Horizon Therapeutics plc Announces U.S. FDA Approval of New Dosage Form for PROCYSBI® (Cysteamine Bitartrate) Delayed-Releas...
February 18 2020 - 8:00AM
Business Wire
-- Oral granules in packets provide an
additional, tear-open option for people living with cystinosis
--
Horizon Therapeutics plc (Nasdaq: HZNP) announced today that the
U.S. Food and Drug Administration (FDA) has approved PROCYSBI®
(cysteamine bitartrate) delayed-release oral granules in packets
for adults and children one year of age and older living with
nephropathic cystinosis. This new dosage form provides another
option for people living with cystinosis, in addition to the
currently available PROCYSBI capsules.
Both the capsules and the packets contain the same PROCYSBI
granules, also called microbeads, that provide 12 hours of cystine
control. Providing oral granules in packets offers a tear-open
option for those living with cystinosis who may have difficulty
swallowing, need to sprinkle the granules on certain foods or
liquids, or administer medication through a gastrostomy tube
(g-tube). Families living with cystinosis are often challenged with
managing multiple, different medications on daily basis and the
oral granules in tear-open packets will provide an alternative
option for accessing PROCYSBI day-to-day.
“We would like to thank the families and health professionals in
the cystinosis community who provided feedback and guidance as the
packets were developed and helped to ensure this alternative to
opening individual capsules to remove the granules provides
meaningful benefit,” said Jeffrey D. Kent, M.D., FACG, FACP,
executive vice president, medical affairs and outcomes research,
Horizon. “Having two options will allow families and physicians to
select what is best for them and reflects our continued commitment
to invest in creating solutions for patients and families based on
their needs.”
PROCYSBI oral granules in packets in 75 mg and 300 mg strengths
are expected to be available in the first half of the year.
PROCYSBI capsules will continue to be available in 25 mg and 75 mg
strengths.
About Nephropathic Cystinosis
Nephropathic 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. If
untreated, elevated cystine accumulation 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 nephropathic cystinosis. Nephropathic or “classic
infantile” cystinosis – the most common and most severe form of the
disease – is typically diagnosed in infancy and requires lifelong
cystine depleting therapy.1
About PROCYSBI
PROCYSBI (cysteamine bitartrate) delayed-release capsules and
delayed-release oral granules is a cystine-depleting agent
indicated for the treatment of nephropathic cystinosis in adults
and pediatric patients 1 year of age and older
IMPORTANT SAFETY INFORMATION
CONTRAINDICATIONS
- Patients with serious hypersensitivity reaction, including
anaphylaxis 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.
Monitor patients for development of skin or bone lesions and reduce
PROCYSBI dosing if patients develop these lesions.
- Skin Rash: Severe skin rashes such as erythema
multiforme bullosa or toxic epidermal necrolysis have been reported
in patients receiving immediate-release cysteamine bitartrate.
Discontinue use if severe skin rash occurs.
- Gastrointestinal (GI) Ulcers and Bleeding: GI ulceration
and bleeding have been reported in patients receiving
immediate-release cysteamine bitartrate. Monitor for GI symptoms
and consider decreasing the dose if severe symptoms occur.
- Central Nervous System (CNS) Symptoms: CNS symptoms such
as seizures, lethargy, somnolence, depression, and encephalopathy
have been associated with immediate-release cysteamine. Monitor for
CNS symptoms; interrupt or reduce the dose for severe symptoms or
those that persist or progress.
- 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; decrease or discontinue the
dose 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 for signs and symptoms of PTC;
interrupt or reduce the dose for signs/symptoms that persist, or
discontinue if diagnosis is confirmed.
ADVERSE REACTIONS
The most common adverse reactions reported in PROCYSBI clinical
trials (≥ 5%): were:
- Patients 2 years of age and older previously treated with
cysteamine: vomiting, nausea, abdominal pain, headache,
conjunctivitis, influenza, gastroenteritis, nasopharyngitis,
dehydration, ear infection, upper respiratory tract infection,
fatigue, arthralgia, cough, and pain in extremity.
- Patients 1 year of age and older naïve to cysteamine treatment:
vomiting, gastroenteritis/viral gastroenteritis, diarrhea, breath
odor, nausea, electrolyte imbalance, headache.
DRUG INTERACTIONS
- Drugs that increase gastric pH may alter the pharmacokinetics
of cysteamine due to the premature release of cysteamine from
PROCYSBI and increase WBC cystine concentration. Monitor WBC
cystine concentration with concomitant use.
- 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: Because of the potential risk for serious adverse
reactions in breastfed children from cysteamine, breastfeeding is
not recommended during treatment with PROCYSBI.
Please see Full Prescribing Information.
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 potential benefits and timing of
availability of PROCYSBI® (cysteamine bitartrate) delayed-release
oral granules in packets. 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 Horizon’s ability to successfully
launch and market PROCYSBI® (cysteamine bitartrate) delayed-release
oral granules in packets and the availability of reimbursement and
payor coverage, 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
- https://ghr.nlm.nih.gov/condition/cystinosis. Accessed July 16,
2019.
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Tina Ventura Senior Vice President, Investor Relations
Investor-relations@horizontherapeutics.com
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Investor-relations@horizontherapeutics.com
U.S. Media Contact: Amanda Phraner Director,
Public Relations and Social Media media@horizontherapeutics.com
Ireland Media Contact: Gordon MRM Ray Gordon
ray@gordonmrm.ie
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