to control inflammation. The drug is only suitable for children under a certain weight. Hepatic dysfunction, including acute liver failure, is a…
