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Masitinib

Tyrosine kinase inhibitor (KIT, LYN, FYN) · also known as AB1010

Investigational KIT and LYN kinase inhibitor; studied in indolent systemic mastocytosis, with no current regulatory approval.

How it relates to mast cells
Mast cell disease
Types of studies cited
Randomised controlled trial
Randomised trials in
indolent systemic mastocytosis
Approved for
nothing — Investigational

What this relationship means: Studied in patients with a different mast cell disease, usually systemic mastocytosis. Those disorders have diagnostic criteria and disease biology that differ from MCAS, so a result there does not establish an MCAS outcome.

Mechanism of action

Masitinib inhibits KIT along with the LYN and FYN kinases that sit downstream of the high-affinity IgE receptor, giving it a proposed action on mast cell activation as well as on proliferation. Unlike avapritinib it is not selective for the D816V mutant, which is the pharmacologic reason its activity in D816V-positive disease has been questioned.

Masitinib is the only entry on this site with a trial registered in MCAS itself: NCT05449444, a Phase 2 study in severe mast cell activation syndrome. Its record has not been updated since February 2023, and ClinicalTrials.gov now carries it as status unknown — the registry’s own flag for a study whose sponsor has stopped updating past the expected completion date. The sponsor’s separate Phase 3 record in systemic mastocytosis, NCT04333108, is in the same state.

That is why this site takes trial statuses from the registry or the sponsor and from nowhere else. Secondary trackers tend to keep displaying the last confident-sounding status — “ongoing”, “recruiting” — with no indication that the underlying record has gone quiet. Two quiet records for the same drug is the situation those trackers describe least well, and it is the situation a reader most needs to know about.

Sources

  1. Masitinib for treatment of severely symptomatic indolent systemic mastocytosis: a randomised, placebo-controlled, phase 3 study Peer-reviewed study 2017 PMID 28069279 · link checked 21 Aug 2026
  2. An evaluation of masitinib for treating systemic mastocytosis Review article 2019 PMID 31381378 · link checked 20 Aug 2026
  3. Tyrosine Kinase Inhibitors for the Treatment of Mast Cell Diseases: Review and Update Review article 2025 PMID 40737063 · link checked 20 Aug 2026
  4. ClinicalTrials.gov record NCT05449444 — masitinib in severe mast cell activation syndrome Trial registry NCT05449444 · link checked 21 Aug 2026
  5. ClinicalTrials.gov record NCT04333108 — masitinib in indolent or smouldering systemic mastocytosis Trial registry NCT04333108 · link checked 21 Aug 2026