New GvHD treatment approved for use in Scotland
Hi,
It's Tom, the Head of Patient Information here at Anthony Nolan.
We've just heard that new GvHD treatment belumosudil (aka Rezurock) has been approved for use in Scotland, for patients with chronic GvHD.
Full details are below, from a press release by manufacturer Sanofi.
NICE is currently reviewing belumosudil for potential use in England and Wales. It is not expected to make a decision on this until September 2023 at the earliest.
If you experience GvHD after your stem cell transplant, we recommend that you continue to discuss your treatment options with your healthcare team.
You can also call our Helpline on 0303 303 0303 or email us: patientinfo@anthonynolan.org
All the best,
Tom
Press release:
Sanofi’s Rezurock® (belumosudil) accepted in Scotland for the treatment of chronic graft-versus-host disease.
• Rezurock® (belumosudil) will be accessible for people with chronic graft-versus host disease (cGVHD) in Scotland aged 12 years or older who have already received at least two prior lines of systemic therapy.
• Belumosudil will be available via the Scottish Medicines Consortium’s (SMC) ultraorphan pathway, designed to bring medicines to people with rare conditions where there is high unmet medical need. The Scottish Government Medicines Policy Branch will notify Health Boards when this medicine is available for prescribing within the ultra-orphan pathway.
• cGVHD is a leading cause of non-relapse death and morbidity in patients who undergo stem cell transplantation from a donor.
• People with cGVHD can experience serious symptoms including joint contractures with pain and movement loss, sight loss, severe lung disease, and life-threatening infections.
• Belumosudil is a first-in class medicine targeting the ROCK2 protein, inhibiting ROCK2 signalling pathways which play a major role in the body’s inflammatory responses.
Sanofi announced today that eligible people in Scotland living with chronic graft-versus-host disease (cGVHD) will be able to access belumosudil.
The Scottish Medicines Consortium (SMC) has accepted belumosudil, a once-daily oral tablet which has been shown to be effective in patients (aged 12 years or older) with cGVHD who have received multiple courses of treatment, for use through NHS Scotland.
cGVHD is a rare, often disabling and sometimes fatal condition. It affects people who have received stem cell transplantation with healthy blood cells given to them from a donor, to treat particular blood cancers or blood disorders.
The approval is based on the results of the ROCKstar trial, a Phase II, open-label randomised study which evaluated the efficacy of belumosudil in 132 patients with cGVHD.
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. Responses to belumosudil were seen in all organs, including the lungs, liver, and skin, which can be especially difficult to treat. Additionally, seven patients of the 126 in the modified intentto-treat population showed a complete response in all affected organs. This can be hard to achieve because of the irreversible organ changes which cGVHD can bring about. The main side effects of belumosudil were consistent with those expected for cGVHD patients
taking corticosteroids and other immunosuppressant medicines. The most common were fatigue (38%), diarrhoea (33%), nausea (31%), cough (28%), and upper respiratory tract infection (27%). Thirty-eight percent of patients had one or more serious side effects, the most common being pneumonia (8%).
Dr Robert Danby
Chief Medical and Scientific Officer, Anthony Nolan
"This is welcome news for stem cell transplant recipients in Scotland who struggle with this debilitating,
life-threatening condition. Through our helpline and forum we regularly hear about the devastating
effect chronic graft-versus-host disease can have on the physical and mental health of patients and their
families. There is a real need for new treatment options so we are very pleased that the SMC has agreed
to make belumosudil available.”
Dr Anne Parker
Consultant Haematologist at Queen Elizabeth University Hospital, NHS Greater
Glasgow and Clyde
“Donor stem cell transplant successfully treats thousands of people with blood cancers, but rejection of
the patient by the donor transplant cells – cGVHD – is a significant clinical challenge. It can cause
damage to any body system which can be life changing and potentially life threatening. Until now there
have been only a few treatment options available for these patients. I’m very pleased that Belumosudil
is now available and can potentially help patients who have failed to respond to current therapies.”
Jessamy Baird
Country Lead and Head of General Medicines, Sanofi UK & Ireland
“We are delighted with the SMC’s acceptance of belumosudil, which is an important step in our journey
to make this novel therapy available to all appropriate patients within Great Britain. There is a clear
need for new options for this difficult-to-treat patient population, who live with burdensome symptoms
that significantly impinge on their quality of life.”
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.6 This also includes further symptoms linked to fibrosis, such as fasciitis
(inflammation of connective tissues), cutaneous sclerosis (hardening of the skin), and
bronchiolitis obliterans (inflammation of the lung’s airways).
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. Some patients with cGVHD experience significant depression or anxiety symptoms.
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.
It is estimated that in the UK over 1,000 adults receive a stem cell transplant from a donor each
year and that the number of these types of transplants is increasing each year.
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.4 Belumosudil is the first and
only accepted therapy inhibiting Rho-associated coiled-coil kinase 2 (ROCK2). ROCK2
signalling pathways play a major role in the body’s inflammatory responses.
In 2019, belumosudil was granted orphan drug designation by the Medicines and Healthcare
products Regulatory Agency (MHRA) and European Commission. 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 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.
Belumosudil has been validated under the SMC ultra-orphan process due to cGVHD being
diagnosed in one in 50,000 or fewer people in Scotland, being a chronic or severely disabling
condition, and requiring highly specialised management.
Belumosudil is currently being assessed by the National Institute for Health and Care
Excellence (NICE) to evaluate its potential for use in England and Wales within the NHS.
