-- First and only FDA-approved medicine for
TED, a serious, progressive, vision-threatening rare disease --
-- Clinical improvements were seen as early as
six weeks, with continued improvement across the 24-week treatment
period --
-- Approval comes ahead of the Prescription
Drug User Fee Act (PDUFA) goal date of March 8, 2020 --
-- Horizon to host investor webcast Tuesday,
January 21 at 5:00 p.m. ET --
Horizon Therapeutics plc (Nasdaq: HZNP) today announced that the
U.S. Food and Drug Administration (FDA) has approved TEPEZZATM
(teprotumumab-trbw) for the treatment of Thyroid Eye Disease (TED).
TEPEZZA is the first and only FDA-approved medicine for the
treatment of TED, a serious, progressive and vision-threatening
rare autoimmune disease that is associated with proptosis (eye
bulging), diplopia (double vision), blurred vision, pain,
inflammation and facial disfigurement. TEPEZZA is a fully human
monoclonal antibody (mAb) and a targeted inhibitor of the
insulin-like growth factor-1 receptor (IGF-1R) that is administered
to patients once every three weeks for a total of eight
infusions.
“Today is a great day for people living with Thyroid Eye
Disease, a rare, vision-threatening disease that previously had no
FDA-approved treatment options,” said Timothy Walbert, chairman,
president and chief executive officer, Horizon. “The TED community
has gone far too long without an FDA-approved therapy, and we are
grateful to the people living with TED and physicians who partnered
with us on the clinical development program that led to today’s
approval of TEPEZZA. This also marks the early approval of
Horizon’s first Biologics License Application – a key step in our
evolution to an innovation-focused biopharma company, developing
new medicines for debilitating diseases with few or no treatment
options.”
The FDA approval of TEPEZZA comes ahead of the Prescription Drug
User Fee Act (PDUFA) goal date of March 8, 2020. The medicine
received Priority Review, Orphan Drug, Fast Track and Breakthrough
Therapy designations from the FDA.
“The FDA approval of TEPEZZA is momentous for the TED community
and has the potential to change the treatment paradigm for TED –
providing new hope for people who are living with this horrible,
vision-threatening disease,” said Raymond Douglas, M.D., Ph.D.,
director of the Orbital and Thyroid Eye Disease Program,
Cedars-Sinai Medical Center and co-principal investigator of the
TEPEZZA Phase 3 confirmatory clinical trial. “Today’s news brings
forward a medicine for patients that targets the underlying biology
of the disease and has been shown to significantly improve eye
bulging and double vision, which are the most debilitating aspects
of the disease.”
“TEPEZZA is a much-needed breakthrough for a community of people
who have historically had to struggle in pain as their symptoms
progress – risking permanent damage to their eyes and making it
extremely difficult to go about their daily lives,” said Jeff Todd,
president and chief executive officer, Prevent Blindness. “This
approval is meaningful to our organization because we are committed
to helping patients with vision impairment and those who are at
significant risk.”
The FDA approval of TEPEZZA is supported by a robust body of
clinical evidence, including statistically significant, positive
results from the Phase 2 clinical study, as well as the Phase 3
confirmatory clinical study OPTIC (Treatment of Graves’
Orbitopathy [Thyroid Eye Disease] to Reduce Proptosis
with Teprotumumab Infusions in a Randomized,
Placebo-Controlled, Clinical Study). The OPTIC study found
that significantly more patients treated with TEPEZZA (82.9%) had a
meaningful improvement in proptosis (≥ 2 mm) as compared with
placebo patients (9.5%) (p˂0.001) without deterioration in the
fellow eye at Week 24. Additional secondary endpoints were also
met, including a change from baseline of at least one grade in
diplopia (double vision) in 67.9% of patients receiving TEPEZZA
compared to 28.6% of patients receiving placebo (p=0.001) at Week
24. In a related analysis of the Phase 2 and Phase 3 clinical
studies, there were more patients with complete resolution of
diplopia among those treated with TEPEZZA (53%) compared with those
treated with placebo (25%). The majority of adverse events
experienced with TEPEZZA treatment were graded as mild to moderate
and were manageable in the trials, with few discontinuations or
therapy interruptions.
Horizon will conduct a post-marketing study to evaluate safety
in a larger patient population as was discussed at the Dermatologic
and Ophthalmic Drugs Advisory Committee (DODAC) FDA Advisory
Committee meeting on December 13, 2019, where the committee voted
unanimously (12-0) that TEPEZZA demonstrated a positive benefit
risk profile. This study will also evaluate retreatment rates
relative to how long patients receive the medicine.
TEPEZZA is expected to be available in the United States in the
coming weeks. To speak with a Nurse Advocate about TED, patients
can call 1-833-483-7399. To learn more about TEPEZZA, visit
TEPEZZA.com.
As a result of the FDA approval of TEPEZZA, Horizon will make
approximately $105 million in milestone payments during the first
half of 2020.
Investor Webcast Information
Horizon will discuss the FDA approval of TEPEZZA during an
investor webcast Tuesday, January 21 at 5:00 p.m. ET. The live
webcast and a replay may be accessed at
http://ir.horizontherapeutics.com. Please connect to Horizon’s
website at least 15 minutes prior to the live webcast to ensure
adequate time for any software download that may be needed to
access the webcast. A replay of the webcast will be available
approximately two hours after the live webcast.
About Thyroid Eye Disease
Thyroid Eye Disease (TED) is a serious, progressive and
vision-threatening rare autoimmune disease.1 While TED often occurs
in people living with hyperthyroidism or Graves’ disease, 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. As TED progresses, it causes
serious damage – including proptosis (eye bulging), strabismus
(misalignment of the eyes) and diplopia (double vision) – and in
some cases can lead to blindness.4,5 Historically, patients have
had to live with TED until the inflammation subsides, after which
they are often left with permanent and vision-impairing
consequences.1,6
About TEPEZZA
INDICATION
TEPEZZA is indicated for the treatment of Thyroid Eye
Disease.
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 managed with medications for
glycemic control, if necessary. Monitor patients for elevated blood
glucose and symptoms of hyperglycemia while on treatment with
TEPEZZA. Patients with preexisting diabetes should be under
appropriate glycemic control before 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 and
dry skin.
For additional information on TEPEZZA, please see Full
Prescribing Information at TEPEZZAhcp.com.
About Horizon
Horizon is focused on researching, developing and
commercializing medicines that address critical needs for people
impacted by rare and rheumatic 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,
please visit www.horizontherapeutics.com, follow us @HorizonNews on
Twitter, like us on Facebook or explore career opportunities on
LinkedIn.
Forward-Looking Statements
This press release contains forward-looking statements,
including statements regarding the timing of TEPEZZA’s availability
in the United States, the potential benefits of TEPEZZA as a
treatment of TED, plans to conduct post-marketing clinical trials
and expected milestone payment obligations. 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 Horizon’s ability
to launch TEPEZZA in the United States, whether TEPEZZA is
successfully commercialized and adopted by physicians and patients,
the extent to which reimbursement is available for TEPEZZA, and
potential delays in initiating and completing clinical trials, 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
- Barrio-Barrio J, et al. Graves' Ophthalmopathy: VISA versus
EUGOGO Classification, Assessment, and Management. Journal of
Ophthalmopathy. 2015;2015:1-16.
- Weightman DR, et al. Autoantibodies to IGF-1 Binding Sites in
Thyroid Associated Ophthalmopathy. Autoimmunity. 1993; 16(4):
251–257.
- 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.
- Ross DS, et al. The 2016 European Thyroid Association /European
Group on Graves' Orbitopathy Guidelines for the Management of
Graves ' Orbitopathy. Eur Thyroid J. 2016;5(1):9-26.
- 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.
- Bothun ED, et al. Update on thyroid eye disease and management.
Clinical Ophthalmology. 2009;3:543-551.
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version on businesswire.com: https://www.businesswire.com/news/home/20200121006017/en/
Tina Ventura Senior Vice President, Investor Relations
Investor-relations@horizontherapeutics.com
Ruth Venning Executive Director, Investor Relations
Investor-relations@horizontherapeutics.com
U.S. Media Contact: Matt Flesch Vice President,
Communications and Patient Advocacy
media@horizontherapeutics.com
Rachel Vann Associate Director, Product Communications
media@horizontherapeutics.com
Ireland Media Contact: Gordon MRM Ray Gordon
ray@gordonmrm.ie
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