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Most expensive gene therapy of Novartis was approved by the US FDA

Updated: Jun 3, 2019

Zolgensma (onasemnogene abeparvovec-xioi), the first gene therapy for the treatment of spinal muscular atrophy (SMA), the leading genetic cause of death in infants was approved by the United States Food and Drug Administration (FDA). ​


Survival motor neuron 1 mutation leads to spinal muscular atrophy (SMA). The gene codes for survival motor neuron (SMN) protein, the protein which is critical for the functioning and maintenance of motor neurons. Lack of the protein leads to the death of motor neurons causing death.

The product is indicated for the treatment of children of less than 2 years of age. The product is based on adeno-associated virus vector-based gene therapy. 

The approval of the drug is based on the clinical trial involving 36 pediatric patients with infantile SMA. The primary evidence was obtained from the ongoing trial involving 21 patients. Considering the history, the patients who received Zolgensma developed major developments in the motor milestones (head control and ability to sit). Side effects include elevated liver enzymes and vomiting. 

Earlier the product has received the breakthrough designation, fast-track designation and orphan designation from FDA.