Horizon Pharma plc to Present Data on RAVICTI® (glycerol phenylbutyrate) Oral Liquid for Urea Cycle Disorder Patients Aged T...
March 22 2017 - 9:00AM
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 new data on the use of RAVICTI® (glycerol
phenylbutyrate) Oral Liquid in children living with urea cycle
disorders (UCD) two months to two years of age. These data
will be presented on Thursday, March 23 at the American College of
Medical Genetics and Genomics (ACMG) Annual Clinical Genetics
Meeting in Phoenix, Arizona.
The study results are from two clinical trials that enrolled
children with UCDs aged two months to less than two years.
Study participants were predominantly on relatively stable doses of
sodium phenylbutyrate and switched to equivalent doses of
RAVICTI. The primary outcomes measured in the study were
targeted mean normalized plasma ammonia of less than 100 μmol/L,
hyperammonemic crises (HACs) and adverse events (AEs). Safety
and efficacy was also evaluated after patients transitioned to
RAVICTI.
“The RAVICTI development program is part of our overall approach
to reinvest in our medicines for people living with rare diseases,”
said Jeffrey W. Sherman, M.D., FACP, executive vice president,
research and development and chief medical officer, Horizon Pharma
plc. “While UCDs are very rare and can be devastating,
continued advancements in diagnosis and ongoing studies evaluating
management options are helping improve the long-term outcome of
people living with the disorder.”
RAVICTI is currently indicated for use as a nitrogen-binding
agent for chronic management of adult and pediatric patients two
years of age or greater with UCDs who cannot be managed by dietary
protein restriction and/or amino acid supplementation alone.
RAVICTI is not indicated for patients less than two years of
age. In June 2016, Horizon submitted a supplemental New Drug
Applications (sNDA) with the U.S. Food and Drug Administration
(FDA) seeking to expand the approved age range for RAVICTI to
include children as young as two months of age.
The poster presentation (abstract #2873; poster #71), entitled
“Safety and Efficacy of Glycerol Phenylbutyrate for Management of
Urea Cycle Disorders in Patients Aged 2 Months to 2 Years,” will be
presented on Thursday, March 23, 2017 from 10:00 a.m. – 11:30 a.m.
at the ACMG Annual Meeting.
About Urea Cycle DisordersA UCD is a rare
genetic disorder that affects approximately 1 in 35,000 live births
in the United States. It is caused by an enzyme
deficiency in the urea cycle, a process that is responsible for
converting excess ammonia from the bloodstream and ultimately
removing it from the body. Because of this, people with a UCD
experience hyperammonemia, or elevated ammonia levels in their
blood that can then reach the brain where it can cause irreversible
brain damage, coma or death. UCD symptoms may first occur at
any age depending on the severity of the disorder, with more severe
defects presenting earlier in life.1
About RAVICTIIn the U.S., RAVICTI was approved
in February 2013 and is indicated for the chronic management of
adult and pediatric patients ≥2 years of age with UCDs who cannot
be managed by dietary protein restriction and/or amino acid
supplementation alone. RAVICTI must be used with dietary
protein restriction and, in some cases, dietary supplements (e.g.,
essential amino acids, arginine, citrulline, protein-free calorie
supplements).
Important Safety Information
LIMITATIONS OF USE
- RAVICTI is not indicated for the treatment of acute
hyperammonemia in patients with UCDs because more rapidly acting
interventions are essential to reduce plasma ammonia levels.
- The safety and efficacy of RAVICTI for the treatment of
N-acetylglutamate synthase (NAGS) deficiency has not been
established.
- The use of RAVICTI in patients <2 months of age is
contraindicated.
CONTRAINDICATIONS:RAVICTI is contraindicated in
patients:
- Less than 2 months of age. Children <2 months of age may
have immature pancreatic exocrine function, which could impair
hydrolysis of RAVICTI, leading to impaired absorption of
phenylbutyrate and hyperammonemia.
- With known hypersensitivity to phenylbutyrate. Signs of
hypersensitivity include wheezing, dyspnea, coughing, hypotension,
flushing, nausea, and rash.
WARNINGS AND PRECAUTIONS
- Neurotoxicity: Phenylacetate (PAA), the major metabolite
of RAVICTI, may be toxic at levels ≥500 µg/mL. Reduce RAVICTI
dosage if symptoms of neurotoxicity, including vomiting, nausea,
headache, somnolence, confusion, or sleepiness, are present in the
absence of high ammonia or other intercurrent illnesses.
- Reduced Phenylbutyrate Absorption in Pancreatic Insufficiency
or Intestinal Malabsorption: Low or absent pancreatic enzymes
or intestinal disease resulting in fat malabsorption may result in
reduced or absent digestion of RAVICTI and/or absorption of
phenylbutyrate and reduced control of plasma ammonia. Monitor
ammonia levels closely.
USE IN SPECIFIC POPULATIONS
- Pregnancy: RAVICTI should be used with caution in patients
who are pregnant or planning to become pregnant. Based on animal
data, RAVICTI may cause fetal harm. A voluntary patient registry
will include evaluation of pregnancy outcomes in patients with
UCDs. For more information regarding the registry program,
visit www.ucdregistry.com or call 1-855-823-2595.
- Nursing Mothers: Caution should be exercised when
administering RAVICTI to nursing mothers, as breastfeeding is not
recommended with maternal use of RAVICTI. It is not known whether
RAVICTI or its metabolites are present in breast milk.
ADVERSE
REACTIONS
- Adverse reactions occurring in ≥10% of adult patients during
short-term treatment (n=44, 4 weeks) with RAVICTI were diarrhea,
flatulence, and headache.
- Adverse reactions occurring in ≥10% of adult patients during
long-term treatment (n=51, 12 months) with RAVICTI were nausea,
vomiting, diarrhea, decreased appetite, hyperammonemia, dizziness,
headache, and fatigue.
- Adverse reactions occurring in ≥10% of pediatric patients
during long-term treatment (n=26, 12 months) with RAVICTI were
upper abdominal pain, rash, nausea, vomiting, diarrhea, decreased
appetite, hyperammonemia, and headache.
DRUG INTERACTIONS
- Corticosteroids, valproic acid, or haloperidol: May increase
plasma ammonia level. Monitor ammonia levels closely.
- Probenecid: May affect renal excretion of metabolites of
RAVICTI, including PAGN and PAA.
You are encouraged to report negative side effects of
prescription drugs to the FDA. Visit www.fda.gov/medwatch or
call 1-800-FDA-1088.
Please see the Full Prescribing
Information and Medication Guide for RAVICTI.
About Horizon Pharma plcHorizon 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.
References:
- Ah Mew N, Lanpher BC, Gropman A, et al.; Urea Cycle Disorders
Consortium. Urea Cycle Disorders Overview. 2003 Apr 29 [Updated
2015 Apr 9]. In: Pagon RA, Adam MP, Ardinger HH, et al., editors.
GeneReviews® [Internet]. Seattle (WA): University of Washington,
Seattle; 1993-2017. Available from:
https://www.ncbi.nlm.nih.gov/books/NBK1217/
Contacts:
Tina Ventura
Senior Vice President, 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
Horizon Therapeutics Pub... (NASDAQ:HZNP)
Historical Stock Chart
From Mar 2024 to Apr 2024
Horizon Therapeutics Pub... (NASDAQ:HZNP)
Historical Stock Chart
From Apr 2023 to Apr 2024