Novartis, a Swiss pharmaceutical giant, has confirmed a substantial acquisition of AveXis, a US-based gene therapy company, for a total of $8.7 billion. This strategic move is set to enhance Novartis’s capabilities in gene therapy and neuroscience, particularly in the treatment of spinal muscular atrophy (SMA).

Strategic Acquisition in Pharma

Under the agreement, Novartis will pay $218 per share for AveXis. This acquisition is part of Novartis’s broader strategy to strengthen its leadership in neuroscience and follows closely on the heels of its $13 billion deal with GlaxoSmithKline to sell its stake in their consumer healthcare joint venture.

AveXis is currently at the forefront of multiple clinical trials focusing on SMA, a rare neuromuscular disorder caused by a defect in the survival motor neuron protein. The primary gene therapy candidate from AveXis, AVXS-101, has received orphan drug and breakthrough therapy designations from the US Food and Drug Administration (FDA) for the treatment of SMA Type 1, the most common form of the disease in infants and a leading genetic cause of infant mortality.

Enhancing Treatment for Spinal Muscular Atrophy

Commenting on the acquisition, 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 acquisition would also accelerate our strategy to pursue high-efficacy, first-in-class therapies and broaden our leadership in neuroscience.”

Paul Hudson, CEO of Novartis Pharmaceuticals, added: “Bringing AveXis on board would support both our ambition to be a leader in neurodegenerative diseases and our Neuroscience franchise priorities to strengthen our position in devastating pediatric neurological diseases such as SMA.”

Broadening the Research on Genetic Disorders

AVXS-101, if approved, promises to be a first-in-class one-time therapy that could substantially replace the defective SMN1 gene, addressing the genetic root of SMA. This gene therapy has demonstrated life-saving efficacy in clinical trials, significantly improving the survival rate compared to historical cohorts.

In addition to SMA, AveXis is also advancing treatments for other severe neurological diseases, including Rett Syndrome (RTT) and a genetic form of amyotrophic lateral sclerosis (ALS), caused by mutations in the superoxide dismutase 1 gene.

Sean Nolan, President and CEO of AveXis, remarked on the acquisition’s broader impact: “With worldwide reach and extensive resources, Novartis should expedite our shared vision of bringing gene therapy to these patient communities across the globe as quickly and safely as possible.”

The acquisition of AveXis by Novartis is a significant step in the pharmaceutical industry, marking a pivotal moment for the advancement of gene therapies for neurological disorders. It not only enhances Novartis’s pipeline but also consolidates its position as a leader in the rapidly evolving field of neuroscience and gene therapy.

Expected to close by mid-2018, the Novartis acquisition of AveXis is poised to set new standards in the treatment of rare and life-threatening neurological conditions, bringing hope to millions affected worldwide.

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