Horizon Pharma plc (NASDAQ:HZNP), a biopharmaceutical company
focused on improving patients' lives by identifying, developing,
acquiring and commercializing differentiated and accessible
medicines that address unmet medical needs, today announced that
four new data analyses evaluating the use of KRYSTEXXA®
(pegloticase) in patients with refractory chronic gout will be
presented at the Annual European Congress of Rheumatology (EULAR)
2017 in Madrid on June 15, 2017.
One study (Characterization of Patients with Chronic Refractory
Gout Who Do and Do Not Have Clinically Apparent Tophi:
Response to Pegloticase; abstract THU0448) retrospectively
analyzed data from two pivotal, six-month, randomized clinical
trials comparing patients with and without clinically apparent
tophi, which are hard uric acid deposits under the skin that
contribute to bone and cartilage destruction. In the
analysis, both groups showed significant clinical benefit,
improvement in tender or swollen joints, quality of life and
reduction in flares, over six months of treatment with
KRYSTEXXA.
“All gout is tophaceous, but many patients dealing with chronic,
painful symptoms may not have visible tophi,” said Jeffrey W.
Sherman, M.D., FACP, executive vice president, research and
development and chief medical officer, Horizon Pharma plc.
“This retrospective analysis indicates that KRYSTEXXA provided
benefit not only among patients dealing with the challenges
typically associated with clinically apparent tophi – such as
disability – but also among those who do not have visible tophi.”
Summary of Results
- The study compared 62 patients who had clinically apparent
tophi at baseline and 23 patients who did not.
- At baseline, patients with clinically apparent tophi had more
tender and swollen joints, greater disability, and greater
arthritis severity, but otherwise their clinical presentation was
similar to patients without clinically apparent tophi.
- Both groups had significant clinical benefit over six months of
treatment with KRYSTEXXA.
- Among patients with clinically apparent tophi, at six months of
KRYSTEXXA treatment there were significant reductions in serum uric
acid (p<0.0001), flares (p<0.0001), Patient Global Assessment
(PGA; p<0.0001), tender and swollen joints (TJC and SJC; both
p<0.0001), the Health Assessment Questionnaire-Disability Index
(HAQ-DI; p=0.02) and Bodily Pain from the Medical Outcomes Study
Short Form 36 item (SF-36; p<0.0001).
- Among patients without clinically apparent tophi, at six months
of KRYSTEXXA treatment there were significant reductions in serum
uric acid (p<0.0001), flares (p=0.003), PGA (p=0.009), TJC
(p=0.01) and SJC (p=0.003), the Arthritis-Specific Health Index
(ASHI; p=0.0001) and SF-36 (p=0.03).
- The most commonly reported adverse reactions in clinical trials
with KRYSTEXXA are gout flares, infusion reactions, nausea,
contusion or ecchymosis, nasopharyngitis, constipation, chest pain,
anaphylaxis and vomiting.
“Too many patients endure regular gout flares and get to a point
where they simply feel out of options,” said Peter E. Lipsky, M.D.,
director of clinical operations for AMPEL BioSolutions, and author
of the study. “These data provide important information for
rheumatologists and other healthcare providers about the use of
KRYSTEXXA for patients living with the painful and debilitating
symptoms of chronic gout, including those with and without visible
tophi.”
Findings from another retrospective analysis (Rapid Tophus
Resolution in Chronic Refractory Gout Patients Treated with
Pegloticase; abstract THU0416) of data from the same pivotal
clinical trials showed that KRYSTEXXA rapidly resolved tophi in
patients considered to be responders by profoundly lowering and
maintaining serum uric acid levels. Responders were defined
as patients who had uric acid levels less than 6.0 mg/dl for 80
percent of the clinical study during extensive
monitoring.
KRYSTEXXA is the only medicine approved by the U.S. Food
and Drug Administration (FDA) for the treatment of refractory
chronic gout, which represents an orphan disease subset of the
common form of gout. In general, gout is a type of chronic
inflammatory arthritis in which uric acid builds up in the blood
and can lead to severe pain and joint destruction. Patients
with refractory chronic gout continue to have abnormally high
levels of uric acid and continued symptoms despite the use of
conventional therapies.
Schedule of Data Analyses Presented at EULAR Congress
2017
- Title: Characterization of Patients with
Chronic Refractory Gout Who Do and Do Not Have Clinically Apparent
Tophi: Response to Pegloticase Authors:
N. L. Edwards, J. Singh, O. Troum, A. Yeo, P. Lipsky
Date: Thursday, June 15, 2017 Time:
11:45 CET Poster #:
THU0448
- Title: Rapid Tophus Resolution in
Chronic Refractory Gout Patients Treated with Pegloticase
Authors: B. Mandell, A. Yeo, P. Lipsky
Date: Thursday, June 15, 2017 Time:
11:45 CET Poster #:
THU0416
- Title: Evidence Based Development of
Criteria for Complete Response in Patients with Chronic Refractory
Gout Authors: N. Schlesinger, P. Khanna, A.
Yeo, P. Lipsky Date: Thursday, June 15, 2017
Time: 11:45 CET Poster #:
THU0449
- Title: Lack of Predictive Value of the
NIAID/FAAN Criteria to Identify Subjects with Evidence of Immune
Activation After Receiving Pegloticase for Chronic Refractory Gout
Authors: L. Calabrese, A. Kavanaugh, A. Yeo,
P. Lipsky Date: Thursday, June 15, 2017
Time: 11:45 CET Poster #:
THU0438
About KRYSTEXXA®
INDICATIONS AND USAGEKRYSTEXXA® (pegloticase) is indicated
for the treatment of chronic gout in adult patients who have failed
to normalize serum uric acid and whose signs and symptoms are
inadequately controlled with xanthine oxidase inhibitors at the
maximum medically appropriate dose or for whom these drugs are
contraindicated.
Important Limitations of Use: KRYSTEXXA is not recommended
for the treatment of asymptomatic hyperuricemia.
IMPORTANT SAFETY INFORMATIONAnaphylaxis and infusion
reactions have been reported
to occur during and after administration of KRYSTEXXA.
Anaphylaxis may occur with any infusion, including a first
infusion, and generally manifests within 2 hours of the infusion.
However, delayed-type hypersensitivity reactions have also
been reported. KRYSTEXXA should be administered in healthcare
settings and by healthcare providers prepared to manage anaphylaxis
and infusion reactions. Patients should be premedicated with
antihistamines and corticosteroids. Patients should be
closely monitored for an appropriate period of time for anaphylaxis
after administration of KRYSTEXXA. Monitor serum uric acid
levels prior to infusions and consider discontinuing treatment if
levels increase to above 6
mg/dL, particularly when 2 consecutive levels
above 6
mg/dL are
observed.
The risk of anaphylaxis and infusion reactions is higher in
patients who have lost therapeutic response.
Concomitant use of KRYSTEXXA and oral urate-lowering agents may
blunt the rise of sUA levels. Patients should discontinue
oral urate-lowering agents and not institute therapy with oral
urate-lowering agents while taking KRYSTEXXA.
In the event of anaphylaxis or infusion reaction, the infusion
should be slowed, or stopped and restarted at a slower rate.
Inform patients of the symptoms and signs of anaphylaxis, and
instruct them to seek immediate medical care should anaphylaxis
occur after discharge from the healthcare setting.
CONTRAINDICATIONS: G6PD DEFICIENCY
ASSOCIATED HEMOLYSIS AND
METHEMOGLOBINEMIAScreen patients for G6PD
deficiency prior to starting KRYSTEXXA. Life threatening
hemolysis and methemoglobinemia have been reported with KRYSTEXXA
in patients with G6PD deficiency. Do not administer KRYSTEXXA
to patients with G6PD deficiency.
GOUT FLARESAn increase in gout
flares is frequently observed upon initiation of anti-hyperuricemic
therapy, including treatment with KRYSTEXXA. If a gout flare
occurs during treatment, KRYSTEXXA need not be discontinued. Gout
flare prophylaxis with a non-steroidal anti-inflammatory drug
(NSAID) or colchicine is recommended starting at least 1 week
before initiation of KRYSTEXXA therapy and lasting at least 6
months, unless medically contraindicated or not tolerated.
CONGESTIVE HEART
FAILUREKRYSTEXXA has not been formally studied in
patients with congestive heart failure, but some patients in the
clinical trials experienced exacerbation. Exercise caution
when using KRYSTEXXA in patients who have congestive heart failure
and monitor patients closely following infusion.
ADVERSE REACTIONSThe most
commonly reported adverse reactions in clinical trials with
KRYSTEXXA are gout flares, infusion reactions, nausea, contusion or
ecchymosis, nasopharyngitis, constipation, chest pain, anaphylaxis
and vomiting.
Please see Full
Prescribing
Information and Medication
Guide for more
information.
About Horizon Pharma plc Horizon Pharma plc is
a biopharmaceutical company focused on improving patients' lives by
identifying, developing, acquiring and commercializing
differentiated and accessible medicines that address unmet medical
needs. The Company markets 11 medicines through its orphan,
rheumatology and primary care business units. For more
information, please visit www.horizonpharma.com.
Follow @HZNPplc on Twitter or view careers on
our LinkedIn page.
Forward-Looking Statements This press release
contains forward-looking statements, including, but not limited to,
statements related to the benefits of KRYSTEXXA to patients with
gout, and other statements that are not historical facts.
These forward-looking statements are based on Horizon Pharma's
current expectations and inherently involve significant risks and
uncertainties. Actual results and the timing of events could
differ materially from those anticipated in such forward looking
statements as a result of these risks and uncertainties, which
include, without limitation, risks related to the impact of
KRYSTEXXA on patients with gout differing from historical results
and healthcare providers not using KRYSTEXXA with gout patients, as
well as other risks related to Horizon Pharma's business detailed
from time-to-time under the caption "Risk Factors" and elsewhere in
Horizon Pharma's SEC filings and reports, including in its Annual
Report on Form 10-K for the year ended December 31, 2016 and
subsequent quarterly reports on Form 10-Q. Horizon Pharma
undertakes no duty or obligation to update any forward-looking
statements contained in this press release as a result of new
information, future events or changes in its expectations.
Contacts:
Tina Ventura
Senior Vice President, Investor Relations
Investor-relations@horizonpharma.com
Ruth Venning
Executive Director, Investor Relations
Investor-relations@horizonpharma.com
U.S. Media Contact:
Matt Flesch
Executive Director, Product Communications
media@horizonpharma.com
Ireland Media Contact:
Ray Gordon
Gordon MRM
ray@gordonmrm.ie
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