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Disease
Mastocytosis
Late-stage therapeutic developmentEmerging research
1
Publications
18
Clinical trials
2016
Latest publication
Latest activity
betaRecent clinical, regulatory, research and industry developments relating to this disease.
Regulatory2017-09-18EMA
What's happening now
An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.
Executive briefingUpdating summary…Momentum: Moderate
Key developments
- 4 clinical trials expected to report results, the earliest in Q1 2027.
Upcoming Milestones
- Q1 2027A Phase 1 Study of Avapritinib in Combination With Decitabine in Patients With Systemic Mastocytosis With an Associated Hematologic Neoplasm (SM-AHN)
- Q2 2027A 3-Part, Randomized, Double-Blind, Placebo-Controlled Phase 2 Study to Evaluate Safety and Efficacy of Avapritinib (BLU-285), a Selective KIT Mutation-Targeted Tyrosine Kinase Inhibitor, in Indolent and Smoldering Systemic Mastocytosis With Symptoms Inadequately Controlled With Standard Therapy
- Q4 2027An Open-label, Multicenter, Rollover Study in Patients Who Participated in an Avapritinib Clinical Study
- Q3 2032A Randomized, Double-Blind, Placebo-Controlled Phase 2/3 Study of BLU-263 in Indolent Systemic Mastocytosis
Major developments
Upcoming trial readoutHigh impact
Results expected Q3 20322026-06-25
Major trial resultsHigh impact
Results posted2026-04-24
Clinical MilestonesViewHide
Results posted
Results expected
- 2026-06-25A Randomized, Double-Blind, Placebo-Controlled Phase 2/3 Study of BLU-263 in Indolent Systemic MastocytosisResults expected Q3 2032
- 2026-04-01A Phase 1 Study of Avapritinib in Combination With Decitabine in Patients With Systemic Mastocytosis With an Associated Hematologic Neoplasm (SM-AHN)Results expected Q1 2027
- 2026-02-09An Open-label, Multicenter, Rollover Study in Patients Who Participated in an Avapritinib Clinical StudyResults expected Q4 2027
- 2025-09-23A 3-Part, Randomized, Double-Blind, Placebo-Controlled Phase 2 Study to Evaluate Safety and Efficacy of Avapritinib (BLU-285), a Selective KIT Mutation-Targeted Tyrosine Kinase Inhibitor, in Indolent and Smoldering Systemic Mastocytosis With Symptoms Inadequately Controlled With Standard TherapyResults expected Q2 2027
Activity timeline6
- 2026-06-25ClinicalA Randomized, Double-Blind, Placebo-Controlled Phase 2/3 Study of BLU-263 in Indolent Systemic MastocytosisResults expected Q3 2032
- 2026-04-24ClinicalAn Open-label, Single Arm, Phase 2 Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor, in Patients With Advanced Systemic MastocytosisResults posted
- 2026-04-01ClinicalA Phase 1 Study of Avapritinib in Combination With Decitabine in Patients With Systemic Mastocytosis With an Associated Hematologic Neoplasm (SM-AHN)Results expected Q1 2027
- 2026-02-09ClinicalAn Open-label, Multicenter, Rollover Study in Patients Who Participated in an Avapritinib Clinical StudyResults expected Q4 2027
- 2025-09-23ClinicalA 3-Part, Randomized, Double-Blind, Placebo-Controlled Phase 2 Study to Evaluate Safety and Efficacy of Avapritinib (BLU-285), a Selective KIT Mutation-Targeted Tyrosine Kinase Inhibitor, in Indolent and Smoldering Systemic Mastocytosis With Symptoms Inadequately Controlled With Standard TherapyResults expected Q2 2027
- 2025-09-19ClinicalA Phase 2 Open-Label, Multicenter Clinical Study of the Safety, Efficacy, Pharmacokinetic, and Pharmacodynamic Profiles of CGT9486 as a Single Agent in Patients With Advanced Systemic MastocytosisPrimary completion
Therapeutic landscape
Therapies with a regulatory footing for this condition, alongside the wider set of agents co-studied with it in the literature.
Approved & established therapies
Avapritinibapproved
Approval — Unresectable or metastatic gastrointestinal stromal tumour (GIST) AYVAKYT is indicated as… (2020)
Midostaurinapproved
Approval — Rydapt is indicated: in combination with standard daunorubicin and cytarabine induction… (2017)
Clinical trials
The current development programme across all trial phases.
Clinical programme
18
6
6
6
Late-stage studies
Recruiting
Recently completed
Regulatory timeline
Drug regulatory events matched to this condition by indication — EMA.
First approvals
2020emaApproval — Avapritinib· Unresectable or metastatic gastrointestinal stromal tumour (GIST) AYVAKYT is indicated as monotherapy for the treatment of adult patients with unresectable or metastatic gastrointestinal stromal tumours (GIST) harbouring the platelet-derived growth factor receptor alpha (PDGFRA) D842V mutation. Advanced systemic mastocytosis (AdvSM) AYVAKYT is indicated as monotherapy for the treatment of adult patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated haematological neoplasm (SM-AHN) or mast cell leukaemia (MCL), after at least one systemic therapy. Indolent systemic mastocytosis (ISM) AYVAKYT is indicated for the treatment of adult patients with indolent systemic mastocytosis (ISM) with moderate to severe symptoms inadequately controlled on symptomatic treatment (see section 5.1). source ↗
2017emaApproval — Midostaurin· Rydapt is indicated: in combination with standard daunorubicin and cytarabine induction and high dose cytarabine consolidation chemotherapy, and for patients in complete response followed by Rydapt single agent maintenance therapy, for adult patients with newly diagnosed acute myeloid leukaemia (AML) who are FLT3 mutation positive (see section 4.2); as monotherapy for the treatment of adult patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated haematological neoplasm (SM AHN), or mast cell leukaemia (MCL). source ↗
European Medicines Agency (CC BY 4.0). Events are matched to this condition by drug indication text — approvals/updates for drugs indicated for it, not disease-specific acts.
Research activity
Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.
Most influential
Leading journals1
- Blood1
Leading researchers8
- Arber DA1
- Bloomfield CD1
- Borowitz MJ1
- Cazzola M1
- Hasserjian R1
- Le Beau MM1
- Orazi A1
- Thiele J1
Affiliations (unnormalised)6
- Comprehensive Cancer Center1
- Institute of Pathology1
- John Hopkins Medical Institutions1
- Massachusetts General Hospital1
- Stanford University1
- University of Chicago1
Reference
Authoritative identity, definition & identifiers.
Defined in MeSH
A rare neoplastic disorder characterized by a clonal proliferation of MAST CELLS, associated with KIT-D816 mutations, and accompanied by aberrant mast cell activation. The abnormal increase of MAST CELLS may occur in only the skin (MASTOCYTOSIS, CUTANEOUS), in extracutaneous tissues involving multiple organs (MASTOCYTOSIS, SYSTEMIC), or in solid tumors (MASTOCYTOMA).
Identifiers
References & data sources
- Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
- Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
- Research activity — Europe PMC (EMBL-EBI) + OpenAlex-derived paper links
- Related entities are derived from literature co-mention (studied together) — associative, not causal.