Novartis to acquire AveXis

Novartis appears set to become a first mover in gene therapy again with the agreed acquisition of AveXis Inc, a company with a late-stage gene replacement therapy for treating spinal muscular atrophy (SMA).

The therapy, AVXS-101, is expected to be filed with the US Food and Drug Administration for marketing approval in the second half of 2018. If authorised, it  would be used to replace a defective form of the SMN1 gene, which is a cause of the disease. The SMN1 gene encodes for a protein that motor neurons, which control muscle movement, need to survive.

In an early clinical study, AveXis was able to show that a single dose of the therapy administered to infants resulted in longer survival, superior achievement of motor milestones and better motor function than historical cohorts. The results were published in the New England Journal of Medicine in November 2017. AVXS-101 consists of an adeno-associated virus (AAV 9) which delivers a normal copy of the SMN1 gene to the brain via the bloodstream.

“The proposed acquisition of AveXis offers an extraordinary opportunity to transform the care of SMA,” Vas Narasimhan, the Novartis chief executive, said in a statement on 9 April.

Novartis has agreed to pay $8.7 billion in cash for the company, which will give it ownership of the lead product, gene therapy manufacturing capabilities for the adeno-associated virus and products for Rett Syndrome and a genetic form of amyotrophic lateral sclerosis caused by mutations in the superoxide dismutase 1 (SOD1) gene.

Novartis became the first mover in chimeric antigen receptor (CAR) T cell therapy in August 2017 when its gene therapy for leukaemia, Kymriah was approved by the FDA.

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