Zolgensma (onasemnogene abeparvovec) was approved by the US Food and Drug Administration (FDA) on Friday for the treatment of spinal muscular atrophy (SMA) for paediatric patients less than two years of age, with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.
The price tag sees the treatment become the most expensive treatment on the market, though the company has broken the cost down to annualised payments of $425,000 (€379,580) for five years.
The Swiss company had already prefaced the discussion regarding the pricing of gene therapy treatments when it acquired the ex-US rights to Luxturna (voretigene neparvovec), the first such treatment approved for genetic disease, which bears a price tag of close to $1m per patient.
At the time, a spokesperson for Novartis told us that bringing this therapy to market would require a ‘rethink’ of how healthcare systems manage costs.
The cost
With Zolgensma doubling the cost Luxturna, the same discussions will arise again.
However, The Institute for Clinical and Economic Review (ICER), an organisation that calculates the value of prescription drugs, released a statement that the price tag settled on by Novartis was within its value-based price benchmark and represented a ‘positive outcome.’
Steven Pearson, president of ICER, said, “Insurers were going to cover Zolgensma no matter the price, and Novartis has spoken publicly about considering prices that approached $5m.
“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.”
In its own calculations, ICER had projected a value-based price for the product of between $1.2m and $2.1m.
The value
How is Novartis able to justify its treatment becoming the most expensive on the market?
The incidence of SMA is approximately one in 10,000 live births and it is the leading cause of infant mortality. If left untreated, the most severe form of SMA, Type 1, sees 90% of patients either needing permanent ventilation or results in death.
At present, there is only one rival treatment approved, which is Biogen’s Spinraza (nusinersen), but this requires a lumbar puncture every four months, leading to research being carried out to discover a more patient-friendly delivery method.
Novartis pointed to the cost of the rival treatment reaching an estimated cost of $4m over the course of 10 years of chronic treatment.
An effective cure
So far, Novartis has five years of data to work from and posted clinical trial results where 10 patients remained alive after a mean time of 3.7 years post-treatment.
In a further study, seven of the patients remained on monotherapy alone, with the three additional patients switching to combination therapy based on ‘parental and physician discretion’ rather than loss of motor function.
Payment concessions
Similarly to when it introduced chimeric antigen receptor (CAR)-T treatment, Kymriah (tisagenlecleucel) – when it waived payment if the treatment did not work for patients – the company said that it is working with payers to provide ‘outcome-based agreements’ for up to five years after treatment.
Vas Narasimhan, CEO of Novartis, said, “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.”
According to the company, 15 payers are currently in advanced discussions and a certain number have already reached an agreement, in principle.
In terms of what Novartis will achieve in sales on the treatment, Evaluate estimates that it will see sales of $1.4bn by 2024, after paying $8.7bn to secure the deal last year.
With a full pipeline of further gene therapies, Novartis’ Zolgensma could become a benchmark that other companies in the area use to determine their own pricing.