Horizon Therapeutics plc Announces FDA Approval of an Update to the Indication Language for TEPEZZA® (teprotumumab-trbw) to Specify its Use in Thyroid Eye Disease (TED) Patients Regardless of Disease Activity or Duration

-- Updated indication language reinforces the importance of unrestricted access for patients across full spectrum of Thyroid Eye Disease --

Horizon Therapeutics plc (Nasdaq: HZNP) today announced that the U.S. Food and Drug Administration (FDA) has approved an update to the Indications and Usage section of the TEPEZZA label to specify its use for the treatment of “Thyroid Eye Disease regardless of Thyroid Eye Disease activity or duration.” TEPEZZA is the first and only medicine approved by the FDA for the treatment of TED – a serious, progressive, debilitating and potentially vision-threatening rare autoimmune disease.¹

The label update follows positive topline results from a randomized, double-masked, placebo-controlled Phase 4 clinical trial (NCT04583735) that were announced earlier this week, which demonstrated that patients with an initial diagnosis of TED between two to 10 years (mean duration of 5.2 years; SD 1.77) and with low disease activity, or CAS, (mean CAS of 0.4; SD 0.49) achieved a statistically significant reduction in proptosis from baseline at Week 24 after receiving TEPEZZA compared to those receiving placebo. In the trial, no new safety signals were observed.

“We worked closely with the FDA on this important label update, which further reinforces the potential benefit of TEPEZZA in people impacted by Thyroid Eye Disease regardless of disease activity or duration,” said Tim Walbert, chairman, president and chief executive officer, Horizon. “While TEPEZZA already had a broad indication for the treatment of Thyroid Eye Disease, the new indication language now references treatment regardless of disease activity or duration. The updated indication reinforces the importance of unrestricted access for all eligible patients across the full spectrum of Thyroid Eye Disease. This creates an opportunity to ease the access burden for patients and physicians with the goal of decreasing time to therapy for patients who may benefit from TEPEZZA.”

The Company plans to present data from the Phase 4 trial at a future medical congress and publish the data in a peer-reviewed medical journal.

About Thyroid Eye Disease (TED)

TED is a serious, progressive, debilitating and potentially vision-threatening rare autoimmune disease.¹ TED often occurs in people living with Graves’ disease, but it is a distinct disease that is caused by autoantibodies activating an IGF-1R-mediated signaling complex on cells within the retro-orbital space.^2,3 This leads to a cascade of negative effects, which may cause long-term, irreversible damage, including blindness. Signs and symptoms of TED may include dry eyes and grittiness; redness, swelling and excessive tearing; eyelid retraction; proptosis; pressure and/or pain behind the eyes; and diplopia.^4,5

About TEPEZZA

INDICATION

TEPEZZA is indicated for the treatment of Thyroid Eye Disease regardless of Thyroid Eye Disease activity or duration.

IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS

Infusion Reactions: TEPEZZA may cause infusion reactions. Infusion reactions have been reported in approximately 4% of patients treated with TEPEZZA. Reported infusion reactions have usually been mild or moderate in severity. Signs and symptoms may include transient increases in blood pressure, feeling hot, tachycardia, dyspnea, headache and muscular pain. Infusion reactions may occur during an infusion or within 1.5 hours after an infusion. In patients who experience an infusion reaction, consideration should be given to premedicating with an antihistamine, antipyretic or corticosteroid and/or administering all subsequent infusions at a slower infusion rate.

Preexisting Inflammatory Bowel Disease: TEPEZZA may cause an exacerbation of preexisting inflammatory bowel disease (IBD). Monitor patients with IBD for flare of disease. If IBD exacerbation is suspected, consider discontinuation of TEPEZZA.

Hyperglycemia: Increased blood glucose or hyperglycemia may occur in patients treated with TEPEZZA. In clinical trials, 10% of patients (two-thirds of whom had preexisting diabetes or impaired glucose tolerance) experienced hyperglycemia. Hyperglycemic events should be controlled with medications for glycemic control, if necessary. Assess patients for elevated blood glucose and symptoms of hyperglycemia prior to infusion and continue to monitor while on treatment with TEPEZZA. Ensure patients with hyperglycemia or preexisting diabetes are under appropriate glycemic control before and while receiving TEPEZZA.

ADVERSE REACTIONS

The most common adverse reactions (incidence ≥5% and greater than placebo) are muscle spasm, nausea, alopecia, diarrhea, fatigue, hyperglycemia, hearing impairment, dysgeusia, headache, dry skin, weight decreased, nail disorders and menstrual disorders.

Please see Full Prescribing Information or visit TEPEZZAhcp.com for more information.

About Horizon

Horizon is focused on the discovery, development and commercialization of medicines that address critical needs for people impacted by rare, autoimmune and severe inflammatory diseases. Our pipeline is purposeful: We apply scientific expertise and courage to bring clinically meaningful therapies to patients. We believe science and compassion must work together to transform lives. For more information on how we go to incredible lengths to impact lives, visit www.horizontherapeutics.com and follow us on Twitter, LinkedIn, Instagram and Facebook.

Forward-Looking Statements

This press release contains forward-looking statements, including statements regarding the benefits of TEPEZZA as a treatment for TED and the impact of the update to the TEPEZZA label. These forward-looking statements are based on management expectations and assumptions as of the date of this press release, and actual results may differ materially from those in these forward-looking statements as a result of various factors. These factors include risks regarding whether additional clinical trial results or data analyses will be consistent with preliminary results or results of other trials or Horizon’s expectations, the risks associated with adoption of novel medicines and factors that may change physician treatment strategies, as well as those described in Horizon's filings with the United States Securities and Exchange Commission, including those factors discussed under the caption “Risk Factors” in those filings. Forward-looking statements speak only as of the date of this press release and Horizon does not undertake any obligation to update or revise these statements, except as may be required by law.

References

1. Barrio-Barrio J, et al. Graves' Ophthalmopathy: VISA versus EUGOGO Classification, Assessment, and Management. Journal of Ophthalmopathy. 2015;2015:249125.
2. Weightman DR, et al. Autoantibodies to IGF-1 Binding Sites in Thyroid Associated Ophthalmopathy. Autoimmunity. 1993;16(4):251–257.
3. Pritchard J, et al. Immunoglobulin Activation of T Cell Chemoattractant Expression in Fibroblasts from Patients with Graves’ Disease Is Mediated Through the Insulin-Like Growth Factor 1 Receptor Pathway. J Immunol. 2003;170:6348-6354.
4. Bartalena L, et al. The 2021 European Group on Graves' orbitopathy (EUGOGO) clinical practice guidelines for the medical management of Graves' orbitopathy. Eur J Endocrinol. 2021;185(4):G43-G67.
5. McKeag D, et al. Clinical features of dysthyroid optic neuropathy: a European Group on Graves' Orbitopathy (EUGOGO) survey. Br J Ophthalmol. 2007;91:455-458.

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