Label Expansion Decisions Expected from U.S.
and European Regulatory Authorities in Q4
SAN
RAFAEL, Calif., Sept. 21,
2023 /PRNewswire/ -- BioMarin Pharmaceutical Inc.
(Nasdaq: BMRN), a global biotechnology company dedicated to
transforming lives through genetic discovery, today announced that
new data on VOXZOGO® (vosoritide) in children with
achondroplasia will be presented at the 61st Annual European
Society for Paediatric Endocrinology (ESPE) Meeting in The Hague, Netherlands.
In an open-label, long-term Phase 2 extension study, VOXZOGO
demonstrated consistent and durable growth benefits in young
children with achondroplasia when initiated before the age of 5.
Over a four-year period, children aged 2 years and above who
received VOXZOGO exhibited a mean (average) height Z-score
improvement exceeding 1 standard deviation compared to control
groups and a height gain of more than 6 centimeters (cm) during
this time period. Additionally, children under the age of 2 years,
treated with VOXZOGO for three years, demonstrated substantial
height gains, reflected in a mean height Z-score improvement of
0.79 standard deviations compared to controls and a height gain of
more than 3 cm during this time period. Both groups of treated
children demonstrated substantial restoration of height when
compared to untreated children with achondroplasia.
VOXZOGO is currently approved in Europe in children with achondroplasia who are
2 years of age and older with open growth plates. Last week, the
European Medicines Agency's (EMA) Committee for Medicinal Products
for Human Use (CHMP) adopted a positive opinion recommending
marketing authorization to expand the indication for VOXZOGO for
injection to treat children with achondroplasia aged 4 months and
older whose epiphyses (growth plates) are not closed. VOXZOGO is
also currently approved in the United
States in children with achondroplasia who are 5 years of
age and older with open growth plates. The U.S. Food and Drug
Administration (FDA) has set a PDUFA Target Action Date of
Oct. 21, 2023, for the company's
Supplemental New Drug Application (sNDA) for VOXZOGO to expand
treatment in the U.S. to include children with achondroplasia under
5 years of age.
"The data presented add to the growing body of evidence
demonstrating the positive effect on growth in younger children
with achondroplasia who receive VOXZOGO," said Hank Fuchs, M.D., president of Worldwide
Research and Development at BioMarin. "The results showed that,
over the course of four years, young children treated earlier with
VOXZOGO exhibited substantial gains in height, suggesting that
VOXZOGO had a significant and positive impact on their growth."
New data also highlighted real-world safety and effectiveness of
VOXZOGO. Results from an early access program in France found that VOXZOGO treated under
real-world conditions had a safety and effectiveness profile
consistent with clinical trials. After 12 months of treatment with
VOXZOGO, boys demonstrated a mean (average) increase in height with
a Z-score improvement of 0.3 standard deviations, and girls showed
a mean increase in height with a Z-score improvement of 0.4
standard deviations.
Key VOXZOGO presentations at ESPE include:
Oral Presentations:
Real-world Safety and Effectiveness of Vosoritide: Results
from an Early Access Program in France
RFC4.5
Thursday, September 21, 2023, 2:45 –
2:50 p.m., Central European Summer
Time
Persistence of Growth Promoting Effects in Infants and
Toddlers with Achondroplasia: Results in Children Aged Over 2 Years
Old from a Phase II Extension Study with Vosoritide
FC4.6
Thursday, September 21, 2023, 3:45 –
3:55 p.m., Central European Summer
Time
Poster:
Design and Objectives of the Acorn Study: A
Non-Interventional Study Evaluating Long-Term Safety in
Achondroplasia Patients Treated with
Vosoritide
T8
Thursday, September 21, 2023, 12:55 –
2:25 p.m., Central European Summer
Time
About VOXZOGO (vosoritide) for Injection
In children with achondroplasia, endochondral bone growth, an
essential process by which bone tissue is created, is negatively
regulated due to a gain of function mutation in fibroblast growth
factor receptor 3 gene (FGFR3). VOXZOGO, a C-type
natriuretic peptide (CNP) analog, represents a new class of
therapy, which acts as a positive regulator of the signaling
pathway downstream of FGFR3 to promote endochondral bone
growth.
Through BioMarin's broad clinical development program, the
company has enrolled 250 children with achondroplasia from eight
countries in seven clinical studies evaluating the safety and
efficacy of VOXZOGO.
VOXZOGO is approved in the U.S. and indicated to increase linear
growth in pediatric patients with achondroplasia who are 5 years of
age and older with open epiphyses. This indication is approved
under accelerated approval based on an improvement in annualized
growth velocity. Continued approval for this indication may be
contingent upon verification and description of clinical benefit in
confirmatory trial(s). To fulfill this post-marketing requirement,
BioMarin intends to use the ongoing open-label extension studies
compared to available natural history.
About Achondroplasia
Achondroplasia, the most common form of skeletal dysplasia
leading to disproportionate short stature in humans, is
characterized by slowing of endochondral ossification, which
results in disproportionate short stature and disordered
architecture in the long bones, spine, face, and base of the skull.
This condition is caused by a change in the fibroblast growth
factor receptor 3 gene (FGFR3), a negative regulator of bone
growth.
More than 80% of children with achondroplasia have parents of
average stature and have the condition as the result of a
spontaneous gene mutation. The worldwide incidence rate of
achondroplasia is about one in 25,000 live births. VOXZOGO is being
tested in children whose growth plates are still "open," typically
those under 18 years of age. Approximately 25% of people with
achondroplasia fall into this category.
VOXZOGO U.S. Important Safety Information
What is VOXZOGO used for?
- VOXZOGO is a prescription medicine used to increase linear
growth in children with achondroplasia who are 5 years of age and
older with open growth plates (epiphyses).
- It is not known if VOXZOGO is safe and effective in children
with achondroplasia under 5 years of age.
- VOXZOGO is approved under accelerated approval based on an
improvement in annualized growth velocity. Continued approval may
be contingent upon verification and description of clinical benefit
in confirmatory trials.
What is the most important safety information about
VOXZOGO?
- VOXZOGO may cause serious side effects including a temporary
decrease in blood pressure in some patients. To reduce the risk of
a decrease in blood pressure and associated symptoms (dizziness,
feeling tired, or nausea), patients should eat a meal and drink 8
to 10 ounces of fluid within 1 hour before receiving VOXZOGO.
What are the most common side effects of VOXZOGO?
- The most common side effects of VOXZOGO include injection site
reactions (including redness, itching, swelling, bruising, rash,
hives, and injection site pain), vomiting, joint pain, decreased
blood pressure, and stomachache. These are not all the possible
side effects of VOXZOGO. Ask your healthcare provider for medical
advice about side effects, and about any side effects that bother
the patient or that do not go away.
How is VOXZOGO taken?
- VOXZOGO is taken daily as an injection given under the skin,
administered by a caregiver after a healthcare provider determines
the caregiver is able to administer VOXZOGO. Do not try to inject
VOXZOGO until you have been shown the right way by your healthcare
provider. VOXZOGO is supplied with Instructions for Use that
describe the steps for preparing, injecting, and disposing VOXZOGO.
Caregivers should review the Instructions for Use for guidance and
any time they receive a refill of VOXZOGO in case any changes have
been made.
- Inject VOXZOGO 1 time every day, at about the same time each
day. If a dose of VOXZOGO is missed, it can be given within 12
hours from the missed dose. After 12 hours, skip the missed dose
and administer the next daily dose as usual.
- The dose of VOXZOGO is based on body weight. Your healthcare
provider will adjust the dose based on changes in weight following
regular check-ups.
- Your healthcare provider will monitor the patient's growth and
tell you when to stop taking VOXZOGO if they determine the patient
is no longer able to grow. Stop administering VOXZOGO if instructed
by your healthcare provider.
What should you tell the doctor before or during taking
VOXZOGO?
- Tell your doctor about all of the patient's medical conditions
including
- If the patient has heart disease (cardiac or vascular disease),
or if the patient is on blood pressure medicine (anti-hypertensive
medicine).
- If the patient has kidney problems or renal impairment.
- If the patient is pregnant or plans to become pregnant. It is
not known if VOXZOGO will harm the unborn baby.
- If the patient is breastfeeding or plans to breastfeed. It is
not known if VOXZOGO passes into breast milk.
- Tell your doctor about all of the medicines the patient takes,
including prescription and over-the-counter medicines, vitamins,
and herbal supplements.
You may report side effects to BioMarin at 1-866-906-6100. 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 additional safety information in the full Prescribing
Information and Patient Information.
About BioMarin
Founded in 1997, BioMarin is a global biotechnology company
dedicated to transforming lives through genetic discovery. The
company develops and commercializes targeted therapies that address
the root cause of genetic conditions. BioMarin's unparalleled
research and development capabilities have resulted in eight
transformational commercial therapies for patients with rare
genetic disorders. The company's distinctive approach to drug
discovery has produced a diverse pipeline of commercial, clinical,
and pre-clinical candidates that address a significant unmet
medical need, have well-understood biology, and provide an
opportunity to be first-to-market or offer a substantial benefit
over existing treatment options. For additional information, please
visit www.biomarin.com.
Forward-Looking Statements
This press release contains forward-looking statements about the
business prospects of BioMarin Pharmaceutical Inc. (BioMarin),
including, without limitation, statements about: the data BioMarin
plans to present at ESPE, including the two oral and one poster
presentations; the development of BioMarin's VOXZOGO program,
including BioMarin's expectations regarding FDA's PDUFA Target
Action Date with respect to its sNDA for VOXZOGO to expand
treatment in the United States to
include children with achondroplasia under 5 years of age; the
potential impact and benefits of VOXZOGO for children with
achondroplasia; and the continued clinical development of VOXZOGO.
These forward-looking statements are predictions and involve risks
and uncertainties such that actual results may differ materially
from these statements. These risks and uncertainties include, among
others: results and timing of current and planned preclinical
studies and clinical trials of VOXZOGO; any potential adverse
events observed in the continuing monitoring of the patients in the
clinical trials; the content and timing of decisions by the FDA,
the EMA, the European Commission and other regulatory authorities;
and those factors detailed in BioMarin's filings with the
Securities and Exchange Commission (SEC), including, without
limitation, the factors contained under the caption "Risk Factors"
in BioMarin's Quarterly Report on Form 10-Q for the quarter ended
June 30, 2023, as such factors may be
updated by any subsequent reports. Stockholders are urged not to
place undue reliance on forward-looking statements, which speak
only as of the date hereof. BioMarin is under no obligation, and
expressly disclaims any obligation to update or alter any
forward-looking statement, whether as a result of new information,
future events or otherwise.
BioMarin® and VOXZOGO® are registered trademarks of BioMarin
Pharmaceutical Inc.
Contacts:
|
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Investors
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Media
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Traci
McCarty
|
Andrew
Villani
|
BioMarin
Pharmaceutical Inc.
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BioMarin
Pharmaceutical Inc.
|
(415)
455-7558
|
(628)
269-7393
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