Medication leaflets

Active substance

Results for the substance mitapivat

5 results

About this active substance

Mitapivat is a medicine used in adults with haemolytic anaemia caused by pyruvate kinase deficiency, a rare inherited disorder in which red blood cells break down faster than normal. Treatment aims to raise haemoglobin and reduce symptoms related to anaemia.

The medicine activates pyruvate kinase in red blood cells, helping them produce energy and survive longer. It is not suitable for every type of anaemia. The diagnosis should be confirmed by tests recommended by a haematologist, including genetic or enzyme testing.

Mitapivat is taken by mouth, usually twice daily, with gradual dose adjustment based on response and tolerability. Do not stop it suddenly, because haemolysis and anaemia may worsen. If treatment has to be discontinued, your doctor will usually prescribe a stepwise dose reduction.

Side effects may include insomnia, nausea, back or joint pain, increased triglycerides, hot flushes, high blood pressure or hormone changes seen in blood tests. Tell your doctor about all other medicines you take, because some can change mitapivat levels and reduce effectiveness or increase side effects.