Drug Updates: September

Following new draft guidance from the National Institute for Health and Care Excellence (NICE), midostaurin (Rydapt, Novartis) has been recommended for the treatment of advanced systemic mastocytosis in adults.

Mastocytosis is a rare condition caused by an excess number of specific blood cells called mast cells, which play a key role in the immune system by triggering inflammation in the body. Advanced systemic mastocytosis is a severe form of the disease where mast cells gather in body tissues, such as the skin, internal organs and bones.

There are three diverse subtypes of the condition:

There is no current standard treatment for advanced systemic mastocytosis. Instead, treatment is based on symptoms, and can include a variety of therapies.

New draft guidance from NICE means that around 170 patients with the condition will now be eligible for treatment with midostaurin, which will be the first targeted therapy for advanced systemic mastocytosis available on the NHS. The treatment is taken orally twice a day and works by blocking multiple enzymes involved in the condition.