Kymriah® (tisagenlecleucel)▼ becomes first CAR-T therapy routinely reimbursed on the NHS across the UK in all its licensed indications, just one year after approval by the European Medicines Agency

Kymriah^® (tisagenlecleucel)▼ becomes first CAR-T therapy routinely reimbursed on the NHS across the UK in all its licensed indications, just one year after approval by the European Medicines Agency

Novartis’ tisagenlecleucel, chimeric antigen receptor T-cell (CAR-T) therapy is now approved in Scotland for all indications, following positive appraisal by the Scottish Medicines Consortium (SMC) for adults with diffuse large B-cell lymphoma (DLBCL)¹

Tisagenlecleucel is the only CAR-T therapy fully reimbursed for eligible UK patients on the NHS in all of its approved indications (DLBCL and paediatric B-cell acute lymphoblastic leukaemia [ALL])²

Milestone comes just one year since the treatment’s approval by the European Medicines Agency (EMA) and the announcement by NHS England that it would be the first CAR-T available on the NHS in England (for the ALL indication)^3,4

If left untreated, people with relapsed/refractory DLCBL have median life expectancy of around 6 months⁵

Camberley, September 09, 2019 – Today, Novartis announced that Kymriah^® (tisagenlecleucel) is now reimbursed for all eligible UK patients on the NHS, following a positive appraisal by Scottish Medicines Consortium for adult patients in Scotland with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) who did not respond to two or more previous systemic treatments.

Today’s announcement is made just one year on since the European Medicines Agency (EMA) granted tisagenlecleucel approval as one of the first CAR-T therapies available in the European Union.³Tisagenlecleucel is also approved across the UK for young adult patients up to 25 years of age with B-cell acute lymphoblastic leukaemia (ALL) that is refractory, in relapse post-transplant or in second or later relapse.^4,6

DLBCL is an aggressive form of blood cancer with a critical need for additional therapies. DLBCL is the most common form of non-Hodgkin lymphoma (NHL), accounting for approximately 30-40% of NHL cases in the UK.⁷ For patients who relapse or do not respond to existing therapies (relapsed/refractory patients), there are limited treatment options.⁸Survival rates are low for the majority of patients due to ineligibility for autologous stem cell transplant (ASCT) or because salvage chemotherapy or ASCT have failed.⁵

Ropinder Gill, CEO of Lymphoma Action said: “This is great news for people affected by lymphoma and means that another significant milestone has been passed in ensuring access to this kind of innovative treatment. It is particularly significant for people affected by diffuse large B-cell lymphoma as CAR-T cell therapy is now available to all suitable patients across the UK.”

The decision from the SMC was based on the results of the ongoing pivotal JULIET clinical trial for tisagenlecleucel, which most recently demonstrated median probabilities of 64% being relapse-free and 43% overall survival at 18 months in patients with relapsed/refractory (r/r) disease. The median duration of response has still not been reached.⁹

“Just over a year ago, CAR-T therapy wasn’t available in the UK or anywhere in Europe. Today’s positive appraisal by the Scottish Medicines Consortium represents a milestone as now all eligible patients across the UK can access Kymriah on the NHS regardless of indication or geography,” said Mari Scheiffele, Novartis Oncology General Manager, UK & Ireland.

“This achievement was only possible due to the close collaboration between industry, governments and hospitals. We are proud that we have been able to work in partnership to quickly deliver such a transformative therapy to patients in need.”

The UK approval milestones for tisagenlecleucel are set out below:

29 June 2018 – European’s Medicines Agency recommended the first marketing authorisations for chimeric antigen receptors (CAR) T-cells medicines in the European Union (EU) – one of which was for tisagenlecleucel for the treatment of paediatric and young adult patients (up to 25 years of age) with B-cell ALL that is refractory or in second or later relapse, and in adult patients with relapsed or refractory DLBCL after two or more lines of systemic therapy.³

16 November 2018 – NICE recommended tisagenlecleucel for use within the Cancer Drugs Fund when treating relapsed or refractory B-cell ALL in people aged up to 25 years, after reaching an agreement with Novartis.⁴

1 February 2019 – NICE recommended tisagenlecleucel for adults with relapsed or refractory DLBCL who did not respond to two or more previous treatments. At this time tisagenlecleucel was called a “game-changing treatment” by NHS representatives and was hailed by NICE as “a step forward for personalised medicine”.²

11 February 2019 – The Scottish Medicines Consortium accepted tisagenlecleucel for use within NHS Scotland for the treatment of paediatric and young adult patients up to 25 years of age with B-cell ALL that is refractory, in relapse post-transplant or in second or later relapse.⁶

9 September 2019 – The Scottish Medicines Consortium accepted tisagenlecleucel for use within NHS Scotland for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) after two or more lines of systemic therapy.

About CAR-T therapy

CAR-T (Chimeric Antigen Receptor – T cell) therapy is different from typical small molecule or biologic therapies because it is manufactured for each individual patient using their own cells. During the treatment process, T cells are drawn from a patient’s blood and reprogrammed in the laboratory to create T cells that are genetically coded to recognise and fight the patient’s cancer cells.

About JULIET

JULIET is the first multi-centre global registration study for tisagenlecleucel in adult patients with r/r DLBCL. JULIET, led by researchers at the University of Pennsylvania, is the largest registration study examining a CAR-T cell therapy in DLBCL, enrolling patients from 27 sites in 10 countries.

The latest analysis from the JULIET study of tisagenlecleucel in adult patients with r/r DLBCL provides a median follow-up of 19 months and showed prolonged durability of response in patients (n=99) who had previously been through multiple rounds of chemotherapy and unsuccessful stem cell transplants. The overall response rate (ORR) after a median of 19 months of follow-up was 54% (95% CI, 43% – 64%; CR, 40%; partial response [PR], 13%) among patients who were followed for at least 3 months or discontinued earlier.⁹

The median duration of response (mDOR) was not reached at the time of analysis indicating most responders were still experiencing a response at the time of analysis. The median relapse-free probability, which was 66% (95% CI, 51%-78%) at 6 months, remained consistent at 64% (95% CI, 48%-76%) at the 12-month and 18-month analyses.⁹ Median overall survival (OS) for all infused patients was 11.1 months (95% CI, 6.6 months-NE) and not reached (95% CI, 21 months-NE) for patients in CR.⁹

The safety profile observed in this latest analysis from JULIET continued to be consistent with previous reports and no deaths occurred due to causes other than disease progression in this longer-term follow up analysis.⁹

Novartis Leadership in Immuno-Oncology

Novartis is at the forefront of investigational immunocellular therapy as the first pharmaceutical company to initiate global CAR-T trials, and has significantly invested in CAR-T research and worked with pioneers in the field. Tisagenlecleucel is the cornerstone of this strategy. Active research programmes are underway targeting other haematologic malignancies and solid tumours, and include efforts focused on next generation CAR-Ts with evolved manufacturing schemes and gene edited cells.

About Novartis

Novartis is reimagining medicine to improve and extend people’s lives. As a leading global medicines company, we use innovative science and digital technologies to create transformative treatments in areas of great medical need. In our quest to find new medicines, we consistently rank among the world’s top companies investing in research and development. Novartis products reach more than 800 million people globally and we are finding innovative ways to expand access to our latest treatments. About 130 000 people of nearly 150 nationalities work at Novartis around the world. Find out more at www.novartis.com.

In the UK, we employ approximately 1,500 people to serve healthcare needs across the whole of the UK, as well as supporting the global operations of Novartis. Since 2014, Novartis has invested over £200 million in R&D and is a leading sponsor of clinical trials, in the UK. For more information, please visit www.novartis.co.uk.

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References

Novartis Pharmaceuticals UK Ltd. Summary of Product Characteristics. Kymriah cells dispersion for infusion. 2019. Available at: https://www.medicines.org.uk/emc/product/9456/smpc [Accessed September 2019]
National Institute for Health and Care Excellence (NICE). 2019 ‘NICE recommends another revolutionary CAR T-cell therapy for adults with lymphoma’. Available at: https://www.nice.org.uk/news/article/nice-recommends-another-revolutionary-car-t-cell-therapy-for-adults-with-lymphoma [Accessed September 2019]
European Medicines Agency. (2019). First two CAR-T cell medicines recommended for approval in the European Union – Medicines Agency. [online] Available at: https://www.ema.europa.eu/en/news/first-two-car-t-cell-medicines-recommended-approval-european-union [Accessed September 2019].
National Institute for Health and Care Excellence. 2018 ‘ NHS to fund Europe’s first CAR-T childhood cancer treatment’. 16 November 2018. Available at: https://www.nice.org.uk/news/article/nice-recommends-cutting-edge-therapy-for-young-people-with-blood-cancer [ Accessed September 2019]
Crump M, et al. Outcomes in refractory diffuse large B-cell lymphoma: results from the international SCHOLAR-1 study. Blood. 2017 130:1800-1808
Scottish Medicines Consortium (2019). Medicines advice tisagenlecleucel (Kymriah). Available at: https://www.scottishmedicines.org.uk/medicines-advice/tisagenlecleucel-kymriah-fullsubmission-smc2129/ [Accessed 14 September 2019]
Chaganti S, Illidge T, Barrington S, et al. Guidelines for the management of diffuse large B‐cell lymphoma. British Journal of Haematology. 2016;174:43-56.
Cancer Research UK. Non Hodgkin lymphoma. High grade NHL. Available at: http://www.cancerresearchuk.org/about-cancer/non-hodgkin-lymphoma/types/high-grade. [Accessed September 2019].
Schuster S., et al. Sustained Disease Control for Adult Patients with Relapsed or Refractory Diffuse Large B-Cell Lymphoma: An Updated Analysis of Juliet, a Global Pivotal Phase 2 Trial of Tisagenlecleucel. Blood. 2018 132:1684; doi: https://doi.org/10.1182/blood-2018-99-115252. [Accessed September 2019].