Zolgensma® gene therapy approved with innovative access program

The one-time gene therapy for spinal muscular atrophy will be available through pay-over-time and outcomes-based agreements.

May 29, 2019
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Zolgensma® (onasemnogene abeparvovec-xioi), the first and only gene therapy to be approved by FDA for the treatment of spinal muscular atrophy (SMA) will be available under a number of innovative access plans. Zolgensma, developed by AveXis (Basel, Switzerland), a Novartis company, will be available under pay-over-time options up to 5 years and outcomes-based agreements up to 5 years.

"Zolgensma is a historic advance for the treatment of SMA and a landmark one-time gene therapy. 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," said Vas Narasimhan, CEO of Novartis.

"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."

AveXs will provide a patient support program, called OneGene Program™ and an education program for commercial payers and state Medicaid programs to encourage rapid treatment, called Time in Neurons. More than 15 US payers are currently in discussion with AveXis over providing access to Zolgensma, AveXis hopes to leverage this approval to support others in Europe and Japan.

"We are at the forefront of an exciting time in healthcare when we'll be able to see major advancements in medical care with potentially curative gene therapies. While there are many questions that we as a healthcare system need to consider, what does not change is our work to ensure that these life-saving medications are affordable and available to the patients that need them," said Steve Miller, chief clinical officer, Cigna Corporation.

"We look forward to continuing the work we have started with AveXis to find unique solutions like installment payments and outcomes-based agreements for these life changing gene therapies."

The challenges of paying for ‘curative’ therapies in a system designed for managing chronic conditions was highlighted in the recent ISPOR 2019 annual meeting. Stakeholders, including payers, were urged to engage with patients to ensure appropriate access to innovative therapies.

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Freya Leask

Publisher, Future Science Group

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