This decision was based partly on the success of Zolgensma, originally developed by AveXis, as a gene therapy for spinal muscular atrophy (SMA) that is approved in the U.S., Japan, Europe, and Brazil as an intravenous infusion.
“Novartis sees tremendous potential in the future of gene therapy, and we’ve seen the impact gene therapy can have on so many lives,” Vas Narasimhan, CEO of Novartis, in a said press release. “With the creation of Novartis Gene Therapies, we will continue to advance our gene therapy pipeline for rare genetic diseases, to accelerate the delivery of transformative innovation in areas of high unmet need, and to reimagine medicine for patients all around the world.”
Zolgensma targets the underlying cause of SMA, delivering a working copy of the SMN1 gene — which is mutated in SMA — to motor neurons, the nerve cells that control muscle contraction.
This therapy, the world’s most costly treatment at $2.125 million for one-time use, relies on a genetically engineered virus, called adeno-associated virus (AAV) 9, to transport a working SMN1 transgene (so-called because its DNA comes from an external source) directly to motor neurons.
In the U.S., the treatment is approved for patients with all SMA types up to the age of 2, given in a single, hourlong intravenous infusion. In Europe, it is approved for those weighing up to 21 kg (about 46 lbs), with a clinical diagnosis of type 1 SMA or up to three copies of the SMN2 gene.
To date, 600 patients worldwide have been treated with Zolgensma, either through clinical trials, managed access programs, or its commercial availability, Novartis said in its release.
Regularly decisions are expected in Switzerland, Canada, Australia, Argentina, and South Korea this year or in early 2021, it added.
The company has placed a priority on AAV-delivered gene therapies to treat other disorders. The Novartis Gene Therapies research group is reported to be working on treatments for people with Rett syndrome, those with a genetic form of amyotrophic lateral sclerosis (ALS), and with Friedreich’s ataxia.
“Our patients and their families are the motivation for everything that we do, and under the banner of Novartis Gene Therapies, our dedicated team will continue to create a lifetime of possibilities to people suffering from rare genetic diseases,” said David Lennon, president of Novartis Gene Therapies who formerly led AveXis. “Becoming Novartis Gene Therapies symbolizes the importance of our gene therapy advances for the future of Novartis and our industry leadership at large.”
By unifying the Novartis and AveXis brands, Novartis intends to establish a global presence for Novartis Gene Therapies, Zolgensma, and for potential gene therapies to come.
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