Horizon Pharma plc Announces Health Canada Approval of PROCYSBI™ (Cysteamine Delayed-Release Capsules) for the Treatment ...
June 19 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, and its
affiliate Horizon Therapeutics Canada, today announced that
Health Canada has issued a Notice of Compliance (NOC) for
PROCYSBI™ (cysteamine delayed-release capsules) for the
treatment of nephropathic cystinosis in adults and children 2 years
of age and older.
“For children diagnosed with nephropathic cystinosis, treatment
with cystine-depleting therapy should be started as soon as
possible to avoid the serious and potentially life-threatening
impact on tissues and organs,” said Durhane Wong-Rieger, president
and chief executive officer, Canadian Organization for Rare
Disorders (CORD). “Prior to PROCYSBI there was no approved
therapy in Canada for treating cystinosis. We are especially
pleased that Health Canada has granted very timely approval of
PROCYSBI, the delayed-release form of the therapy, which will help
assure patients avoid toxic build-up of cystine in the
cells.” PROCYSBI is the only cystine-depleting agent
approved in Canada for the treatment of nephropathic
cystinosis. It is a delayed-release form of cysteamine
bitartrate that works by continuously reducing the toxic
concentration of cystine in the cells to limit or prevent the
damage too much cystine can cause to cells, tissue and
organs.
“The Cystinosis Research Foundation is proud to have funded
every bench and early clinical trial that led to the discovery of
PROCYSBI,” said Nancy Stack, chair of the board, Cystinosis
Research Foundation. “The approval of PROCYSBI in Canada is a
milestone achievement for the Canadian cystinosis
community.”
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 is the most common
and most severe form of the disease, and is typically diagnosed in
infancy and requires lifelong therapy.1
“The Health Canada approval would not have been possible without
the dedication of the entire nephropathic cystinosis community,
particularly the people who participated in the PROCYSBI clinical
studies, their families, as well as advocacy organizations and
healthcare professionals,” said Jared Rhines, group vice
president and general manager, Canada, LATAM and APAC, Horizon
Pharma plc. “We look forward to making PROCYSBI available to
Canadians living with this extremely challenging disease.”
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.
Forward Looking StatementThis press release
contains forward-looking statements, including statements regarding
the future availability of PROCYSBI in Canada and the potential of
PROCYSBI to treat patients with nephropathic cystinosis.
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 PROCYSBI will be successfully
commercialized and sufficiently available in Canada, 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 June 16, 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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