Novartis signs $8.7bn gene therapy deal
Swiss drug maker Novartis is set to strengthen its gene therapy expertise with the $8.7 billion addition of Nasdaq-listed AveXis to its portfolio.
The clinical-stage gene therapy company has ongoing clinical studies for the treatment of SMA, an inherited neurodegenerative disease caused by a defect in a single gene, the survival motor neuron (SMN1).
AveXis’s lead candidate, AVXS-101, has “highly compelling clinical data” in treating SMA type 1, said Novartis in a press release, published yesterday, April 9. The one-time therapy addresses the root genetic cause of SMA by effectively replacing the defective gene.
According to the Swiss drug maker, SMA type 1 is the number one genetic cause of death in infants, where nine out of ten infants do not live to their second birthday or are permanently dependent on ventilators.
The US Food and Drug Administration has granted AVXS-101 an orphan drug designation for the treatment of SMA as well as a breakthrough therapy designation for SMA type 1.
A biologics licence application with the FDA is expected in the second half of 2018, with approval and launch in the US expected in 2019.
If approved, AVXS-101 would be a first-in-class one-time therapy that addresses the root genetic cause of SMA by effectively replacing the defective SMN1 gene.
Vas Narasimhan, CEO of Novartis, said: “The proposed acquisition of AveXis offers an extraordinary opportunity to transform the care of SMA. We believe AVXS-101 could create a lifetime of possibilities for the children and families impacted by this devastating condition.”
The deal means Novartis will gain another gene therapy platform, in addition to its chimeric antigen receptor T cell (CAR T) platform for cancer.
AveXis’s other pipeline products include therapy for Rett Syndrome and a genetic form of amyotrophic lateral sclerosis.
In March, LSIPR reported that GSK had revealed plans to buy Novartis out of their joint consumer healthcare business for $13 billion.
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