Our Pipeline

Research and Development

Developing transformative therapies begins with our personal investment in the lives of people our medicines help wherever they are in their journey, from diagnosis through ongoing care. At Horizon, we aim to make a powerful difference for our patients, their caregivers and physicians through research of breakthrough medicines as well as exploration of new applications for existing medicines. Our long-term strategy focuses on building a pipeline of clinically meaningful development-stage medicines in order to bring new options forward for people living with rare and rheumatic diseases.


Rare Diseases

HZN-001 (teprotumumab)
  • OPTIC confirmatory Phase 3 trial
  • OPTIC-X Phase 3 extension trial

HZN-001 (teprotumumab) is a fully human monoclonal antibody, insulin-like growth factor-1 receptor (IGF-1R) inhibitor being studied in a confirmatory Phase 3 clinical trial for the treatment of active thyroid eye disease (TED). Teprotumumab has received Breakthrough Therapy, Orphan Drug and Fast Track designations from the U.S. Food and Drug Administration.

In patients with active TED, IGF-1R is overexpressed on orbital fibroblasts, resulting in local inflammation and orbital tissue expansion, which can lead to proptosis, or bulging of the eye.

For more information about TED, please visit our disease information page. For more information on teprotumumab, please visit clinicaltrials.gov.

Teprotumumab is an investigational medicine and its safety and efficacy have not been established.

RAVICTI® (glycerol phenylbutyrate) Oral Liquid
  • Label expansion: birth to 2 months

A supplemental New Drug Application (sNDA) has been submitted to the U.S. Food and Drug Administration (FDA) to expand the approved indication for RAVICTI (glycerol phenylbutyrate) Oral Liquid to include infants younger than two months of age living with urea cycle disorders (UCDs). The sNDA submission is based off an open-label clinical study evaluating the safety, efficacy and pharmacokinetics of RAVICTI in pediatric subjects under two years of age with UCDs. The FDA is expected to complete their review during the second half of 2018.

For more information about UCDs, please visit our disease information page. For more information on RAVICTI, please see RAVICTI.com.

(Submitted Feb. 2018 / U.S.)

RAVICTI is not approved for use in patients younger than two months of age by any Regulatory authority.

Rheumatic Diseases

  • Optimized uricase and optimized PEGylation for uncontrolled gout

HZN-003 is a genetically engineered uricase derivative, next generation gout biologic with optimized uricase and optimized PEGylation technology.

PASylated uricase technology
  • Optimized uricase and PASylation for uncontrolled gout

Collaboration agreement with XL-protein GmbH to identify clinical-stage product candidates that could be used to construct a next-generation gout biologic. If the collaboration agreement identifies clinical stage candidates, Horizon will have the right to license the candidates.

KRYSTEXXA® (pegloticase injection)
  • MIRROR trial: immunomodulation (Phase 4)
  • TRIPLE trial: tolerization and immunomodulation (IIT)
  • RECIPE trial: immunomodulation (IIT)
KRYSTEXXA Investigator-Initiated Trials:

For more information about gout, please visit our disease information page. For more information on KRYSTEXXA, please see KRYSTEXXA.com.

These are ongoing investigational trials and have not yet been approved by any Regulatory authority.

RAYOS® (prednisone) Delayed-Release Tablets
  • RIFLE trial: lupus

For more information on RAYOS, please see RAYOSRx.com.

This is an ongoing investigational trial and has not yet been approved by any Regulatory authority.

Clinical Trials

Clinical trials are an important part of the research and development process. People who participate in clinical trials play a vital role in helping physicians and investigators understand how an investigational medicine may be able to safely and effectively treat a specific disease or condition.

For a comprehensive list of current clinical trials involving Horizon medicines, please visit www.clinicaltrials.gov.