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GlaxoSmithKline

Drugmaker with rare disease work · based in London, United Kingdom · founded 2000 · ticker GSK · gsk.com

GlaxoSmithKline works on 19 rare diseases we track, including Adrenocortical Carcinoma, Autoimmune Hepatitis and 17 more. It has 23 active clinical trials, and 11 are looking for volunteers now. It also has 5 FDA-approved rare disease drugs.

GSK plc, called GlaxoSmithKline until May 2022, is a London drugmaker whose rare disease work centers on blood cancers and immune diseases.

GlaxoSmithKline pipeline

Each row lists the diseases GlaxoSmithKline is studying at that stage, from the ones closest to approval down to the earliest work.

Approved
Drugs the FDA has approved.
Phase 3
Large studies, usually the last step before a company asks the FDA for approval.

Multiple Myeloma 4 trials recruiting · Connective Tissue Diseases recruiting · Systemic Sclerosis recruiting · Immune Thrombocytopenia recruiting · Eosinophilic Granulomatosis with Polyangiitis

Phase 2
Mid-size studies that check whether a drug works and at what dose.

Multiple Myeloma 6 trials recruiting · Gastrointestinal Neoplasms recruiting · Adrenocortical Carcinoma recruiting · Myelofibrosis

Phase 1
First studies in people, mostly checking safety.

Myelofibrosis recruiting · Multiple Myeloma · Kidney Disease

Other studies
Studies with no phase listed, such as registries and natural history studies.

GlaxoSmithKline clinical trials

These are GlaxoSmithKline 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.

11 trials looking for volunteers now

Looking for volunteersPhase 3Recently updatedNCT07285239

Belantamab Mafodotin or Daratumumab With Bortezomib, Lenalidomide and Dexamethasone for Newly Diagnosed Multiple Myeloma

Tests Arm A: Belantamab Mafodotin, Arm B: Daratumumab Hyaluronidase, Bortezomib, Lenalidomide, Dexamethasone

For Multiple Myeloma

Eligible participants with newly diagnosed myeloma who are not considered eligible or refuse bone marrow transplant will be enrolled. Participants will be randomized to either belantamab mafodotin or daratumumab given in combination with bortezomib, lenalidomide and dexamethasone. Treatment will continue until disease progression, unacceptable side effects or withdrawal of consent. Belantamab mafodotin is a targeted cancer treatment that works against multiple myeloma cells. It combines a homing device (an antibody) with a powerful cell-killing drug (a toxin), delivering the toxin directly to cancer cells while largely sparing healthy cells. Minimal residual disease (MRD) testing will be done on bone marrow samples obtained standardly during your treatment. MRD shows whether a very small number of cancer cells can still be detected after treatment, even if standard lab tests shows no signs of cancer. The purpose of this study is to evaluate if belantamab mafodotin, bortezomib, lenalidomide and dexamethasone (BVRd) improves minimal residual disease (MRD) negative status and/or prolongs progression-free survival (PFS) compared with daratumumab, bortezomib, lenalidomide and dexamethasone (DVRd) in participants with newly diagnosed multiple myeloma.

Ages 18 Years - 79 Years3 locations
View full study
Looking for volunteersPhase 2Recently updatedNCT07689201

A Study of Velzatinib in Participants With Metastatic and/or Unresectable GIST After Imatinib, Sunitinib, Regorafenib, and Pimitespib

Tests Velzatinib

For Gastrointestinal Neoplasms

The goal of this study is to evaluate the study drug velzatinib (GSK6042981 / IDRX-42) to see whether velzatinib can shrink or control tumors and is safe and tolerated in Japan participants with gastrointestinal tumors (GIST) who progressed or were intolerant to standard therapies.

Ages 18 Years+3 locations
View full study
Looking for volunteersPhase 2Recently updatedNCT05789303

Study of Belantamab Mafodotin With Carfilzomib, Pomalidomide, and Dexamethasone in Relapsed Multiple Myeloma

Tests Belantamab mafodotin, Carfilzomib, Pomalidomide, Dexamethasone

For Multiple Myeloma and Relapse Multiple Myeloma

Doctors leading this study hope to learn if the combination of belantamab mafodotin, carfilzomib, pomalidomide, and dexamethasone is effective and safe when given to people who have multiple myeloma that has gotten worse and is not responding to standard drugs that are used for treating multiple myeloma, including chimeric antigen receptor T-cell therapy. Participation in this research will last about 6 -24 months, but it may be less or more depending on your response to treatment.

Ages 18 Years+2 locations
View full study
Looking for volunteersPhase 3Recently updatedNCT06716606

A Study to Investigate the Long-term Safety and Efficacy of Belimumab in Adults With Interstitial Lung Disease (ILD) Associated With Systemic Sclerosis (SSc) and Other Connective Tissue Diseases (CTD) (BLISSconneCTD-OLE)

Tests Belimumab

For Connective Tissue Diseases

This is an open label extension (OLE) study of an ongoing randomized controlled parent clinical studies 218224 (NCT05878717) and 221672 (NCT06572384) which aim to assess the efficacy and safety of belimumab on reducing the decline in lung function in participants with interstitial lung disease associated with diffuse cutaneous systemic sclerosis (dcSSc-ILD) and interstitial lung disease associated with other connective tissue diseases (CTD-ILD), respectively. The OLE study will describe how well tolerated belimumab will be long term, and whether it might continue to slow progression of lung function decline, slow overall disease progression and improve quality of life.

Ages 18 Years+41 locations
View full study
Looking for volunteersPhase 2/3Recently updatedNCT05878717

A Study of the Efficacy and Safety of Belimumab in Adults With Systemic Sclerosis Associated Interstitial Lung Disease

Tests Belimumab, Placebo

For Systemic Sclerosis Associated Interstitial Lung Disease and Scleroderma, Systemic

This study investigates the efficacy and safety of belimumab compared to placebo, in addition to standard therapy, for the treatment of participants with systemic sclerosis associated interstitial lung disease (SSc-ILD). The study will evaluate the effect of belimumab treatment on lung function as well as on extra-pulmonary disease manifestations, including skin thickening and general symptoms, such as fatigue, that impact quality of life (QoL).

Ages 18 Years+134 locations
View full study
Looking for volunteersRecently updatedNCT00315380

Longitudinal Study for Eosinophilic Granulomatosis With Polyangiitis

For Eosinophilic Granulomatosis With Polyangiitis and Churg-Strauss Syndrome

Eosinophilic granulomatosis with polyangiitis (EGPA) is a rare immune system disorder that causes asthma, an excessive number of eosinophils (a type of white blood cell) in the blood, and the inflammation of blood vessels, or vasculitis. In order to properly treat EGPA, it is critical that the level of disease activity can be determined over the course of the disease. The purpose of this study is to determine new biological markers, or biomarkers, that may be used to assess the severity of this disease in people with EGPA.

Ages not specified15 locations
View full study
Looking for volunteersPhase 1No updates in a whileNCT07104799

Momelotinib During and After HCT in Myelofibrosis

Tests Momelotinib

For Myelofibrosis and Hematopoietic Cell Transplantation (HCT)

This is a single-center, open-label, phase I study to determine the safety and tolerability of momelotinib in patients with myelofibrosis during and after hematopoietic cell transplantation (HCT).

Ages 18 Years+1 location
View full study
Looking for volunteersPhase 2No updates in a whileNCT05922501

Isatuximab, Bela Maf, Pom, and Dex in Relapsed/Refractory Multiple Myeloma

Tests Isatuximab, Belantamab mafodotin, Pomalidomide, Dexamethasone

For Relapsed Cancer, Refractory Multiple Myeloma and Multiple Myeloma

The main goal of this phase II study is to evaluate the overall response rate of isatuximab, belantamab mafodotin, pomalidomide, and dexamethasone in relapsed and refractory multiple myeloma. The study drugs provided for research purposes are isatuximab and belantamab mafodotin.

Ages 18 Years+2 locations
View full study
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FDA-approved drugs from GlaxoSmithKline

From the openFDA database. Tap a drug for side effects, dosing and help paying.

Benlysta belimumab

For Lupus Nephritis. Approved Mar 9, 2011.

Blenrep belantamab mafodotin

For Multiple Myeloma. Approved Oct 23, 2025. Taken by intravenous route.

Lamictal lamotrigine

For Lennox-Gastaut Syndrome. Approved Dec 27, 1994. Taken by mouth.

Nucala mepolizumab

For Eosinophilic Granulomatosis with Polyangiitis. Approved Nov 4, 2015. Taken by subcutaneous route.

Ojjaara momelotinib

For Myelofibrosis. Approved Sep 15, 2023. Taken by mouth.

More about GlaxoSmithKline

Its FDA-approved medicines with rare disease uses include Ojjaara (momelotinib) for intermediate or high-risk myelofibrosis in adults with anemia, Blenrep (belantamab mafodotin) with bortezomib and dexamethasone for multiple myeloma that has returned after at least 2 earlier lines of treatment, Nucala (mepolizumab) for adults with EGPA and Benlysta (belimumab) for lupus nephritis from age 5.

GSK's October 2026 pipeline list shows velzatinib in Phase 3 for gastrointestinal stromal tumors, depemokimab in Phase 3 for EGPA, belimumab in Phase 3 for lung disease linked to systemic sclerosis, belantamab mafodotin in Phase 2 for AL amyloidosis and momelotinib in Phase 2 for myelodysplastic syndrome. In 2018 GSK handed its rare disease gene therapies, including programs for Wiskott-Aldrich syndrome and metachromatic leukodystrophy, to Orchard Therapeutics.

Where GlaxoSmithKline trials take place

The trials above run at 768 study sites in 34 countries. Tap a country to see its states, cities and hospitals.

United States
94▼
China
69▼
Spain
67▼
Japan
58▼
Italy
54▼
France
53▼
Australia
40▼
South Korea
39▼
United Kingdom
37▼
Germany
33▼
Poland
28▼
Israel
23▼

Rare diseases GlaxoSmithKline works on

Each disease page lists every open trial, not only GlaxoSmithKline's, plus approved treatments.

Adrenocortical Carcinoma

Adrenocortical carcinoma is a rare, aggressive cancer of the adrenal cortex, the outer layer of the adrenal glands that sit on top of each kidney.

How common: About 1-2 per million people per year; approximately 600 new cases per year in the U.S

Autoimmune Hepatitis

Autoimmune Hepatitis (AIH) is a chronic liver disease characterized by persistent inflammation and progressive fibrosis of the liver due to loss of immune tolerance to hepatocyte antigens.

How common: About 16 per 100,000 people worldwide in a 2023 pooled analysis of 37 studies, with wide differences by region and a rise over time

Diffuse Intrinsic Pontine Glioma

Diffuse intrinsic pontine glioma (DIPG) is an aggressive childhood brain tumor that grows in the pons, the part of the brainstem controlling breathing, swallowing, and movement.

How common: About 300 children diagnosed in the U.S. each year, most often between ages 5 and 10

Eosinophilic Granulomatosis with Polyangiitis

Eosinophilic granulomatosis with polyangiitis (EGPA, formerly Churg-Strauss syndrome) is a systemic necrotizing vasculitis characterized by asthma, blood eosinophilia, and inflammation of small- and...

How common: 10 to 14 cases per million people; approximately 3,000 to 4,500 individuals in the U.S

Essential Thrombocythemia

Essential Thrombocythemia is a myeloproliferative neoplasm characterized by overproduction of platelets by bone marrow, resulting in very high platelet counts.

How common: About 1 new case per 100,000 people each year, with an estimated 30 to 60 per 100,000 living with it (NORD); average age at diagnosis is in the mid-fifties

Focal Epilepsy

Focal epilepsy is a neurological disorder characterized by recurrent seizures that originate in a specific area of the brain.

How common: Up to about 61% of people with epilepsy have focal seizures; about 2.9 million U.S. adults and 456,000 children have active epilepsy

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

Hodgkin Lymphoma

Hodgkin lymphoma is a cancer of the lymphatic system characterized by the presence of abnormal Reed-Sternberg cells.

How common: About 8,920 new cases expected in the U.S. in 2026 (American Cancer Society); approximately 2.6 per 100,000 people

Immune Thrombocytopenia

Immune thrombocytopenia (ITP) is a rare autoimmune disorder in which the immune system destroys platelets, the blood cells that stop bleeding, and slows their production.

How common: Estimated 9 to 20 per 100,000 people; children often have a temporary form that resolves within months

Lennox-Gastaut Syndrome

Lennox-Gastaut Syndrome is a severe childhood epilepsy characterized by multiple types of seizures that are often resistant to medication, intellectual disability, and characteristic EEG findings...

How common: About 1-4% of childhood epilepsy cases, with about 2 new cases per 100,000 children each year (NORD)

Light Chain Amyloidosis

Light Chain Amyloidosis is a rare, serious blood disorder caused by plasma cells producing misfolded immunoglobulin light chains that accumulate in tissues as amyloid fibrils, progressively damaging...

How common: Approximately 600-1,000 new cases per year in the United States; median age of diagnosis is 63 years

Lupus Nephritis

Lupus nephritis is kidney inflammation caused by systemic lupus erythematosus (SLE), in which the immune system attacks the kidneys' filters and, without treatment, can lead to kidney failure.

How common: About 21 per 100,000 people in a Minnesota population study in 2015 (Lupus Midwest Network, 2022)

Membranous Nephropathy

Membranous nephropathy is a rare autoimmune kidney disease where the body's own antibodies attack proteins in the kidney's filtering units (glomeruli).

How common: About 12 new cases per million people each year in the U.S.; roughly 40,000-50,000 Americans living with the disease

Multiple Myeloma

Multiple myeloma is a cancer of plasma cells, the white blood cells that make antibodies.

How common: About 36,000 new US diagnoses expected in 2026 and roughly 203,000 people living with it, which puts myeloma right at the edge of the US rare disease threshold; it is 1.7% of all new cancers (NCI SEER)

Myelodysplastic Syndromes

Myelodysplastic syndromes are a group of blood cancers in which the bone marrow fails to produce enough healthy blood cells.

How common: 10,000 to 15,000 new cases annually in the U.S.; estimated 60,000 to 170,000 Americans living with MDS

Myelofibrosis

Myelofibrosis is a myeloproliferative neoplasm in which bone marrow develops scarring (fibrosis), leading to anemia, enlarged spleen, and abnormal blood cell counts.

How common: Approximately 0.5-1.5 cases per 100,000 people; median age of diagnosis is 65 years

Polycystic Kidney Disease

Polycystic Kidney Disease is a genetic disorder characterized by the growth of thousands of cysts in the kidneys, gradually replacing normal kidney tissue and leading to kidney failure.

How common: Autosomal dominant PKD affects about 1 in 400 to 1,000 people worldwide and the recessive form about 1 in 20,000 children; about 500,000 people in the United States have PKD (NIDDK)

Polycythemia Vera

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

Systemic Sclerosis

Systemic sclerosis (scleroderma) is a rare autoimmune disease in which the immune system triggers scarring (fibrosis) of the skin and internal organs while also damaging small blood vessels.

How common: About 276 per million U.S. adults in a large Detroit-area study; about 19 new cases per million adults each year

Where to get support

Patient groups can explain a diagnosis, point you to specialists and tell you about trials before they open. GlaxoSmithKline's own site is gsk.com, and our help paying for treatment page lists drugmaker programs and charity funds.

Questions about GlaxoSmithKline