Novartis’ gene therapy takes additional step towards EU approval

By Ben Hargreaves

- Last updated on GMT

(Image: Getty/Vladimir_Timofeev)
(Image: Getty/Vladimir_Timofeev)

Related tags Novartis Zolgensma Gene therapy

Zolgensma has received a recommendation for approval despite running into problems recently, including manufacturing questions and data integrity issues.

Novartis’ treatment for spinal muscular atrophy (SMA) has been recommended to receive conditional approval by the European Medicines Agency (EMA), ahead of a final decision expected in approximately two months.

The potential approval of Zolgensma (onasemnogene abeparvovec) in the European Union (EU) would allow babies and young children with SMA to receive the one-time gene therapy treatment.

The EMA’s committee for advanced therapies reviewed the company’s application and the committee for human medicine (CHMP) subsequently provided a positive opinion.

Both committees based their decisions on preliminary results of one completed clinical trial and three supporting studies of patients at different stages of disease severity.

The data showed that 20 patients out of 22 were still alive and did not need permanent ventilatory support at 14 months of age, which compared favorably to the usual disease progression where only 25% of patients would still be alive at that interval.

The treatment previously received approval by the US Food and Drug Administration (FDA) in May 2019​, with the product becoming the most expensive treatment on the market – at $2.1m (€1.9m) per patient.

Anticipating potential pushback on pricing, Novartis reacted to the positive opinion by outlining the different payments schemes that will be made available to European countries looking to utilize the treatment.

The payment options include deferred payments, installment options, and outcomes-based rebates.

Moving beyond manufacturing questions

Despite receiving the positive opinion by the EMA, the journey for Novartis’ treatment post-US approval has not been entirely smooth.

The EMA’s assessment of the treatment has been slowed down over manufacturing queries​ regarding chemistry, manufacturing and controls. With the process in Japan also expected to be slowed by such questions, and a final decision there expected in the third quarter of 2020.

Further than this, Novartis disclosed that there were potential data inaccuracies​ in its biologics license application to the FDA. The revelations led to the departures of two senior executives of AveXis, the company that originally developed the treatment before being bought out by Novartis.

However, the product has become a commercial success in the US, achieving sales of $160m in third quarter results – above what had been expected for the product.

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