Horizon Therapeutics
Horizon Therapeutics works on 6 rare diseases tracked on Trial Friend, including Chronic Granulomatous Disease, IgG4-Related Disease, Myasthenia Gravis and 3 more, with 1 active clinical trial and 5 FDA-approved rare disease drugs.
Horizon Therapeutics is now part of Amgen, which completed its $27.8 billion purchase of the company on October 6, 2023. Its medicines for rare autoimmune and severe inflammatory diseases are now in Amgen's portfolio. They include Tepezza (teprotumumab) for thyroid eye disease, Krystexxa (pegloticase) for chronic gout that has not responded to standard treatment, Ravicti (glycerol phenylbutyrate) and Buphenyl (sodium phenylbutyrate) for urea cycle disorders, Actimmune (interferon gamma-1b) for chronic granulomatous disease, and Procysbi (cysteamine bitartrate) for nephropathic cystinosis. Uplizna (inebilizumab) is approved for neuromyelitis optica spectrum disorder, and since 2025 for IgG4-related disease and generalized myasthenia gravis.
Horizon Therapeutics Drug Pipeline
Horizon Therapeutics has 1 active clinical trial across 1 development stage. 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.
Horizon Therapeutics Clinical Trials (1)
Active and recruiting clinical trials sponsored by Horizon Therapeutics, 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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Horizon Therapeutics FDA-Approved Drugs (5)
Medications developed or marketed by Horizon Therapeutics 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 |
|---|---|---|---|
| INTERFERON GAMMA-1B Interferon gamma [EPC] | ACTIMMUNE subcutaneous | Feb 25, 1999 | |
| SODIUM PHENYLBUTYRATE | BUPHENYL oral | Apr 30, 1996 | |
| PEGLOTICASE Uric Acid-specific Enzyme [EPC] | Krystexxa intravenous | — | Sep 14, 2010 |
| CYSTEAMINE BITARTRATE | PROCYSBI oral | Apr 30, 2013 | |
| GLYCEROL PHENYLBUTYRATE | Ravicti oral | Feb 1, 2013 |
Horizon Therapeutics Trial Locations
Horizon Therapeutics clinical trials are running at 2 sites in 1 country. 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 (6)
Diseases targeted by Horizon Therapeutics's clinical trial and drug development programs
Chronic granulomatous disease is a rare inherited immunodeficiency affecting the ability of neutrophils to produce reactive oxygen species. Recurrent severe infections and granulomas characterize the ...
IgG4-related disease is a chronic fibroinflammatory condition characterized by tumor-like swelling in affected organs, dense lymphoplasmacytic infiltration rich in IgG4-positive plasma cells, storifor...
Myasthenia gravis is a chronic autoimmune neuromuscular disorder causing muscle weakness and rapid fatigue. Antibodies attack acetylcholine receptors at the neuromuscular junction, preventing proper m...
Neuromyelitis Optica (NMO) is a rare inflammatory autoimmune disorder of the central nervous system characterized by recurrent attacks of optic neuritis (vision loss) and acute myelitis (spinal cord i...
Thyroid eye disease (TED) is a rare autoimmune condition in which the immune system attacks the muscles and fat behind the eyes, causing bulging eyes, double vision, pain, and in severe cases vision l...
Urea Cycle Disorders (UCDs) are a group of genetic conditions affecting the enzymes that convert toxic ammonia to urea for excretion. Deficiency of any of the eight enzymes in the urea cycle leads to ...
Patient Resources
Organizations and resources related to Horizon Therapeutics's rare disease focus areas
Frequently Asked Questions About Horizon Therapeutics
Common questions about Horizon Therapeutics's rare disease programs, clinical trials, and treatments.