NICE publishes positive final draft guidance for Sanofi’s innovative medicine Rezurock® (belumosudil) for patients with chronic graft-versus-host disease (cGVHD) that will receive immediate funding through the Innovative Medicines Fund (IMF)
- Rezurock® (belumosudil) is the first treatment of its kind to be made available for people with cGVHD in England and Wales, licensed for patients with the disease aged 12 years or older who have received at least two prior lines of systemic therapy.
- Belumosudil is also one of the first medicines to utilise the Innovative Medicines Fund (IMF) providing interim funding and fast-tracking it to be immediately available to eligible patients in England.
- cGVHD is a leading cause of non-relapse death and morbidity in patients who undergo stem cell transplantation from a donor.1,2 cGVHD often strikes people just when they have had to deal with the trauma of a life-threatening condition, such as blood cancer or immune system and metabolic disorders.
Reading, Thursday, 21st December 2023. We are pleased that today NICE has announced that belumosudil, a daily tablet for the treatment of cGVHD, has received a positive final draft guidance from the National Institute for Health and Care Excellence (NICE). Following an agreement we have made with NHS England, belumosudil will immediately receive interim funding from the Innovative Medicines Fund (IMF) and will enable a fast-tracked supply to eligible patients in England several months prior to routine NHS England reimbursement.
The NICE guidance will also apply to Wales where belumosudil is expected to be made available within two months. This guidance marks a continuation of patient access to belumosudil across Great Britain following the recent evaluation by the Scottish Medicines Consortium (SMC) through the ultra-orphan pathway.
cGVHD is a rare, often disabling and sometimes fatal condition. It affects people who have received a stem cell transplantation from a healthy donor, to treat blood cancers or blood disorders. People with cGVHD can experience life-threatening infections, joint contractures with pain and movement loss, sight loss and end-stage lung disease.3
This news represents a positive step in delivering on our broader global commitment to build an immunology powerhouse that aspires to deliver new, innovative treatment options for patients. We believe we can transform the practice of medicine and lead the way in immunology.
The positive NICE appraisal is based on the results of the ROCKstar trial, a Phase II, non-controlled, open label randomised multicentre study which evaluated the efficacy of belumosudil in 132 patients with cGVHD aged 12 years or older. Patients enrolled had already received multiple courses of treatment.4 A 12-month analysis of the trial showed that three out of every four (75%) patients receiving belumosudil achieved a response within a year of treatment.4
Henny Braud, MBE
Chief Executive, Anthony Nolan
"This is a significant milestone in the management of chronic graft-versus-host disease. We are very pleased that a much-needed new treatment option will now be available to patients across Great Britain who have not responded to other therapies.”
Dr Ram Malladi
Consultant Haematologist in BMT & Cellular Therapy, Cambridge University Hospitals NHS Foundation Trust
“Chronic graft-versus-host disease or cGVHD is a chronic inflammatory condition that occurs in some patients who undergo donor stem cell transplant, when the donor cells attack the recipient’s organs or tissues. cGVHD can eventually cause scarring of almost any organ in the body, which can be life threatening, but also life changing – significantly affecting quality of life. Belumosudil is a welcome new treatment option for patients who have failed to respond to current therapies and otherwise would have few further options remaining.”
Jessamy Baird
Country Lead and Head of General Medicines, Sanofi UK & Ireland
“Belumosudil is one of the very first therapies approved via NHS England’s Innovative Medicines Fund (IMF) – a significant recognition of the value that belumosudil can offer to patients with cGVHD. We know from our research just how burdensome cGVHD can be – robbing patients and carers of the life they hoped they were getting back after their stem cell transplant. We are proud to be able to provide another treatment option for these patients in Great Britain.”
About chronic graft-versus-host disease
cGVHD causes inflammation (swelling) and fibrosis (scarring or hardening) which can affect almost any organ in the body. The skin, mouth, and eyes are the most common organs involved, with 86% of patients showing involvement in at least one of these organs at diagnosis.5 Other symptoms of cGVHD can include fasciitis (inflammation of connective tissues), cutaneous sclerosis (hardening of the skin), and bronchiolitis obliterans (inflammation of the lung’s airways).3
cGVHD affects patients who have already undergone intensive clinical interventions and have often had to deal with the trauma of being diagnosed with a life-threatening condition, including many blood cancers and certain blood, immune system and metabolic disorders. Many patients with cGVHD experience significant depression or anxiety symptoms.6,7
cGVHD is a leading cause of non-relapse death and morbidity in patients who undergo stem cell transplantation from a donor, often because of organ failure or infection.1,2 It is estimated that in the UK over 1,500 adults receive a stem cell transplant from a donor each year and that the number of these types of transplants is increasing each year.8
About the ROCKstar trial
In the trial, responses to belumosudil were seen in all organs, including the liver and lungs which can be especially difficult to treat, and organs with fibrotic manifestations such as the skin.4 Additionally, seven patients out of the 126 in the modified intent-to-treat population showed a complete response in all affected organs.4 This can be hard to achieve because of the irreversible organ changes which cGVHD can bring about, most notably in the eyes and the lungs.4
The main side effects of belumosudil were consistent with those expected for cGVHD patients taking corticosteroids and other immunosuppressant medicines. The most common (>25%) were fatigue (38%), diarrhoea (33%), nausea (31%), cough (28%), and upper respiratory tract infection (27%). Thirty-eight percent (38%) of patients had one or more serious side effects, the most common being pneumonia (7%).4
About belumosudil
Belumosudil is indicated for the treatment of patients aged 12 years and older with cGVHD who have received at least two prior lines of systemic therapy. Belumosudil is the first approved therapy inhibiting Rho-associated coiled-coil kinase 2 (ROCK2). ROCK2 signalling pathways play a major role in the body’s inflammatory and fibrotic responses.
In 2019, belumosudil was granted orphan drug designation by the Medicines and Healthcare products Regulatory Agency (MHRA) and European Commission.9,10 Orphan drug status is only given to medicines for rare, serious conditions where current treatment options are limited.
In 2022, belumosudil then received marketing authorisation in Great Britain and was further granted an Innovation Passport by the MHRA through the Innovative Licensing and Access Pathway (ILAP) and added to the list of Project Orbis approvals, designed to deliver faster patient access to innovative treatments which offer potential benefits over existing therapies.11
Following the final draft guidance from NICE in December 2023, belumosudil was approved for early funding via NHS England’s Innovative Medicines Fund (IMF). The primary function of the Innovative Medicines Fund is to operate as a managed access fund while evidential uncertainty is resolved in medicines that otherwise show significant clinical promise. In addition, the Innovative Medicines Fund (IMF) could provide a discretionary source of early funding for certain medicines that NICE can recommend for routine commissioning in the NHS from the point of marketing authorisation. Legislation requires the funding of NICE approved medicines within 90 days of final guidance being published. However, under these arrangements, funding could be brought forward by up to five months, starting at the point NICE issues a draft positive final guidance. 12
Belumosudil is currently licensed in several countries, including the USA and Canada.
The Summary of Product Characteristics (SmPC) for belumosudil can be found here.
About Sanofi
We are an innovative global healthcare company, driven by one purpose: we chase the miracles of science to improve people’s lives. Our team, across some 100 countries, is dedicated to transforming the practice of medicine by working to turn the impossible into the possible. We provide potentially life-changing treatment options and potentially life-saving vaccine protection to millions of people globally, while putting sustainability and social responsibility at the center of our ambitions.
Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY
Contacts
Gemma Lumber, Communications Lead, General Medicines, Sanofi UK & Ireland
+44 (0) 7713 313170 | gemma.lumber@sanofi.com
Clara Bentham, Head of Corporate Affairs, Sanofi UK & Ireland
+44 (0)7841 033734| clara.bentham@sanofi.com
References
1 Styczynski, J., Tridello, G., Koster, L. et al (2020). Death after hematopoietic stem cell transplantation: changes over calendar year time, infections and associated factors. Bone Marrow Transplant, 55 (1): 126-36.
2 DeFilipp, Z., Alousi, A., Pidala, J. et al (2021). Nonrelapse mortality among patients diagnosed with chronic GVHD: an updated analysis from the Chronic GVHD Consortium. Blood Advances, 5 (20):4278-84.
3 Filipovich, A., Weisdorf, D., Pavletic, S. et al (2005). National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus-host disease: I. Diagnosis and staging working group report. Biology of Blood and Bone Marrow Transplantation, 11(12): 945-55.
4 Cutler, C., Lee S., Arai S. et al (2021). Belumosudil for chronic Graft-Versus Host Disease (cGVHD) after 2 or more lines of therapy: The ROCKstar Study. Blood, 138: 2278-89.
5 Salhotra, A., Eiznhamer D., Hennegan, K. et al (2020). Presentation and management of chronic graft-versus-host disease in real-world clinical practice: A medical chart audit. EHA Virtual Congress, Abstract EP1436
6 El-Jawahri, A., Pidala, J., Khera, N. et al (2018). “Impact of Psychological Distress on Quality of Life, Functional Status, and Survival in Patients with Chronic Graft-versus-Host Disease.” Biology of Blood and Bone Marrow Transplantation, 24(11): 2285-92
7 Pidala, J., Kurland, B., Chai, X. et al (2011). “Patient-reported quality of life is associated with severity of chronic graft-versus-host disease as measured by NIH criteria: report on baseline data from the Chronic GVHD Consortium.” Blood, 117(17): 4651-7.
8 British Society of Blood and Marrow Transplantation and Cellular Therapy (2023) Transplant Activity by Disease. Available at: https://bsbmtct.org/activity/2021/. (Accessed December 2023)
9 Medicines & Healthcare products Regulatory Agency (2023) Orphan Register: https://www.gov.uk/government/publications/orphan-registered-medicinal-products/orphan-register#rezurock (Accessed December 2023)
10 The European Medicines Agency (2019): Orphan designation for the treatment of graft-versus-host disease: https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2205 (Accessed December 2023)
11 Medicines & Healthcare products Regulatory Agency (2023) Guidance: Project Orbis: https://www.gov.uk/guidance/guidance-on-project-orbis (Accessed December 2023)
12 National Institute for Health and Care Excellence. (2022) The Innovative Medicines Fund Principles: B1686-the-innovate-medicines-fund-principles-june-2022.pdf (england.nhs.uk). (Accessed December 2023)
