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  • Matthias Reichert

Zolgensma® continues to set new pricing precedents for gene therapies in Europe and Japan

Updated: Jul 29, 2022

The new gene therapy Zolgensma® recently received the highest ever reimbursement price in Japan and the conditional EMA approval is paving the way for launch in Europe, with early access performance-based contracts agreed in Germany and a joint HTA assessment planned for Belgium, Ireland and the Netherlands.

Post by Matthias Reichert, Senior Analyst

Setting new precedents

Gene therapies typically promise a one-off treatment approach with potentially long-term therapeutic benefit. Zolgensma® (onasemnogene abeparvovec) is the most recent example - a gene therapy for the treatment of spinal muscular atrophy (SMA), a rare and devastating condition in infants causing motor neuron defects leading to severe difficulties with breathing, speaking and walking, which ultimately results in death by the age of 2 years in 90% of cases. Zolgensma® promises to halt disease progression by delivering a functional copy of the SMN1 gene, which is defective in these patients.

Historically, gene therapies have faced a rocky road with decision-makers due to intrinsic challenges in determining cost-benefit aspects and designing feasible reimbursement mechanisms. Despite these obstacles, Zolgensma® is now the most expensive drug on the US market with a list price of $2.12 million1, representing a trend that seemingly continues with its expansion to Europe and Japan.

Record reimbursed price set in Japan

In Japan, the Central Social Insurance Medical Council (Chuikyo) approved the addition of Zolgensma® to the National Health Insurance (NHI) reimbursement list.

  • On May 13th 2020, Zolgensma® was listed with a reimbursement price of Yen 167 million (US$1.55 million; €1.4 million) per single administration2, representing the highest list price ever approved for a drug in Japan, and about five times higher than the previous highest price of Yen 34 million, for the CAR T-cell therapy Kymriah®.

  • The pricing was set using the comparative price methodology, via “Similar Efficacy Comparison Method I”, using Spinraza® as the chosen comparator. Due to Zolgensma®’s one-time administration, Chuikyo used the cost of a full course of Spinraza® as the basis for price comparison, which equalled the cost of 11 vials of Spinraza®, with an additional 50% price premium for usefulness and a 10% sakigake price premium (fast track pathway awarded to innovative pharmaceutical products).

Conditional approval and access strategy in Europe

In Europe, Zolgensma® received a conditional approval on 19th May 2020 for patients with only the most severe form of SMA3, meaning that the EU label differs from its US and Japanese counterpart.

  • The EU label excludes SMA patients who could develop mild or late-onset SMA (type 3 and 4), in contrast to the US and Japanese label which includes all SMA types.

  • Children weighing up to 21kg are included in the EU label, which accounts for most patients under the age of 5 years; however, the indication in the US and Japan only allows treatment for children less than 2 years of age.

  • These differences in indication will lead to a different eligible patient population in the EU, with patients being more restricted in terms of SMA type, but with more lenient inclusion criteria in terms of patient age.

Novartis offers a ‘Day One’ access scheme to European countries3 with the aim of driving rapid access, leveraging a combination of annual staged payments, outcomes guarantee and services supporting patients and treatment centres. Under this scheme, the initial list price will be reimbursed, but importantly, Novartis guarantees rebates in line with the country-specific reimbursed prices which will be negotiated later in the HTA process.

  • If widely embraced, this EU wide access scheme could lead to more patients being treated at an earlier stage in the market access process, and would support the generation of real-world evidence (RWE).

  • At the same time, payers may be reluctant to accept the upfront impact on budget due to reimbursement at list price, and may be hesitant to allow the setting of a high pricing benchmark which could undermine later price negotiations.

Innovative contracting in Germany

In Germany, Zolgensma® is set to launch with a similarly unprecedented price tag of €1.945 million4 and specific agreements have been signed to ensure early access for patients via outcome-based reimbursement5,6.

  • The healthcare provider for industrial health insurance funds “GWQ ServicePlus”, as well as the association of private health insurers (PKV) have each agreed on separate early access performance-based reimbursement agreements for Zolgensma® (16th and 23rd April 2020, respectively), but details on the performance-based aspects of the agreements have not been disclosed.

  • This continues the recent trend of German payers engaging in outcome-based agreements for high-cost, single-use therapies - the GWQ entered a similar initial agreement for Kymriah®, where reimbursement depended on survival outcomes. German payers have historically been resistant to these types of deals but showed willingness to consider them on arrival of CAR T-cell therapies.

Joint HTA collaboration

Belgium, Ireland and the Netherlands are to conduct a joint HTA and pricing negotiations for Zolgensma® under the BeNeLuxA collaboration7, which was announced on the 19th May 2020.

  • This represents the third joint pricing negotiation carried out as part of the BeNeLuxA initiative. A previous joint pricing negotiation was conducted for Spinraza® in SMA by Belgium and the Netherlands, representing the only successful joint negotiation under BeNeLuxA to date.

Ongoing negotiations in France and the UK

Pricing and access negotiations with payers in other European countries are ongoing. In France, Zolgensma® has been dispensed in the context of a cohort ATU (Temporary Authorisation for Use) since 25th May 20208 and, in the UK, Zolgensma® is currently being assessed via the Highly Specialised Technology pathway. A “smart deal” agreement involving undisclosed discounts is planned9, similar to previous agreements which led to patients in the UK being the first to get access to Kymriah® outside of the US.

A key question is to what extent the lifelong cost of treating SMA (estimated to be between €2.5 to 4 million per child for the first 10 years alone) will be seen as a relevant cost offset for Zolgensma®’s high upfront cost in the UK. If successful, this would represent a significant milestone for Zolgensma® in its continuing market access success story, including within HTA systems characterised by a cost-effectiveness hurdle.

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1. Novartis secures US approval - and payers’ blessing – for $2m gene therapy Zolgensma. PMLive$2m_gene_therapy_zolgensma_1289221 (2019).

2. Zolgensma to Get Japan’s Highest List Price 167 Million Yen, but Peak Sales Put at Just 4 Billion Yen. PHARMA JAPAN (2020).

3. AveXis receives EC approval and activates “Day One” access program for Zolgensma®, the only gene therapy for spinal muscular atrophy (SMA). GlobeNewswire News Room (2020).

4. Ärzteblatt, D. Ä. G., Redaktion Deutsches. Spinale Muskelatrophie: Gentherapie Zolgensma ab 1. Juli auf dem... Deutsches Ärzteblatt (2020).

5. Vertrag sichert Versorgung schwerkranker Kinder mit innovativer Gentherapie. PKV (2020).

6. Sucker-Sket, K. Zolgensma: PKV und GWQ vereinbaren erfolgsorientierte Erstattung. (2020).

7. België, Nederland en Ierland gaan samen onderhandelen over terugbetaling van medicijn tegen zeldzame spierziekte. Maggie De Block (2020).

8. ATU de cohorte en cours - ANSM : Agence nationale de sécurité du médicament et des produits de santé.

9. Staines, R. Germany first in EU to get Novartis’ SMA gene therapy, costing almost 2m euros. (2020).


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