Ultragenyx Launches Evkeeza® (evinacumab for injection) in Canada for the Treatment of Homozygous Familial Hypercholesterolemia (HoFH)
25 Setembro 2023 - 6:13PM
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical
company focused on the development and commercialization of novel
therapies for rare and ultrarare genetic diseases, today announced
the approval of Evkeeza® (evinacumab) in Canada. Evkeeza® was
approved by Health Canada as an adjunct to diet and other
low-density lipoprotein cholesterol (LDL-C) lowering therapies for
the treatment of adult and pediatric patients aged 5 years and
older with homozygous familial hypercholesterolemia (HoFH) and will
be commercially available in Canada effective 25-November-2023.
Evkeeza is a first-of-its-kind treatment for HoFH and is the
first monoclonal antibody inhibiting the angiopoietin-like 3
protein (ANGPTL3). Evkeeza is delivered via 60-minute
intravenous infusion every 4 weeks. The treatment is now available
to prescribe for patients with HoFH in Canada.
“The approval and launch of Evkeeza in Canada for the treatment
of homozygous familial hypercholesterolemia exemplifies our
commitment to bring innovative therapies for people living with
rare and ultrarare genetic diseases,” said Monty Keast, Vice
President and General Manager at Ultragenyx Canada. “We look
forward to working collaboratively with health care providers, the
HoFH patient community and payers from across the country to make
this potentially life-changing therapy accessible to people living
with this serious disease.”
“HoFH is a severe atherosclerotic disease associated with
premature cardiovascular morbidity, which most often does not
adequately respond to cholesterol-lowering treatment currently
available,” said Prof. Daniel Gaudet, Lipidologist and Director of
the Severe Lipid Disorders Unit at the Community Genetic Medicine
Center, Department of Medicine, Université de Montréal. “Our
experience in recent years in phase 2 and 3 clinical studies,
clearly demonstrates that in combination with diet and other
low-density lipoprotein-cholesterol (LDL-C) lowering therapies,
evinacumab represents an effective option to substantially lower
the levels of LDL-cholesterol and other atherogenic lipoproteins in
HoFH patients.”
About Homozygous Familial Hypercholesterolemia
(HoFH)
HoFH, also known as homozygous FH, is the most severe form of
inherited hypercholesterolemia. This ultrarare disease affects 1 in
300,000 people worldwide. There is a founder effect in the
French-Canadian population with a prevalence estimated to be 1 in
250,000 people. Homozygous familial hypercholesterolemia runs in
families and is usually passed down by both father and mother.
People with this condition have extremely high levels of
LDL-cholesterol (‘bad cholesterol’) from birth. Such high levels
can lead to heart attacks, heart valve disease, or other problems
at an early age. For more information about HoFH please go to
www.hofhdisease.ca.
About
Evkeeza® (evinacumab)
Evinacumab, the active substance in Evkeeza, attaches to a
protein in the body called ANGPTL3 and blocks its effects. ANGPTL3
is involved in controlling cholesterol levels and blocking its
effect reduces the level of cholesterol in the blood. Evkeeza
is delivered via an infusion every 4 weeks.
Evkeeza is approved by Health Canada as an adjunct to diet and
other low-density lipoprotein cholesterol (LDL-C) lowering
therapies for the treatment of adult and pediatric patients aged 5
years and older with homozygous familial hypercholesterolemia
(HoFH). The effects of Evkeeza on cardiovascular morbidity and
mortality have not been determined. Regeneron Pharmaceuticals, Inc.
(NASDAQ: REGN) discovered and developed Evkeeza, and solely
commercializes the product in the U.S. Ultragenyx is responsible
for commercialization efforts for Evkeeza in countries outside of
the U.S.
Indication
In Canada, EVKEEZA® (evinacumab for injection) is indicated as
an adjunct to diet and other low-density lipoprotein cholesterol
(LDL-C) lowering therapies for the treatment of adult and pediatric
patients aged 5 years and older with homozygous familial
hypercholesterolemia (HoFH).
The effects of Evkeeza on cardiovascular morbidity and mortality
have not been determined.
IMPORTANT SAFETY INFORMATION FOR
EVKEEZA® (evinacumab)
INFUSION
To help avoid side effects and ensure proper use, talk to your
healthcare professional before you take Evkeeza. Hypersensitivity
reactions, including anaphylaxis and infusion reactions (e.g.,
infusion site pruritus), have been reported with Evkeeza. If signs
or symptoms of serious hypersensitivity reactions occur,
discontinue treatment with Evkeeza, treat according to the standard
of care, and monitor until signs and symptoms resolve.
The most common adverse reactions to Evkeeza were
nasopharyngitis (13.7% vs. 13.0% in placebo), influenzalike illness
(7.7% vs. 5.6% in placebo), dizziness (6.0% vs. 0% in placebo),
back pain (5.1% vs. 3.7% in placebo), and nausea (5.1% vs. 1.9% in
placebo). Fatigue (15%) was identified as an adverse reaction
to Evkeeza for pediatric patients aged ≥ 5 to 11 years
only.
Who should not use Evkeeza?
Evkeeza may not be used if you are
allergic to evinacumab or to any of the ingredients in Evkeeza.
If you are pregnant, think you might be pregnant, or plan to
become pregnant, ask your healthcare professional for advice before
taking Evkeeza. Evkeeza may harm your unborn baby. Tell your
healthcare professional if you become pregnant while using Evkeeza.
For people who are able to become pregnant:
- Your healthcare professional may do
a pregnancy test before you start treatment with Evkeeza.
- You should use an effective method
of birth control during treatment and for at least 5 months
after the last dose of Evkeeza. Talk to your healthcare
professional about birth control methods that you can use during
this time.
- If you are breastfeeding or plan to
breastfeed, ask your healthcare professional for advice before you
are given Evkeeza. It is not known if Evkeeza passes into your
breast milk. You and your healthcare professional should decide if
you will receive Evkeeza or breastfeed.
You may report side effects via email to
ultragenyx@primevigilance.com.
Please see PRODUCT MONOGRAPH
INCLUDING PATIENT MEDICATION INFORMATION for more
information.
About Ultragenyx Pharmaceutical Inc.
Ultragenyx is a biopharmaceutical company committed to bringing
novel products to patients for the treatment of serious rare and
ultrarare genetic diseases. The company has built a diverse
portfolio of approved therapies and product candidates aimed at
addressing diseases with high unmet medical need and clear biology
for treatment, for which there are typically no approved therapies
treating the underlying disease.
The company is led by a management team experienced in the
development and commercialization of rare disease therapeutics.
Ultragenyx’s strategy is predicated upon time- and cost-efficient
drug development, with the goal of delivering safe and effective
therapies to patients with the utmost urgency.
For more information on Ultragenyx, please visit
ultragenyx.ca.
Contacts
Ultragenyx Pharmaceutical, Inc.MediaJeff
Blake+1-415-612-7784media@ultragenyx.com
Ultragenyx Pharmaceutical (NASDAQ:RARE)
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