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news.The UK’s National Institute for Health and Care Excellence (NICE) has recommended Novartis’ Rydapt (midostaurin) as the first treatment for rare blood disorder.
Midostaurin is recommended as the first targeted therapy for adult patients with rare blood disorder. Credit: Narupon Promvichai from Pixabay .
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The UK’s National Institute for Health and Care Excellence (NICE) has recommended Novartis’ Rydapt (midostaurin) as the first treatment for rare blood disorder.
It has been recommended as the first targeted therapy for adult patients with advanced systemic mastocytosis, a rare condition caused by an excess number of specific blood cells called mast cells.
This condition is a severe form of the disease where mast cells gather in body tissues including the skin, internal organs and bones.
It has three diverse subtypes that include aggressive systemic mastocytosis, systemic mastocytosis with associated hematological neoplasm, and mast cell leukaemia.
Midostaurin is taken orally twice a day and blocks multiple enzymes which are involved in the condition.
According to the new draft guidance published, nearly 170 patients will now be eligible for treatment with midostaurin.
NICE Centre for Health Technology Evaluation deputy chief executive and director Meindert Boysen said: “We are pleased to be able to recommend midostaurin as a treatment option for people with advanced systemic mastocytosis, despite the limitations in the clinical and comparative effectiveness evidence.
“The symptoms experienced by patients with this rare disease can be devastating and limiting. By recognising that patients with advanced systemic mastocytosis have a limited life expectancy without midostaurin, and by working closely with the company, we can support access to this innovative treatment.”
Data obtained from clinical trial suggests that midostaurin improves the overall survival of patients compared to many other comparator treatments.
It was also found that there is a substantial improvement in the quality of the patients’ life, though the evidence around its effectiveness is uncertain.
