FDA, Gene Therapy, Regulatory

Novartis gains priority review for SMA gene therapy as investors grapple with sticker shock

An FDA approval for the world’s first $4 million gene therapy could arrive in as soon as five months.

Novartis says that regulators have opened up the expressway for AVXS-101, a one-time treatment for spinal muscular atrophy type 1 that’s supposed to cure the devastating neuromuscular disease.

Now dubbed Zolgensma, the gene therapy first entered Novartis’ portfolio this April through the $8.7 billion AveXis buyout and has since become one of CEO Vas Narasimhan’s favorite drugs in late-stage development. It is designed to cure SMA type 1 — restoring motor function and preventing further muscle degeneration — by inserting the gene responsible for the ailment.

David Lennon

The new priority review bolsters the ambitions that Novartis had stated for Zolgensma since day 1. Soon after it shocked the biotech world with data from a small study in which 15 infants with SMA were able to reach 20 months without the need for constant respiratory support, or dying, AveXis mapped a strategy to gain the fastest possible marketing approval for their therapy.

“The introduction of one-time, potentially curative therapies will require rethinking how our healthcare system manages diagnosis, treatment, care and associated costs for patients with genetic disease,” said AveXis president David Lennon in a statement.

And require rethinking it will.

Just days ago, Lennon told investors that the company believes AVXS-101 is cost-effective “in the range of $4-$5 million” using a slide that plotted several drugs’ 10-year cost against incremental quality-adjusted life year, or QALY. Both the US’ ICER and the UK’s NICE, the company points out, have backed drugs with 10-year costs of more than $4 million.

Also in Novartis’ and AveXis’ favor: Rivals at Biogen charge $750,000 for the first year of Spinraza, then $375,000 a year after that — for life. That’s $1.87 million over 4 years. And they don’t get much grief for it.

While the therapy waits its turn at the FDA, the commercial team will have plenty of pricing questions to grapple with.


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Biotech Investment Analyst
SV Health Investors Boston, MA
Research Scientist - Disease Biology
Recursion Pharmaceuticals Salt Lake City, UT
Senior Scientist - Predictive Pharmacology
Recursion Pharmaceuticals Salt Lake City, UT
Director, Translational Sciences
Cadent Therapeutics Cambridge, MA
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twoXAR Mountain View, CA

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