Horizon Pharma plc Announces Four Poster Presentations on PROCYSBI® (Cysteamine Bitartrate) Delayed-Release Capsules at Cy...
July 14 2017 - 8:00AM
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 the
presentation of results from a new study evaluating the effects of
treatment with PROCYSBI® (cysteamine bitartrate)
delayed-release capsules or immediate-release cysteamine bitartrate
on the breath of people living with nephropathic cystinosis.
In the study, patients receiving PROCYSBI had a 26 percent
reduction in exhaled dimethyl sulphide – a metabolite of cysteamine
bitartrate associated with halitosis (i.e., bad breath) – compared
to those receiving immediate-release cysteamine. The study is
one of four PROCYSBI poster presentations that can be viewed at the
Cystinosis Research Network (CRN) 2017 Family Conference in
Snowbird, Utah, on Saturday, July 15.
“Treatment with cysteamine bitartrate may cause an unpleasant
odor, particularly affecting the patient’s breath, and can impact
their willingness to maintain continuous cystine-depleting
therapy,” said Larry Greenbaum, M.D., Ph.D., division director,
Department of Pediatric Nephrology, Emory University School of
Medicine, who served as principal investigator for the study.
“This study is important because people living with
nephropathic cystinosis need to be treated with cystine-depleting
therapy early and continuously to avoid the serious and potentially
life-threatening impact of the disease on tissues and organs.”
Summary of Study Results:
- This was an optional substudy of an ongoing Phase 3b long-term
open-label study of the safety, tolerability and effectiveness of
PROCYSBI versus immediate-release cysteamine. The substudy
evaluated 20 patients experiencing halitosis as a side effect while
receiving immediate-release cysteamine during the long-term
open-label study.
- Patients in the substudy received immediate-release cysteamine
for three months, followed by a one-month dose adjustment period
before transitioning to PROCYSBI for an additional three months.
- The study compared exhaled dimethyl sulphide concentration in
patients treated with immediate-release cysteamine every six hours
to levels when treated with PROCYSBI every 12 hours.
- Patients taking PROCYSBI had a 26 percent reduction in exhaled
dimethyl sulphide compared with patients taking immediate-release
cysteamine.
Most frequently reported adverse events seen in ≥5 percent of
patients taking PROCYSBI were vomiting, nausea, abdominal pain,
breath odor, diarrhea, skin odor, fatigue and rash.
“We are very pleased to support the Cystinosis Research
Network’s Family Conference, as these events provide an
understanding of the many different challenges faced by people
living with nephropathic cystinosis,” said Jeffrey W.
Sherman, M.D., FACP, executive vice president, research and
development and chief medical officer, Horizon Pharma plc.
“This halitosis substudy is part of our ongoing effort to analyze
the effects of PROCYSBI, because there are many considerations –
such as the effect of a medicine on body odor and breath – that are
very important for people working to manage their condition while
living as normal a life as possible.”
In addition to the halitosis substudy (Quantification of
Dimethyl Sulphide Associated with Cysteamine Bitartrate-Induced
Halitosis Using Breath Analysis in Cystinosis Patients Treated with
Delayed-Release and Immediate-Release Cysteamine Bitartrate), the
following encore presentations, which were presented at previous
medical meetings, will be available for viewing at CRN:
- Title: A Pragmatic Trial of
Delayed-Release Cysteamine Bitratrate in Children < 6 Years Old
with Cystinosis Lead Author: Craig B.
Langman, M.D., Feinberg School of Medicine, Northwestern
University
- Title: Delayed-Release Cysteamine in
Nephropathic Cystinosis Patients after Renal Transplant: A Subgroup
AnalysisLead Author: Paul Grimm, M.D.,
Stanford University School of Medicine
- Title: The Effect of Food and Liquid pH
on the Integrity of Enteric-Coated Beads from Cysteamine Bitartrate
Delayed-Release CapsulesLead Author: Nadine
Pavloff, Ph.D., Horizon Pharma plc
About Cystinosis Cystinosis is a rare,
life-threatening metabolic lysosomal storage disorder that causes
toxic accumulation of cystine in all cells, tissues, and organs in
the body. Elevated cystine leads to progressive, irreversible
tissue damage and multi-organ failure, including kidney failure,
blindness, muscle wasting and premature death. It is
estimated that only about 2,000 people worldwide are currently
diagnosed with cystinosis. Nephropathic or “classic
infantile” cystinosis – the most common and most severe form of the
disease – is typically diagnosed in infancy and requires lifelong
therapy.1
About PROCYSBIIn the United States, PROCYSBI®
(cysteamine bitartrate) delayed-release capsules is a cystine
depleting agent indicated for the treatment of nephropathic
cystinosis in adults and pediatric patients two years of age and
older.
Important Safety Information
CONTRAINDICATIONS:
- Hypersensitivity to penicillamine or cysteamine.
WARNINGS AND PRECAUTIONS:
- Ehlers-Danlos-like Syndrome: Skin and bone lesions that
resemble clinical findings for Ehlers-Danlos-like syndrome have
been reported in patients treated with high doses of
immediate-release cysteamine bitartrate or other cysteamine salts.•
These include molluscoid pseudotumors (purplish hemorrhagic
lesions), skin striae, bone lesions (including osteopenia,
compression fractures, scoliosis and genu valgum), leg pain, and
joint hyperextension. • One patient on immediate-release
cysteamine bitartrate with serious skin lesions subsequently died
of acute cerebral ischemia with marked vasculopathy.• Monitor
patients for development of skin or bone lesions and interrupt
PROCYSBI dosing if patients develop these lesions. PROCYSBI
may be restarted at a lower dose under close supervision, then
slowly increase to the appropriate therapeutic dose.
- Skin Rash: Severe skin rashes such as erythema multiforme
bullosa or toxic epidermal necrolysis have been reported in
patients receiving immediate-release cysteamine bitartrate.
If severe skin rashes develop, permanently discontinue use of
PROCYSBI.
- Gastrointestinal Ulcers and Bleeding: Gastrointestinal
(GI) ulceration and bleeding have been reported in patients
receiving immediate-release cysteamine bitartrate.• GI tract
symptoms including nausea, vomiting, anorexia and abdominal pain,
sometimes severe, have been associated with cysteamine. If
severe GI tract symptoms develop, consider decreasing the dose of
PROCYSBI.
- Central Nervous System Symptoms: Central Nervous System
(CNS) symptoms such as seizures, lethargy, somnolence, depression,
and encephalopathy have been associated with immediate-release
cysteamine.• Neurological complications have also been
described in some patients with cystinosis who have not been
treated with cysteamine. • Carefully evaluate and monitor
patients who develop CNS symptoms. Interrupt medication or adjust
the dose as necessary for patients with severe symptoms or with
symptoms that persist or progress. • Inform patients that
PROCYSBI may impair their ability to perform tasks such as driving
or operating machinery.
- Leukopenia and/or Elevated Alkaline Phosphatase Levels:
Cysteamine has been associated with reversible leukopenia and
elevated alkaline phosphatase levels. Monitor white blood
cell counts and alkaline phosphatase levels. If tests values
remain elevated, consider decreasing the dose or discontinuing the
drug until values revert to normal.
- Benign Intracranial Hypertension: Benign intracranial
hypertension (pseudotumor cerebri; PTC) and/or papilledema has been
reported in patients receiving immediate-release cysteamine
bitartrate treatment.• Monitor patients for signs and symptoms of
PTC, including headache, tinnitus, dizziness, nausea, diplopia,
blurry vision, loss of vision, pain behind the eye or pain with eye
movement. If signs/symptoms persist, interrupt dosing or
decrease the dose and refer the patient to an ophthalmologist.
If the diagnosis is confirmed, permanently discontinue use of
PROCYSBI.
ADVERSE REACTIONS:
The most common adverse reactions (≥5%) in patients treated in
clinical trials are vomiting, nausea, abdominal pain, breath odor,
diarrhea, skin odor, fatigue, rash and headache.
DRUG INTERACTIONS:
- Drugs that Increase Gastric pH: Administer PROCYSBI at
least one hour before or one hour after medications containing
bicarbonate or carbonate.
- Consumption of alcohol with PROCYSBI may increase the rate of
cysteamine release and/or adversely alter the pharmacokinetic
properties, as well as the effectiveness and safety of
PROCYSBI.
- PROCYSBI can be administered with electrolyte (except
bicarbonate) and mineral replacements necessary for management of
Fanconi Syndrome as well as vitamin D and thyroid hormone.
USE IN SPECIFIC POPULATIONS:
Lactation: Breastfeeding is not recommended while taking
PROCYSBI.
Please see the Full Prescribing Information
at www.PROCYSBI.com.
To report SUSPECTED ADVERSE REACTIONS, contact Horizon Pharma at
1-866-479-6742 (Option 1) or FDA at 1-800-FDA-1088
or www.fda.gov/medwatch.
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 statements regarding
the potential of PROCYSBI to treat patients with nephropathic
cystinosis and the side effects of PROCYSBI. 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 whether future PROCYSBI results will be in line with prior
PROCYSBI results and whether patients are willing to use PROCYSBI
to treat nephropathic cystinosis, as well as those factors
described in Horizon Pharma'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 Pharma does not undertake any obligation to
update or revise these statements, except as may be required by
law.
References:
- Cystinosis Research Foundation. “About cystinosis.”
Available
at http://www.cystinosisresearch.org/About-Cystinosis/.
Accessed July 12, 2017.
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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