Horizon Therapeutics
Horizon Therapeutics works on 5 rare diseases tracked on Trial Friend, including CDKL5 Deficiency Disorder, Chronic Granulomatous Disease, Myasthenia Gravis and 2 more, with 1 recruiting clinical trial and 7 FDA-approved rare disease drugs.
Horizon Therapeutics, acquired by Amgen in 2023 for $27.8 billion, develops medicines for rare autoimmune and severe inflammatory diseases. Key products include Tepezza (teprotumumab) for thyroid eye disease, Krystexxa (pegloticase) for chronic refractory gout, and Ravicti (glycerol phenylbutyrate) for urea cycle disorders. The company also markets Uplizna (inebilizumab) for neuromyelitis optica spectrum disorder.
Focus areas at Horizon Therapeutics
As a rare disease specialist, Horizon Therapeutics has active clinical trial programs and drug development efforts across 5 rare diseases, including CDKL5 Deficiency Disorder, Chronic Granulomatous Disease, Myasthenia Gravis, Neuromyelitis Optica, Urea Cycle Disorders. 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 all active and recruiting studies sponsored by Horizon Therapeutics, 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.
Horizon Therapeutics is headquartered in Dublin, Ireland, founded in 2008, a subsidiary of Amgen. 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.
Horizon Therapeutics Drug Pipeline
Horizon Therapeutics has 1 active clinical trial across 1 development stage, with 1 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 includes all of Horizon Therapeutics's active interventional trials, not only those targeting rare diseases. We show the full pipeline because a company's broader research activity, therapeutic expertise, and development infrastructure directly shape its ability to advance rare disease programs. A strong overall pipeline often signals deeper clinical operations, faster enrollment capabilities, and greater commitment to bringing new treatments to patients.
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.
Horizon Therapeutics FDA-Approved Drugs (7)
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 | May 13, 1996 | |
| PEGLOTICASE Uric Acid-specific Enzyme [EPC] | Krystexxa intravenous | — | Sep 14, 2010 |
| CYSTEAMINE BITARTRATE | PROCYSBI oral | — | Feb 14, 2020 |
| 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 (5)
Diseases targeted by Horizon Therapeutics's clinical trial and drug development programs
CDKL5 deficiency disorder is a severe genetic condition that causes early-onset seizures, usually beginning within the first few months of life, along with significant developmental delays and movemen...
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 ...
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...
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.