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Disease

Systemic mastocytosis

Late-stage therapeutic development
Also known as SMCD - systemic mast cell disease, systemic tissue Mast cell disease, systemic tissue mast cell disease, Aggressive systemic mastocytosis+4 more

SMCD - systemic mast cell disease, systemic tissue Mast cell disease, systemic tissue mast cell disease, Aggressive systemic mastocytosis, SM, systemic mast cell disease, systemic mastocytosis with associated hemotologic non-mast cell lineage disease (SM-AHNMD), Mast cell disease.

17
Clinical trials
4
Associated genes
1
Related proteins

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
  • 3 clinical trials expected to report results, the earliest in Q1 2027.
Upcoming Milestones
Clinical Milestones5View
Results posted 1
Results expected 3
Recently completed 1
Activity timeline5

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

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

The current development programme across all trial phases.

Clinical programme
17
All trials
5
Active
4
Late-stage
6
Completed
Recently completed

An 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 Mastocytosis

Phase 2 · Completed · Blueprint Medicines Corporation

Interest of Denosumab Treatment in Osteoporosis Associated to Systemic Mastocytosis

Phase 3 · Completed · Assistance Publique - Hôpitaux de Paris

A Study of Brentuximab Vedotin (SGN-35) in CD30-Positive Systemic Mastocytosis With or Without an Associated Hematological Clonal Non-Mast Cell Lineage Disease (AHNMD)

Phase 2 · Completed · Jason Robert Gotlib

Randomized, Double-blind, Placebo-controlled, Crossover Design, Efficacy and Safety Study With PA101 in Patients With Indolent Systemic Mastocytosis

Phase 2 · Completed · Patara Pharma

Randomized, Placebo-controlled, Phase 3 Study to Compare Efficacy and Safety of Masitinib at 6 mg/kg/Day to Placebo in Treatment of Patients With Smouldering Systemic, Indolent Systemic or Cutaneous Mastocytosis With Handicap

Phase 3 · Completed · AB Science

Imatinib Mesylate Therapy in Systemic Mastocytosis Patients Lacking KIT Mutations

Phase 4 · Completed · Hospital Virgen de la Salud

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.

Associated genes

4 matches

Genes associated with this disease in the canonical knowledge graph (Open Targets evidence). Number shows the association score.

KIT0.63OR51Q10.18LAX10.02SMR3B0.00

Disease biology

1 match

Proteins whose encoding gene is associated with this disease, reached through the canonical gene→disease graph. Number shows the gene's association score.

Mast/stem cell growth factor receptor Kitvia KIT0.63

Reference

Authoritative identity, definition & identifiers.

Synonyms

SMCD - systemic mast cell disease, systemic tissue Mast cell disease, systemic tissue mast cell disease, Aggressive systemic mastocytosis, SM, systemic mast cell disease, systemic mastocytosis with associated hemotologic non-mast cell lineage disease (SM-AHNMD), Mast cell disease

References & data sources
  • Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
  • Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
  • Related entities are derived from literature co-mention (studied together) — associative, not causal.