Novartis to ‘transform SMA’ care with $8.7bn AveXis acquisition

Novartis is acquiring US-based Nasdaq-listed clinical stage gene therapy company, AveXis, for $8.7 billion.

AveXis has several ongoing clinical studies for the treatment of SMA, an inherited neurodegenerative disease caused by a defect in a single gene, the survival motor neuron.

The lead AveXis gene therapy candidate, AVXS-101, has highly compelling clinical data in treating SMA Type 1, which is the number one genetic cause of death in infants, where 9 out of 10 infants do not live to their second birthday or are permanently ventilator dependent. It is estimated that one out of every 6,000-10,000 children born is affected by some form of SMA.

Novartis CEO Vas Narasimhan 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. We would gain with the team at AveXis another gene therapy platform, in addition to our CAR-T platform for cancer, to advance a growing pipeline of gene therapies across therapeutic areas.”

The US FDA has granted AVXS-101 Orphan Drug designation for the treatment of SMA as well as Breakthrough Therapy designation for SMA Type 1.

A BLA filing with the FDA for AVXS-101 is expected in the second half of 2018 and approval and launch in the US is expected in 2019. PRIME and Sakigake designations have been secured in Europe and Japan, respectively.

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.

In a clinical study, AVXS-101 showed life-saving efficacy, with all 15 infants treated event free at 20 months compared with an event-free survival rate of 8 percent in an historical cohort (NEJM, November 2017). AveXis will also present two-year data to the American Academy of Neurology on April 25, 2018.

The transaction is expected to close in mid-2018.

A message from the Editor:

Thank you for reading this story on our news site - please take a moment to read this important message:

As you know, our aim is to bring you, the reader, an editorially led news site but journalism costs money and we rely on advertising and digital revenues to help to support them.

With the Covid-19 lockdown having a major impact on our industry as a whole, the advertising revenues we normally receive, which helps us cover the cost of our journalists and this website, have been drastically affected.

As such we need your help. If you can support our news sites with a small donation of even £1, your generosity will help us weather the storm and continue in our quest to deliver quality journalism.

In the meantime may I wish you the very best.

- Advertisement -

Novavax selects PolyPeptide to manufacture critical component of COVID-19 vaccine

Novavax has selected CDMO PolyPeptide Group to manufacture a critical component of its novel coronavirus vaccine candidate, NVX-CoV2373. NVX-CoV2373 is a stable, prefusion protein made...

COVID-19 could hinder blockchain implementation in pharma supply chains

Even though blockchain stands out as a potential solution to improve pharma supply chains, COVID-19 may become a hindrance rather than a trigger in...

Emergent joins partnership for COVID-19 vaccine development & manufacturing

Emergent BioSolutions has been issued a task order under an existing contract with the Biomedical Advanced Research and Development Authority (BARDA) to deploy its...

Brilinta approved in US to reduce the risk of first heart attack or stroke

AstraZeneca’s Brilinta (ticagrelor) has been approved in the US to reduce the risk of a first heart attack or stroke in high-risk patients with...

GSK to manufacture 1bn doses of pandemic vaccine

GlaxoSmithKline (GSK) is aiming to manufacture one billion doses of its pandemic vaccine adjuvant system, in 2021, to support the development of multiple adjuvanted...

Related news

In brief: New head of R&D for Cellbox Solutions

Germany’s Cellbox Solutions has appointed Lonza Pharma and Biotech’s former Head of Biosolutions R&D as its new Head of R&D. Dr Herbert A. Mueller-Hartmann will...

Novavax selects PolyPeptide to manufacture critical component of COVID-19 vaccine

Novavax has selected CDMO PolyPeptide Group to manufacture a critical component of its novel coronavirus vaccine candidate, NVX-CoV2373. NVX-CoV2373 is a stable, prefusion protein made...

COVID-19 could hinder blockchain implementation in pharma supply chains

Even though blockchain stands out as a potential solution to improve pharma supply chains, COVID-19 may become a hindrance rather than a trigger in...

Emergent joins partnership for COVID-19 vaccine development & manufacturing

Emergent BioSolutions has been issued a task order under an existing contract with the Biomedical Advanced Research and Development Authority (BARDA) to deploy its...