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Disease

Mastocytosis, Systemic

Late-stage therapeutic developmentEmerging research
MeSHMastocytosis, Systemic
1
Publications
2
Clinical trials
2009
Latest publication
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this disease.

What's happening now

An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.

Executive briefingUpdating summary…Momentum: Low
Key developments
  • 1 clinical trial expected to report results, the earliest in Q4 2027.
Upcoming Milestones
Major developments
Upcoming trial readoutImportant
An Open-label, Multicenter, Rollover Study in Patients Who Participated in an Avapritinib Clinical Study
Results expected Q4 20272026-02-09
Clinical Milestones1View
Results expected 1
Activity timeline1

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

2 sponsors · 0 new · 0 completed in the last 12 months (net +0)

The current development programme across all trial phases.

Clinical programme
2
All trials
2
Active
1
Late-stage
0
Completed
Late-stage studies

An Open-label, Multicenter, Rollover Study in Patients Who Participated in an Avapritinib Clinical Study

Phase 4 · Recruiting · Blueprint Medicines Corporation

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

1 papers

Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.

Leading journals1
  • Blood1
Leading researchers8
  • Arber DA1
  • Bloomfield CD1
  • Borowitz MJ1
  • Brunning RD1
  • Harris NL1
  • Hellström-Lindberg E1
  • Le Beau MM1
  • Porwit A1
Affiliations (unnormalised)1
  • University of Chicago1

Reference

Authoritative identity, definition & identifiers.

Defined in MeSH

A group of disorders caused by the abnormal proliferation of MAST CELLS in a variety of extracutaneous tissues including bone marrow, liver, spleen, lymph nodes, and gastrointestinal tract. Systemic mastocytosis is commonly seen in adults. These diseases are categorized on the basis of clinical features, pathologic findings, and prognosis.

Identifiers
MeSHMastocytosis, Systemic
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.