EMA Recommends Changing Marketing Authorization for Avapritinib for Systemic Mastocytosis

According to a recent article, the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) is now recommending changing the marketing authorization for avapritinib, which is used for advanced systemic mastocytosis.


Mastocytosis is a chronic condition that results when an unusually large number of mast cells accumulate in the skin, bone marrow, gastrointestinal tract, lymph nodes, liver, and spleen. A mast cell, also called a mastocyte or labrocyte, is a specific type of white blood cell that is part of the immune system, which helps protect the body against pathogens and aids the healing process. There are many different types of mastocytosis.

  1. Cutaneous mastocytosis affects the skin, causing small flat brownish spots that are made more obvious by scratching. For children under the age of three, blistering of these lesions can occur. Cutaneous mastocytosos can evolve into many different subtypes, including maculopapular, dodular, and diffuse cutaneous mastocytosis (DCM), which is the most severe form and usually presents itself during childhood. DCM causes the skin to thicken and become discolored, and other symptoms include blistering, low blood pressure, gastrointestinal bleeding, itchiness, diarrhea, and anaphylactic shock.
  2. Indolent systemic mastocytosis is generally associated with low mast cell burden, mediator-related symptoms, and skin involvement. People with this form have been known to have an enlarged liver or spleen, and the gastrointestinal tract may also be affected.
  3. Systemic mastocytosis, most commonly seen in adults, is associated with clonal hematological non-mast cell lineage disease and affects one in three cases of this type of mastocytosis. People generally do not have skin lesions with this form.
  4. Aggressive systemic mastocytosismanifests as an impaired or loss of liver, bone marrow, and/or bone function due to the overload of mast cells.
  5. Mast cell leukemiais an aggressive blood cancer that quickly affects the body’s organs. While rare, it is a fatal disease progression.


The symptoms of mastocytosis can range from mild to life-threatening. Some people experience an enlarged liver or spleen, at which time medical intervention becomes necessary. Depending on the type (listed above), heartburn, stomach aches, abdominal discomfort, and diarrhea may occur, and bones can become soft.

Avapritinib (AYVAKIT)

Avapritinib is a kinase inhibitor used to treat adults diagnosed with advanced systemic mastocytosis. It comes in the form of a tablet that patients will take once a day on an empty stomach.

Changing the Marketing Authorization

After two clinical trials that revealed positive results, the EMA’S Committee for Medicinal Products for Human Use (CHMP) has recommended expanding the current indication of avapritinib.

Now, the treatment will be indicated for use in patients with “aggressive systemic mastocytosis, systemic mastocytosis with an associated hematological neoplasm, or mast cell leukemia, after at least one systemic therapy.” Furthermore, CHMP has decided on two new strengths of the drug, 25 mg and 50 mg.

The updated summary and recommended use of avapritinib will now be published, and the European Commission will have to grant this change to marketing authorization. Typically this process will take up to 70 days.

Blueprint Medicines

Blueprint Medicines, based in the Netherlands, submitted the marketing authorization for avapritinib. They focus on creating life-altering therapies for patients with blood disorders and cancer.

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