Systemic mastocytosis

Last reviewed

🖨 Print for my doctorAdvocacy Hub →
ORPHA:2467C96.2
Who is this for?
Show terms as
4FDA treatments1Active trials24Specialists8Treatment centers2Financial resources

Where are you in your journey?

UniteRare data is sourced from FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, and NORD.
Report missing data

Overview

Systemic Mastocytosis is treated with 2 medications in our database, including Gastrocrom, Cromolyn Sodium Oral Solution (Concentrate). 2 of these have manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by Viatris Specialty LLC, Mylan. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Systemic Mastocytosis treatment below.

Inheritance

Sporadic

Usually appears on its own, not inherited from a parent

Age of Onset

Adult

Begins in adulthood (age 18 or older)

Orphanet ↗NORD ↗

FDA & Trial Timeline

1 event
Nov 2021(Apex) Bezuclastinib in Patients With Advanced Systemic Mastocytosis

Cogent Biosciences, Inc. — PHASE2

TrialRECRUITING

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

Treatments

4 available

Ayvakit

avapritinib· Blueprint Medicines Corporation
the treatment of adult patients with AdvSM. AdvSM includes patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated hematological neoplasm (SM-AHN), and mast cell

the treatment of adult patients with AdvSM. AdvSM includes patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated hematological neoplasm (SM-AHN), and mast cell leukemia (MCL)

Cimetidine

CIMETIDINE· Chartwell RX, LLC

The treatment of pathological hypersecretory conditions (i.e., Zollinger-Ellison Syndrome, systemic mastocytosis, multiple endocrine adenomas)

Gastrocrom

cromolyn sodium· Viatris Specialty LLC

* Patient Copay Amount: Not Publicly Available * Maximum Annual Benefit Limit: Not Publicly Available * Core Eligibility Restrictions: Not Publicly Available * RxBIN, PCN, and Group numbers: Not Publi

Cromolyn Sodium Oral Solution (Concentrate)

Cromolyn Sodium· Mylan

Detailed copay and financial assistance information is not publicly available for this medication at this time. Please consult your pharmacist or the manufacturer's official patient support program fo

Clinical Trials

1 recruitingView all trials with filters →
Phase 21 trial
(Apex) Bezuclastinib in Patients With Advanced Systemic Mastocytosis
Phase 2
Actively Recruiting
PI: Rachael Easton, MD, Ph.D. (Cogent Biosciences, Inc.) · Sites: Birmingham, Alabama; Phoenix, Arizona +40 more · Age: 1899 yrs

Specialists

24 foundView all specialists →
SM
Srdan Verstovsek, MD
HOUSTON, TX
Specialist
PI on 9 active trials
MP
Mariann Hedström, PhD
Specialist
PI on 1 active trial
JM
Joseph H Butterfield, MD
ROCHESTER, MN
Specialist
PI on 1 active trial
LM
Laurent FRENZEL, MD
Specialist
PI on 1 active trial1 Systemic mastocytosis publication
OP
Olivier Lortholary, MD, PhD
Specialist
PI on 5 active trials
RN
Ryotaro Nakamura
Duarte, California
Specialist

Rare Disease Specialist

PI on 6 active trials
RP
Rachael Easton, MD, PhD
TROY, MI
Specialist
PI on 3 active trials
DM
David Miklos
CHICAGO, IL
Specialist
PI on 2 active trials
JG
Jason Gotlib
PALO ALTO, CA
Specialist
PI on 2 active trials6 Systemic mastocytosis publications
CM
Cristina Livideanu, MD
Specialist
PI on 1 active trial
LP
Luis Escribano, MD, PhD
Specialist
PI on 2 active trials
AW
Ann Woolfrey
SEATTLE, WA
Specialist
PI on 5 active trials
MM
Melody C Carter, M.D.
Specialist
PI on 3 active trials
CM
Cristina Bulai Livideanu, MD
Specialist
PI on 3 active trials
AP
Anthony L Boral, MD, PhD
CAMBRIDGE, MA
Specialist
PI on 1 active trial
TM
Tom R. Fitch, M.D.
GLENDALE, AZ
Specialist
PI on 1 active trial
MM
Michael Heinrich, MD
Specialist
PI on 1 active trial
PM
Paul Armistead, MD
CHAPEL HILL, NC
Specialist
PI on 1 active trial

Treatment Centers

8 centers
🏥 NORD

Baylor College of Medicine Rare Disease Center

Baylor College of Medicine

📍 Houston, TX

🏥 NORD

Stanford Medicine Rare Disease Center

Stanford Medicine

📍 Stanford, CA

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program

National Institutes of Health

📍 Bethesda, MD

🔬 UDN

UCLA UDN Clinical Site

UCLA Health

📍 Los Angeles, CA

🔬 UDN

Baylor College of Medicine UDN Clinical Site

Baylor College of Medicine

📍 Houston, TX

🔬 UDN

Harvard/MGH UDN Clinical Site

Massachusetts General Hospital

📍 Boston, MA

🏥 NORD

Mayo Clinic Center for Individualized Medicine

Mayo Clinic

📍 Rochester, MN

👤 Mayo Clinic Center for Individualized Medicine

🏥 NORD

UCLA Rare Disease Day Program

UCLA Health

📍 Los Angeles, CA

Financial Resources

2 resources
Ayvakit(avapritinib)Blueprint Medicines Corporation

HealthWell Foundation — Systemic mastocytosis

HealthWell Foundation

Systemic mastocytosis

Verified 3d ago
Foundation Grant
foundation grant
Accepting applications

Travel Grants

No travel grants are currently matched to Systemic mastocytosis.

Search all travel grants →NORD Financial Assistance ↗

Community

Open Systemic mastocytosisForum →

No community posts yet. Be the first to share your experience with Systemic mastocytosis.

Start the conversation →

Latest news about Systemic mastocytosis

1 articles
NewsRSSApr 22, 2026
Test Your Knowledge About the History of SM
This article invites people to take a quiz about the history of SM (Systemic Mastocytosis) and compare their answers with other people who took the quiz. It's a
See all news about Systemic mastocytosis

Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

Rare disease caregiving can be isolating. Connect with counseling and peer support.

Family & Caregiver Grants

Financial assistance programs specifically for caregivers of rare disease patients.

Social Security Disability

Learn how rare disease patients may qualify for SSDI/SSI benefits.

Common questions about Systemic mastocytosis

What is Systemic mastocytosis?

Systemic Mastocytosis is treated with 2 medications in our database, including Gastrocrom, Cromolyn Sodium Oral Solution (Concentrate). 2 of these have manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by Viatris Specialty LLC, Mylan. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Systemic Mastocytosis treatment below.

How is Systemic mastocytosis inherited?

Systemic mastocytosis follows a sporadic inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Systemic mastocytosis typically begin?

Typical onset of Systemic mastocytosis is adult. Age of onset can vary across affected individuals.

Are there clinical trials for Systemic mastocytosis?

Yes — 1 recruiting clinical trial is currently listed for Systemic mastocytosis on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat Systemic mastocytosis?

24 specialists and care centers treating Systemic mastocytosis are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.

What treatment and support options exist for Systemic mastocytosis?

2 FDA-approved treatments and 2 patient support programs are currently tracked on UniteRare for Systemic mastocytosis. See the treatments and support programs sections for copay assistance, eligibility, and contact details.