Avapritinib has been approved by the National Institute for Health and Care Excellence (NICE) in the UK for the treatment of adults with advanced systemic mastocytosis (ASM). This approval offers a new treatment option for patients with this rare and aggressive blood cancer, where life expectancy can range from 6 months to 3 years.
The approval was based on clinical trial results showing that approximately one-third of patients treated with avapritinib experienced complete remission. Furthermore, almost all patients on the drug experienced significant improvements in the signs and symptoms of their cancer.
Mechanism of Action
Avapritinib is a once-daily oral medication designed to target and block the KIT mutation in bone marrow cells, which is a known driver of ASM. By inhibiting this mutation, the drug aims to halt the progression of the disease and alleviate its debilitating symptoms.
Clinical Impact
ASM can lead to a range of severe symptoms, including skin lesions, extreme diarrhea, bone pain, fatigue, organ damage, and life-threatening allergic reactions. These symptoms significantly impair patients' quality of life and pose substantial challenges for healthcare management.
Dr. Deepti Radia, a consultant haematologist at Guy's and St Thomas' NHS Foundation Trust, highlighted the transformative potential of avapritinib, stating, "Until now, the quality of life and the prognosis for patients with the aggressive form of the cancer was terrible. This is the first drug which has shown we can completely clear the body from this disease... The drug has reversed organ damage, improved quality of life and significantly improved survival for these patients."
Patient Experience
Susan Rudland, a 60-year-old patient diagnosed with ASM, experienced a dramatic turnaround after starting avapritinib. "It was like night and day," she said, describing how the drug cleared her disease and restored her quality of life.
Expert Commentary
Jessica Hobart, Chair and Trustee at The UK Mastocytosis Support Group, welcomed the approval, stating, "For the first time, we have a treatment that is not only able to significantly extend life but also one that is tolerable so people can live their lives more fully."
