Our Pipeline

We are relentless in pursuing breakthroughs that boldly change the lives of the communities we serve.

Our pipeline is built on purpose, and our scientific courage drives us to tackle many marginalized, overlooked or forgotten diseases.

Pipeline

Rare Diseases

MEDICINE / CANDIDATE

PHASE

Teprotumumab

  • OPTIC trial: Phase 3 (complete)
  • OPTIC-X trial: Phase 3 extension

Learn More

PHASE 3

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PROCYSBI® (cysteamine bitartrate)

  • NDA submitted for delayed-release oral granules in packets

PHASE 3b / 4

In July, the U.S. Food and Drug Administration (FDA) accepted the New Drug Application (NDA) for PROCYSBI Delayed-Release Oral Granules in Packets. If approved, this new dosage form would provide another administration option for patients, in addition to the currently available PROCYSBI delayed-release capsules, which are FDA-approved for children one year of age and older and adults living with nephropathic cystinosis.

For more information about cystinosis, please visit our disease information page. For more information on PROCYSBI, please see PROCYSBI.com.

Rheumatic Diseases

MEDICINE / CANDIDATE

PHASE

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HZN-003

  • Optimized uricase and optimized PEGylation for uncontrolled gout

PRE-CLINICAL

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

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HZN-007

  • Optimized uricase and PASylation for uncontrolled gout

PRE-CLINICAL

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.

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KRYSTEXXA® (pegloticase)

  • MIRROR immunomodulation study: KRYSTEXXA + methotrexate

PHASE 3b / 4

Methotrexate to Increase Response Rates in Patients with Uncontrolled GOut Receiving KRYSTEXXA (MIRROR Randomized Controlled Trial [RCT]): A randomized, double-blind, placebo-controlled efficacy and safety study for methotrexate co-administered with KRYSTEXXA to evaluate the impact of methotrexate weekly for one month prior to dosing with KRYSTEXXA and then throughout Month 6 of treatment with KRYSTEXXA. The MIRROR RCT follows the MIRROR open-label evaluation that was initiated in fall 2018 and is now fully enrolled.

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

Recent Clinical Milestones

MEDICINE

STATUS

RAVICTI® (glycerol phenylbutyrate) Oral Liquid

  • Label expansion: birth to 2 months

Discovery Collaborations

MEDICINE / CANDIDATE

PHASE

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Gout Discovery Collaboration

  • Exploration of novel approaches to treating gout

PRE-CLINICAL

Collaboration with HemoShear Therapeutics, LLC, to explore novel approaches to identify and validate biological targets to treat gout using HemoShear’s proprietary disease modeling platform, REVEAL-Tx™. The platform combines physiological and computational models of disease to identify novel treatment approaches. This collaboration may lead to potential clinical targets or new studies of which Horizon will have the rights to commercialize any successful candidates.

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.

 

This information was last updated on September 17, 2019.