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Novartis' Zolgensma poised for European approval in latest round of CHMP decisions

Country : Europe, U.S.

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LONDON, 27 Mar (APM) - The European Medicines Agency's CHMP on Friday said Novartis' gene therapy Zolgensma should be approved in Europe during its latest round of recommendations.
The committee said in a statement that it recommends a conditional approval for Zolgensma (onasemnogene abeparvovec) in babies and young children with spinal muscular atrophy (SMA), a rare and often fatal genetic disease that causes muscle weakness and progressive loss of movement.
Because Zolgensma is an advanced therapy medicinal product (ATMP), it was assessed by the Committee for Advanced Therapies (CAT), the EMA's committee for cell- and gene-based medicines.
Conditional approval allows the agency to recommend a medicine for approval with less complete data than normally expected, which is quite often the case with newer ATMP products as mature data is not always available.
These approvals are given in cases where the benefit of a medicine's immediate availability to patients outweighs the risk inherent in the fact that not all the data are yet available.
There are currently limited treatment options for children with SMA in the EU and patients with severe SMA do not survive early childhood.
However, additional efficacy and safety data are being collected through three on-going studies, a long-term registry and further investigations on the product, including recommendations for future quality development.
As for all CHMP recommendations, final approval decisions will be in the hands of the European Commission.
Dave Lennon, president of Novartis' AveXis, said in a separate statement that the positive CHMP opinion for Zolgensma marks a "critical step closer to [European] approval and to bringing the only gene therapy for SMA to Europe, helping to address the devastating impact the disease has on patients and their families".

'Day One' access programme

While Lennon had insisted in November that EU-based payment models for Zolgensma will be approached with a "flexible mindset", Novartis confirmed on Friday that it will implement the 'Day One' access programme (APMHE 65116).
Zolgensma, which was first approved in the U.S. in May 2019, is the world's most expensive therapy priced at $2.1 million (APMHE 63120).
The 'Day One' programme will help healthcare systems manage the price, ensuring that the cost of patients treated before national pricing and reimbursement agreements are aligned with the value-based prices negotiated following clinical and economic assessments.
Novartis said: "Designed to work within existing pricing and reimbursement frameworks, yet recognising the novel nature of a one-time gene therapy for a devastating and progressive disease, the 'Day One' access programme offers ministries of health and reimbursement bodies (in countries without pre-existing early access pathways) a variety of flexible options that can be implemented immediately at time of approval."
The company confirmed it is in "advanced discussions with multiple European countries" to agree to the terms of the programme, components of which can be customised to suit the variety of healthcare systems in Europe.
Such components include:
  • Retroactive rebates ensuring early access costs are aligned with negotiated prices following local clinical and economic assessment processes
  • Deferred payments and instalment options allowing reimbursement bodies to manage budget impact during the early access phase
  • Outcomes-based rebates negotiated following clinical and economic assessments can be applied to patients treated during the early access period
  • Robust training for treating institutions on administration and follow-up care
  • Access to RESTORE, a global SMA registry of patients who have been diagnosed withSMA that draws upon existing country registries
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