-- If approved, teprotumumab would be the first
and only approved medicine for people living with TED, fulfilling a
significant unmet need --
Horizon Therapeutics plc (Nasdaq: HZNP) today announced that the
Dermatologic and Ophthalmic Drugs Advisory Committee (DODAC) of the
U.S. Food and Drug Administration (FDA) voted unanimously that the
potential benefits of teprotumumab, a fully human monoclonal
antibody (mAb), outweigh the potential risks for the treatment of
Thyroid Eye Disease (TED).
TED is a rare, serious, progressive and vision-threatening
autoimmune disease, and is associated with proptosis (eye bulging),
diplopia (double vision), blurred vision, pain and facial
disfigurement that can significantly impact patients’ quality of
life. If approved, teprotumumab would be the first FDA-approved
medicine for TED. Teprotumumab is an investigational medicine and
its safety and efficacy have not been established.
“Today’s unanimously positive vote marks a significant step
towards the first FDA-approved treatment for patients with TED, a
vision-threatening autoimmune disease,” said Timothy Walbert,
chairman, president and chief executive officer, Horizon. “Our
comprehensive set of data presented today on teprotumumab’s
efficacy and safety is a testament to the extraordinary efforts of
the physicians who partnered with us on the clinical development
program, the TED patients who enrolled in our studies, and
Horizon’s research and development team. We believe that
teprotumumab has the potential to address a significant unmet need
for these patients and we look forward to working with the FDA as
it completes its review of our application.”
“TED can affect patients both physically and emotionally,
limiting their ability to perform everyday activities like driving,
working, reading, sleeping and participating in social activities,”
said Jeff Todd, president and chief executive officer, Prevent
Blindness. “As an organization dedicated to helping patients with
vision impairment, and those who are at significant risk, we are
extremely encouraged by today’s vote and hopeful this will change
the future of TED treatment by giving patients an option that has
been shown to improve the painful and vision-threatening aspects of
the disease.”
The FDA is currently evaluating a Biologics License Application
(BLA) for teprotumumab for the treatment of TED. Teprotumumab is
one of less than five medicines ever to receive Priority Review,
Orphan Drug, Fast Track and Breakthrough Therapy designations from
the FDA. The Prescription Drug User Fee Act (PDUFA) action date is
March 8, 2020. The FDA will consider today’s vote as it reviews the
BLA, although it is not obligated to follow the Committee’s
recommendation.
The efficacy and safety of teprotumumab for the treatment of TED
is supported by a robust body of clinical evidence. The BLA
submission is based on positive results from the Phase 3
confirmatory clinical trial, called OPTIC (Treatment of Graves’
Orbitopathy (Thyroid Eye Disease) to Reduce Proptosis with
Teprotumumab Infusions in a Randomized, Placebo-Controlled,
Clinical Study), as well as positive Phase 2 results.
The OPTIC study found that significantly more patients treated
with teprotumumab had a meaningful improvement in proptosis, or
bulging of the eye, as compared with placebo (82.9% of teprotumumab
patients compared to 9.5% of placebo patients). These results were
achieved within a six-month course of therapy. All secondary
endpoints were also met, including reduced diplopia (double
vision), improved quality of life (QoL) and reductions in Clinical
Activity Score (CAS), which measures the degree of inflammation,
including pain, swelling and redness. As previously reported, the
majority of adverse events experienced with teprotumumab treatment
were graded as mild to moderate and were managed in the trials,
with few discontinuations.
The OPTIC study was initiated after the Phase 2 study
demonstrated clinically meaningful and highly statistically
significant results in reducing proptosis and in the symptoms of
TED as measured by Overall Treatment Response. The Phase 2 study
was published in The New England Journal of Medicine in May 2017.
The Phase 2 and Phase 3 studies form a robust body of clinical
evidence on the use of teprotumumab in people living with TED.
About Thyroid Eye Disease
Thyroid Eye Disease (TED) is a serious, progressive and
vision-threatening autoimmune disease with a limited window of
activity that can last up to three years.1,2,3 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 orbit.4,5
This leads to a cascade of negative effects, which may cause
long-term, irreversible damage. Active TED lasts for up to three
years and is characterized by inflammation and tissue expansion
behind the eye.1,6 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.2,7 TED has only been shown to respond to pharmacotherapy
while the disease is active and inflammation is ongoing.8
Currently, patients must live with active TED until the
inflammation subsides, after which they are often left with
permanent and vision-impairing consequences.1,6
About Teprotumumab
Teprotumumab is a fully human monoclonal antibody (mAb) and a
targeted inhibitor of the insulin-like growth factor 1 receptor
(IGF-1R). Teprotumumab has received Priority Review, Orphan Drug,
Fast Track and Breakthrough Therapy designations from the FDA. The
clinical development program for teprotumumab in the treatment of
TED includes positive results from the Phase 2 clinical study,
which were published in The New England Journal of Medicine, as
well as positive results from the Phase 3 OPTIC confirmatory
clinical trial. The [Phase 3 OPTIC confirmatory trial was conducted
at leading centers in the U.S., Germany and Italy, with
co-principal investigators Raymond Douglas, M.D., Ph.D.,
Cedars-Sinai Medical Center and George Kahaly, M.D., Ph.D.,
Johannes Gutenberg University Medical Center. Horizon is also
conducting the OPTIC‐X extension trial to gather further insight
into the long-term efficacy and safety of teprotumumab.
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 an FDA decision on the
teprotumumab BLA, expectations regarding the review of the BLA and
the potential benefits of teprotumumab to patients. 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
the actual timing and process of review of the teprotumumab BLA and
whether the BLA is ultimately approved, 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
Ophthalmology. 2015.
https://www.hindawi.com/journals/joph/2015/249125/cta/. Accessed
Feb 22, 2019.
- Ross DS, et al. The 2016 European Thyroid Association/European
Group on Graves' Orbitopathy Guidelines for the Management of
Graves' Orbitopathy. European Thyroid Journal.2 March 2016.
https://www.ncbi.nlm.nih.gov/pubmed/27099835. Accessed Feb 22,
2019.
- Shan SJ, Douglas RS. The Pathophysiology of Thyroid Eye
Disease. Journal of Neuro-Ophthalmology. 2014; 34: 177-185.
- Bahn RS. Graves' Ophthalmopathy. The New England Journal of
Medicine. 25 February 2010.
https://www.nejm.org/doi/full/10.1056/NEJMra0905750. Accessed Feb
22, 2019.
- Pritchard J, et al. Igs from patients with Graves' disease
induce the expression of T cell chemoattractants in their
fibroblasts. The Journal of Immunology. 15 January 2002.
https://www.ncbi.nlm.nih.gov/pubmed/11777993. Accessed Feb 22,
2019.
- McKeag D, et al. Clinical features of dysthyroid optic
neuropathy: a European Group on Graves' Orbitopathy (EUGOGO)
survey. British Journal of Ophthalmology. 11 October 2006.
https://www.ncbi.nlm.nih.gov/pubmed/17035276. Accessed Feb 22,
2019.
- Mamoojee Y, Pearce SHS. Natural History. In: Wiersinga WM,
Kahaly GJ (eds): Graves’ Orbitopathy: A Multidisciplinary Approach
– Questions and Answers. Basel, Karger. 2017:93-104.
- Bothun ED, et al. Update on thyroid eye disease and management.
Clinical Ophthalmology. 19 October 2009.
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2770865/. Accessed Feb
22, 2019.
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version on businesswire.com: https://www.businesswire.com/news/home/20191213005479/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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