GlaxoSmithKline
GlaxoSmithKline works on 46 rare diseases tracked on Trial Friend, including Adrenocortical Carcinoma, Amyotrophic Lateral Sclerosis, Autoimmune Hepatitis and 43 more, with 24 active clinical trials (12 recruiting) and 4 FDA-approved rare disease drugs.
GlaxoSmithKline (GSK) operates across multiple therapeutic areas including specialty medicines and rare diseases. The company divested its gene therapy portfolio to Orchard Therapeutics and maintains focus on rare disease treatments across its specialty care division.
Focus areas at GlaxoSmithKline
Within its broader pharmaceutical portfolio, GlaxoSmithKline has active clinical trial programs and drug development efforts across 46 rare diseases, including Adrenocortical Carcinoma, Amyotrophic Lateral Sclerosis, Autoimmune Hepatitis, Cholangiocarcinoma, Chronic Graft-versus-Host Disease, and 41 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 GlaxoSmithKline, 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.
GlaxoSmithKline is headquartered in London, United Kingdom, founded in 2000, publicly traded under the ticker symbol GSK. The company maintains a dedicated rare disease division alongside its broader therapeutic portfolio, working to bring innovative treatments to patients with conditions that have historically had limited or no treatment options.
GlaxoSmithKline Drug Pipeline
GlaxoSmithKline has 24 active clinical trials across 4 development stages, with 12 currently recruiting participants. Clinical trials advance through phases: Phase 1 tests safety in a small group, Phase 2 evaluates effectiveness and side effects, Phase 3 confirms benefit in a larger population, and Phase 4 monitors long-term safety after FDA approval.
Note: This pipeline counts the active and recruiting trials listed on this page, from ClinicalTrials.gov. For companies that work outside rare disease, we list only trials for rare diseases we track, so the company's full pipeline may be larger.
GlaxoSmithKline Clinical Trials (24)
Active and recruiting clinical trials sponsored by GlaxoSmithKline, sourced live from ClinicalTrials.gov. Each trial card shows the study phase, current recruitment status, conditions under investigation, study locations, eligibility criteria, and a direct link to the full ClinicalTrials.gov record. You can also download a one-page PDF summary to share with your doctor.
Note: Recruitment statuses on ClinicalTrials.gov may not immediately reflect recent FDA decisions, sponsor announcements, or enrollment changes. Always confirm a trial's current status directly with the study coordinator.
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GlaxoSmithKline FDA-Approved Drugs (4)
Medications developed or marketed by GlaxoSmithKline that have received U.S. Food and Drug Administration approval. Drug data is sourced from the openFDA database and includes brand names, generic names, approval dates, and matched rare disease indications. Where a drug treats a condition covered by Trial Friend, the disease name links directly to that disease page.
| Drug Name | Brand Name | Rare Diseases | Approval Date |
|---|---|---|---|
| BELIMUMAB B Lymphocyte Stimulator-specific Inhibitor [EPC] | BENLYSTA subcutaneous, intravenous | Mar 9, 2011 | |
| LAMOTRIGINE Anti-epileptic Agent [EPC] | LAMICTAL oral | Dec 27, 1994 | |
| MEPOLIZUMAB Interleukin-5 Antagonist [EPC] | Nucala subcutaneous | Nov 4, 2015 | |
| MOMELOTINIB | Ojjaara oral | Sep 15, 2023 |
GlaxoSmithKline Trial Locations
GlaxoSmithKline clinical trials are running at 697 sites in 32 countries. Click any country to drill down by state, city, and individual research facility. Proximity to a trial site is one of the most important factors in deciding whether to participate.
Rare Disease Focus Areas (46)
Diseases targeted by GlaxoSmithKline's clinical trial and drug development programs
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. These glands produce essential hormones including cor...
Amyotrophic lateral sclerosis is a progressive neurodegenerative disease destroying motor neurons in the brain and spinal cord. This causes progressive weakness and paralysis of voluntary muscles whil...
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. The immune ...
Cholangiocarcinoma is a rare and aggressive cancer that forms in the bile ducts, the thin tubes that carry digestive fluid (bile) from the liver to the small intestine. It can occur inside the liver (...
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. It can affect the...
Cystic fibrosis is an autosomal recessive genetic disorder affecting the CFTR protein, which normally regulates chloride transport. Defective CFTR causes thick, sticky secretions in the lungs and dige...
Patient Resources
Organizations and resources related to GlaxoSmithKline's rare disease focus areas
Frequently Asked Questions About GlaxoSmithKline
Common questions about GlaxoSmithKline's rare disease programs, clinical trials, and treatments.