Novartis’ AveXis Prices Zolgensma Gene Therapy at $2+ Million, Offers Access Programs for US Payers and Families

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The company says it will work with payers to create payment options for its recently approved gene therapy, Zolgensma (onasemnogene abeparvovec-xioi), priced at $2.125 million.

AveXis, a Novartis company, announced on May 24, 2019 that it is establishing access programs for Zolgensma (onasemnogene abeparvovec-xioi), its gene therapy for treating pediatric spinal muscular atrophy (SMA) that was recently approved by FDA, which the company has priced at a 10-year cost of $2.125 million. AveXis says it is working closely with payers to offer pay-over-time options up to five years and outcomes-based agreements up to five years and is providing a patient program to support affordability and access.

AveXis has partnered with Accredo, a specialty pharmaceutical and service provider, to offer a pay-over-time option of up to five years to help ease possible short-term budget constraints, especially for states, small payers, and self-insured employers. In addition, CuraScript SD, a specialty distributor for medical providers, has been selected as the sole specialty distributor because of its experience in rare disease, including gene and cell therapies.

According to AveXis, the current 10-year cost of chronic therapy for pediatric SMA, administered over the patient's lifetime, can often exceed $4 million in the first 10 years of a young child's life. Furthermore, the therapy stops working if treatment is stopped. Based on this assessment, Zolgensma is expected to save costs in the healthcare system compared to the current chronic therapy used to treat and care for pediatric patients with SMA, the company reports.

The $2.125-million wholesale acquisition cost for Zolgensma entails:

  • 50% of the 10-year cost of current chronic SMA treatment (estimated at $4.1 million)

  • 50% below 10-year treatment costs for genetic pediatric ultra-rare diseases (estimated at $4.4 million to $5.7 million)

  • 50% below the Institute for Clinical and Economic Review (ICER) ultra-rare disease cost-effectiveness threshold; Zolgensma pricing places it at approximately $250,000 per quality-adjusted life-year (QALY)

ICER updates its benchmark

In a May 24, 2019 addendum published to its Final Evidence Report on treatments for SMA, the ICER updated its value-based price benchmark for Zolgensma, stating that: “Zolgensma is dramatically transforming the lives of families affected by this devastating disease, and given the new efficacy data for the presymptomatic population, the price announced [on May 24, 2019] falls within the upper bound of ICER’s value-based price benchmark range,” according to comments made by Steven D. Pearson, MD, MSc, president of ICER, in a press announcement.

Pearson went on to state that “[i]nsurers were going to cover Zolgensma no matter the price, and Novartis has spoken publicly about considering prices that approached $5 million. 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 updated value-based price benchmark assumes that Zolgensma’s effectiveness will be long lasting, and that the United States widely and rapidly adopts the recommendation to add screening for SMA to routine newborn screening. Once additional data are available, ICER may perform a New Evidence Update for both Zolgensma and nusinersen (Spinraza, Biogen).

According to its initially published Final Evidence Report on April 3, 2019, prior to FDA’s approval, ICER had shared its value-based price benchmark for Zolgensma, based on current data it had at that time, as the following: 

  • To reach commonly cited cost-effectiveness thresholds of $100,000 to $150,000 per QALY gained, a value-based price benchmark for Zolgensma would be between $1.1 million to $1.9 million per treatment.

  • 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.

Zolgensma is indicated for treating pediatric patients less than 2 years of age with SMA who have bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.

"Zolgensma is a historic advance for the treatment of SMA and a landmark one-time gene therapy. Our goal is to ensure broad patient access to this transformational medicine and to share value with the healthcare system," said Vas Narasimhan, CEO of Novartis, in a company press release. "We have used value-based pricing frameworks to price Zolgensma at around 50% less than multiple established benchmarks including the 10-year current cost of chronic SMA therapy.  In addition, the price of Zolgensma is expected to be within the range of traditional cost-effectiveness thresholds used by ICER when updated for its full labeled indications. 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."

Source: Novartis and Institute for Clinical and Economic Review