WASHINGTON -- The FDA approved the for pediatric patients with spinal muscular atrophy (SMA) today.
The treatment, known as onasemnogene abeparvovec-xioi (Zolgensma), is approved for children under age 2 with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.
"Today's approval marks another milestone in the transformational power of gene and cell therapies to treat a wide range of diseases," Acting FDA Commissioner Ned Sharpless, MD, said in a statement. "With each new approval, we see this exciting area of science continue to move beyond the concept phase into reality."
"The potential for gene therapy products to change the lives of those patients who may have faced a terminal condition, or worse, death, provides hope for the future," he added. "The FDA will continue to support the progress in this field by helping to expedite the development of products for unmet medical needs through the use of review pathways designed to advance innovative, safe, and effective treatment options."
The therapy targets SMA by with a single, one-time intravenous infusion to deliver a fully functional copy into target motor neuron cells. Data from the ongoing phase III STRIVE trial showed children with SMA Type 1 experienced prolonged event-free survival, increases in motor function, and significant milestone achievements with the drug.
The treatment comes with a wholesale acquisition cost of $2.125 million, making it the in the world. The cost can be spread out over a 5-year installment plan, drugmaker Novartis said.
"Our goal is to ensure broad patient access to this transformational medicine and to share value with the healthcare system," Novartis CEO Vas Narasimhan . "We have used value based pricing frameworks to price Zolgensma at around 50% less than multiple established benchmarks including the 10-year current cost of chronic SMA therapy."
The most common side effects seen in the STRIVE trial were elevated liver enzymes and vomiting. The drug comes with a boxed warning that acute serious liver injury can occur.