Zolgensma has grown into a blockbuster, or a medicine that brings in over $1 billion in yearly revenue. Novartis expects sales to grow further with a tweaked version that's delivered into the spine.
Despite the success of Zolgensma, a driver of the $8.7 billion deal was the belief that AveXis could produce a series of gene therapies for several diseases, not just SMA. That hasn't happened; Narasimhan said one reason has to do with an unexpected finding related to how Zolgensma is delivered inside the body.
Zolgensma is ferried into cells by a type of engineered, hollowed-out virus called AAV9. Narasimhan said further research has found that AAV9 is"very good for neuromuscular diseases, but it doesn't penetrate the brain very well."that aimed to treat Rett syndrome, a rare genetic brain disorder. "I would say it's a solid success with Zolgensma," Narasimhan said, adding that his expectations for a tweaked version of the drug that's still being tested in older people with SMA would justify the $8.7 billion acquisition price."In order for it to be a spectacular success, we have to translate this now into a larger clinical-stage pipeline and ultimately launches in gene therapy, and there I'd say the jury is still out.
He described making each therapy as"a more nuanced puzzle you have to solve," particularly in choosing the right delivery tool to get into the desired cells.During its presentation at the conference, Novartis said it had 18 gene therapies in development, ranging from the earliest stages of development to early-to-mid-stage clinical studies.to acquire Gyroscope Therapeutics, a gene-therapy startup testing a treatment for a common cause of blindness.