Indolent systemic mastocytosis

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At a Glance

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Regulatory Watchboard

6 events
Feb 2026An Observational Study in Participants With Indolent Systemic Mastocytosis (ISM)

Blueprint Medicines Corporation

TrialNOT YET RECRUITING
Dec 2025A Non-Interventional Study in Participants With Indolent Systemic Mastocytosis (ISM) in Germany

Blueprint Medicines Corporation

TrialRECRUITING
Jun 2022(Summit) A Study to Evaluate the Efficacy and Safety of CGT9486 Versus Placebo in Patients With Indolent or Smoldering Systemic Mastocytosis

Cogent Biosciences, Inc. — PHASE2

TrialACTIVE NOT RECRUITING
Nov 2021(HARBOR) Study to Evaluate Efficacy and Safety of BLU-263 Versus Placebo in Patients With Indolent Systemic Mastocytosis

Blueprint Medicines Corporation — PHASE2, PHASE3

TrialRECRUITING
Oct 2020Study of TL-895 in Subjects With Myelofibrosis or Indolent Systemic Mastocytosis

Telios Pharma, Inc. — PHASE2

TrialRECRUITING
Apr 2019(PIONEER) Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor, Versus Placebo in Patients With Indolent Systemic Mastocytosis

Blueprint Medicines Corporation — PHASE2

TrialACTIVE NOT RECRUITING

Data from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Financial Assistance

FINANCIAL LANDSCAPE SUMMARY

0

Total programs

No financial assistance programs currently listed for Indolent systemic mastocytosis.
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Approved Treatments

1 FDA-approved

Ayvakit

(avapritinib)Orphan drugstandard

Blueprint Medicines Corporation

Kinase Inhibitor [EPC]

12.1 Mechanism of Action Avapritinib is a tyrosine kinase inhibitor that targets KIT D816V, PDGFRA and PDGFRA D842 mutants as well as multiple KIT exo...

FDA label ↗

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Clinical Trial Landscape

5 active trials
3Phase 2
1Unknown
1PHASE2, PHASE3
5Total recruiting
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Recent News & Research

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Specialist Network

Top 6 by expertise

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