Common brand names

• Ayvakit

How it is administered

Avapritinib is taken by mouth as a film-coated tablet. It is available in different strengths: 25 mg, 50 mg, 100 mg, 200 mg, and 300 mg. The medication should be taken on an empty stomach, at least 1 hour before or 2 hours after a meal. The specific dose and duration depend on the condition being treated and your doctor's recommendations.

How it works

Avapritinib is a type of medicine called a tyrosine kinase inhibitor. It works by blocking certain proteins (kinases) in the body that are involved in the growth and survival of cancer cells. Specifically, avapritinib targets abnormal forms of the KIT and PDGFRA proteins, which can be mutated in some blood cancers and tumors. These mutations can cause the proteins to be constantly active, leading to uncontrolled cell growth.

By inhibiting these proteins, avapritinib helps slow down or stop the growth of cancer cells, particularly in conditions like advanced systemic mastocytosis (a blood disorder involving mast cells) and gastrointestinal stromal tumors (GIST) with specific mutations. This targeted approach helps address the underlying cause of these cancers, offering a more personalized treatment option.

Who should take it

Avapritinib is approved for adults with certain blood cancers and related conditions:

• Adults with advanced systemic mastocytosis (AdvSM), which includes aggressive systemic mastocytosis, systemic mastocytosis with an associated hematological neoplasm (SM-AHN), and mast cell leukemia (MCL).
• Adults with indolent systemic mastocytosis (ISM) who have moderate to severe symptoms.
• Adults with unresectable or metastatic gastrointestinal stromal tumor (GIST) that has a specific mutation in the PDGFRA gene (exon 18 mutation, including D842V).

Your doctor will determine if avapritinib is right for you based on your diagnosis, genetic test results, and overall health.

Who should not take it

There are no absolute contraindications listed for avapritinib, but it is not recommended for patients with advanced systemic mastocytosis or indolent systemic mastocytosis who have platelet counts less than 50 × 10⁹/L, as it can increase the risk of bleeding.

Patients with a history of intracranial hemorrhage, recent stroke, or those taking certain medications that interact with avapritinib (such as strong CYP3A inhibitors or inducers) should discuss these risks with their doctor. Pregnant women should avoid avapritinib due to the risk of harm to the unborn baby. Always inform your healthcare provider about all your medical conditions and medications before starting avapritinib.

Commonly used with

For indolent systemic mastocytosis, avapritinib is often used along with best supportive care, which may include antihistamines, leukotriene inhibitors, and other medications to manage symptoms. For GIST and AdvSM, it is typically used as a single agent, especially when specific mutations are present, but your doctor may recommend other supportive treatments as needed.

Commonly tested with

Avapritinib has been tested in combination with best supportive care therapies, especially for indolent systemic mastocytosis. Supportive care may include antihistamines, leukotriene inhibitors, and cromolyn sodium. For GIST and AdvSM, avapritinib is usually studied as a single agent, but patients may have received other treatments before starting avapritinib.