Novartis Prices Zolgensma at $2.1 Million, Offers Innovative Access Programs

Henrietta Strickland
May 31, 2019

Called Zolgensma, it is a type of cutting-edge, one-time treatment called a "gene therapy" that treats the disease spinal muscular atrophy at its genetic root.

For the growing number of cell and gene therapies in the pipeline, Zolgensma will be a test of how commercially sustainable the field is.

The FDA green light came ahead of the weekend and means that Novartis will move straight ahead with a launch of Zolgensma (onasemnogene abeparvovec) for children with all types (1-3) of SMA aged less than two years, with 60 centres across the United States already geared up to deliver the therapy. An analysis by the Institute for Clinical and Economic Review, which was recently revised to include additional clinical trial data, determined the price falls within what it would consider cost-effective.

CEO Vas Narasimhan recently signaled that the therapy could be worth more than $5 million, considering the years of intensive care the drug can replace. "We believe by taking this responsible approach, we will help patients benefit from this transformative medical innovation and generate significant cost savings for the system over time". Novartis said it would allow insurance companies to spread out payments over five years, for an annual cost of about $425,000.

To reach the alternative thresholds of $100,000 to $150,000 per life year gained (LYG), a value-based price benchmark for Zolgensma would be between $1.2 million to $2.1 million.

Spinraza is priced at $750,000 for the first year, and $350,000 afterwards.

The Zolgensma treatment is an adeno-associated virus vector-based gene therapy that targets the cause of SMA. The sanction for the Zolgensma (onasemnogene abeparvovec-xioi) treatment has been given to AveXis, a biotechnology company specialising in gene therapies in the US. "It is a positive outcome for patients and the entire health system that Novartis instead chose to price Zolgensma at a level that more fairly aligns with the benefits for these children and their families".

The company is offering this "pay-over-time model", which it says fits with the U.S. healthcare system, and has already established outcomes-based agreements in principle with as many as 15 payers.

The team at AveXis, the company that first developed Zolgensma and was purchased by Novartis in 2018, designed two delivery routes for its therapy. It has established a partnership with finance lender Accredo to offer credit to healthcare payers via the five yearly instalment option.

AveXis Targeting to Make Therapy Available to Children's Across the Globe! Industry analysts expect the drug to hit peak sales of $1.8 billion to $2.6 billion per year, according to PMLiVE, a pharmaceutical industry news site. Furthermore, the therapy stops working if treatment is stopped.

Another unknown is just how many patients Novartis can treat over the longer term, after an initial surge of patients come forward in the US.

But products like Zolgensma, which introduce modified genes that are uniquely tailored for each patient, is extremely costly to produce - making it unclear if generic competition would flourish in the same way it has for less complicated treatments.

Other reports by Click Lancashire

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