Deciphera Pharmaceuticals, LLC
Rare disease drugmaker · part of Ono Pharmaceutical · based in Waltham, United States · founded 2003 · deciphera.com
Deciphera Pharmaceuticals, LLC works on 6 rare diseases we track, including Chronic Graft-versus-Host Disease, Gastrointestinal Stromal Tumor and 4 more. It has 7 active clinical trials, and 4 are looking for volunteers now. It also has 2 FDA-approved rare disease drugs.
Deciphera Pharmaceuticals is a drug company based in Waltham, Massachusetts, founded in 2003 and part of Japan's Ono Pharmaceutical since 2024. It sells Qinlock (ripretinib) for advanced gastrointestinal stromal tumor and Romvimza (vimseltinib) for tenosynovial giant cell tumor. It is seeking US approval of tirabrutinib, discovered by Ono, for primary central nervous system lymphoma that has come back or not responded to treatment, with a decision due December 18, 2026.
Deciphera Pharmaceuticals, LLC pipeline
Each row lists the diseases Deciphera Pharmaceuticals, LLC is studying at that stage, from the ones closest to approval down to the earliest work.
Qinlock (ripretinib) · Romvimza (vimseltinib)
Polycythemia Vera recruiting · Primary Central Nervous System Lymphoma recruiting · Gastrointestinal Stromal Tumor 2 trials
Gastrointestinal Stromal Tumor 2 trials recruiting · Chronic Graft-versus-Host Disease recruiting
Deciphera Pharmaceuticals, LLC clinical trials
These are Deciphera Pharmaceuticals, LLC trials for the rare diseases we track, from ClinicalTrials.gov. Statuses there can lag behind company news, so confirm with the study team before you plan around one.
4 trials looking for volunteers now
INTREPID: A Study of Sapablursen Evaluating the Safety and Efficacy in Participants With Polycythemia Vera (PV)
Tests Sapablursen, Placebo
IGNITE: Study of Tirabrutinib vs Rituximab and Temozolomide for Relapsed/Refractory Primary Central Nervous System Lymphoma (PCNSL)
Tests Tirabrutinib, Rituximab, Temozolomide
For Relapsed/Refractory Primary Central Nervous System Lymphoma
A Master Protocol to Evaluate DCC-3009 in Gastrointestinal Stromal Tumor (GIST)
Tests DCC-3009
For Gastrointestinal Stromal Tumor (GIST)
A Study to Evaluate Vimseltinib in Adults With Active Chronic Graft-Versus-Host Disease (cGVHD)
Tests Vimseltinib
For Chronic Graft-Versus-Host Disease (cGVHD)
3 trials underway, not taking new volunteers
A Study of Inlexisertib (DCC-3116) in Combination With Anticancer Therapies in Participants With Advanced Malignancies
Tests Inlexisertib, Ripretinib
For GIST
A Study of Ripretinib vs Sunitinib in Advanced GIST Patients After Treatment With Imatinib
Tests Ripretinib, Sunitinib
For Gastrointestinal Stromal Tumors
A Study of Ripretinib vs Sunitinib in Patients With Advanced GIST With Specific KIT Exon Mutations Who Were Previously Treated With Imatinib
Tests Ripretinib, Sunitinib
For GIST
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FDA-approved drugs from Deciphera Pharmaceuticals, LLC
From the openFDA database. Tap a drug for side effects, dosing and help paying.
Qinlock ripretinib
For Gastrointestinal Stromal Tumor. Approved May 15, 2020. Taken by mouth.
Romvimza vimseltinib
Approved Feb 14, 2025. Taken by mouth.
More about Deciphera Pharmaceuticals, LLC
As a rare disease specialist, Deciphera Pharmaceuticals, LLC has active clinical trial programs and drug development efforts across 6 rare diseases, including Chronic Graft-versus-Host Disease, Gastrointestinal Stromal Tumor, Multiple System Atrophy, Polycythemia Vera, Primary Central Nervous System Lymphoma, and 1 additional rare conditions. These programs may span orphan drug designation, novel therapeutic mechanisms, and precision medicine approaches targeting the underlying causes of each disease.
The clinical trials section below shows active and recruiting rare disease studies sponsored by Deciphera Pharmaceuticals, LLC, sourced live from ClinicalTrials.gov. Each trial includes its current recruitment status, study phase (Phase 1 through Phase 4), conditions under investigation, and the number of active trial sites.
The FDA-approved drugs section lists treatments that have received U.S. Food and Drug Administration approval, with brand names, generic names, approval dates, and matched rare disease indications from the openFDA database.
Deciphera Pharmaceuticals, LLC is headquartered in Waltham, United States, founded in 2003, a subsidiary of Ono Pharmaceutical. The company dedicates its research and development resources exclusively to rare and orphan diseases, working to bring innovative treatments to patients with conditions that have historically had limited or no treatment options.
Where Deciphera Pharmaceuticals, LLC trials take place
The trials above run at 335 study sites in 27 countries. Tap a country to see its states, cities and hospitals.
Rare diseases Deciphera Pharmaceuticals, LLC works on
Each disease page lists every open trial, not only Deciphera Pharmaceuticals, LLC's, plus approved treatments.
Chronic Graft-versus-Host Disease
Chronic graft-versus-host disease is an immune-mediated complication after allogeneic stem cell or bone marrow transplant, in which donor immune cells attack the recipient's tissues.
How common: Affects 30-70% of patients who receive allogeneic hematopoietic stem cell transplant; approximately 14,000 new cases annually in the U.S
Gastrointestinal Stromal Tumor
Gastrointestinal stromal tumors (GISTs) are the most common mesenchymal tumors of the gastrointestinal tract, arising from the interstitial cells of Cajal or their precursors.
How common: About 6,000 new cases per year in the U.S. (American Cancer Society); true incidence may be higher as small GISTs are often incidentally discovered
Multiple system atrophy is a rare, progressive neurodegenerative disorder characterized by dysfunction of the autonomic nervous system and movement disorders.
How common: 2-5 per 100,000 people
Polycythemia Vera is a myeloproliferative neoplasm in which bone marrow produces too many red blood cells, leading to thick blood that carries oxygen less effectively and increases clot risk.
How common: About 44 to 57 per 100,000 people in the US, from 2 large health plans in 2008 to 2010 (Mehta et al., Leukemia and Lymphoma 2014); median age of diagnosis is 65 years
Primary Central Nervous System Lymphoma
Primary central nervous system lymphoma (PCNSL) is a fast-growing lymphoma that starts in the brain, spinal cord, eyes or the membranes around them and usually stays there.
How common: About 5 new cases per million people a year in the US (Ono Pharmaceutical and Deciphera); more than half of patients are 60 or older
Systemic Mastocytosis is a rare hematologic disorder characterized by accumulation and proliferation of abnormal mast cells in bone marrow, lymphoid tissues, and other organs.
How common: Approximately 1 case per 10,000 to 20,000 people; can occur at any age but more common in adults
Where to get support
Patient groups can explain a diagnosis, point you to specialists and tell you about trials before they open. Deciphera Pharmaceuticals, LLC's own site is deciphera.com, and our help paying for treatment page lists drugmaker programs and charity funds.
- National Marrow Donor Program (NMDP/Be The Match)Chronic Graft-versus-Host Disease
- BMT InfoNet - GVHDChronic Graft-versus-Host Disease
- The Life Raft Group (GIST Support)Gastrointestinal Stromal Tumor
- National Cancer Institute - GISTGastrointestinal Stromal Tumor
- Multiple System Atrophy CoalitionMultiple System Atrophy
- NIH GARD - Multiple System AtrophyMultiple System Atrophy
- National Organization for Rare Disorders (NORD)Polycythemia Vera
- Blood Cancer United (formerly The Leukemia & Lymphoma Society)Polycythemia Vera