Therapy treats patients with chronic graft-versus-host disease who have already received other treatments.
Sanofi has announced that eligible people in Scotland living with chronic graft-versus-host disease (cGVHD) will have access to the company’s Rezurock therapy.
Indeed, the Scottish Medicines Consortium (SMC) has accepted Rezurock, also known as belumosudil – a once-daily oral tablet that is effective among patients aged 12 years and above – for use across Scotland.
These patients concerned are living with cGVHD and have already received multiple courses of treatment. cGVHD is a rare, disabling and sometimes fatal condition. It impacts people who have received stem cell transplantation with healthy blood cells given to them from a donor, to treat certain blood disorders or blood cancers.
The approval is based on the results emerging from the ROCKstar trial, a phase 2, open-label randomised study, which evaluated the efficacy of belumosudil among 132 patients with cGVHD.
A 12-month analysis of the research demonstrated that three out of every four patients receiving belumosudil achieved a response within a year of treatment. Responses to belumosudil were seen in all organs, notably those which are difficult to treat including the liver, skin and lungs.
Furthermore, seven patients in the 126 showed a complete response in all affected organs. This is typically hard to achieve because of irreversible organ changes caused by cGVHD.
The side effects triggered by belumosudil were consistent with those expected for cGVHD patients taking corticosteroids and other immunosuppressant therapies. The most common were fatigue, diarrhoea, nausea cough and upper respiratory tract infection. Meanwhile, 38% of patients had one or more serious side effects, the most prevalent being pneumonia.
Jessamy Baird, country lead and head of general medicines at Sanofi UK and Ireland, reflected: “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.”
Dr Robert Danby, chief medical and scientific officer at Anthony Nolan, explained: “This is welcome news for stem cell transplant recipients in Scotland who struggle with this debilitating, life-threatening condition.
He added: “Through our helpline and forum we regularly hear about the devastating effect cGVHD 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.”