European Commission Approval Decision Expected
Q4 2023
Opinion Based on Positive Results from Global
Phase 2 and Ongoing Extension Study
U.S. Food and Drug Administration PDUFA
Target Action Date for Supplemental New Drug Application for
VOXZOGO for Children Under 5 is Oct. 21,
2023
SAN RAFAEL, Calif.,
Sept. 15, 2023 /PRNewswire/ --
BioMarin Pharmaceutical Inc. (Nasdaq: BMRN), a global biotechnology
company dedicated to transforming lives through genetic discovery,
today announced that the European Medicines Agency's (EMA)
Committee for Medicinal Products for Human Use (CHMP) has adopted a
positive opinion recommending marketing authorization to expand the
indication for VOXZOGO® (vosoritide) for injection to
treat children with achondroplasia aged 4 months and older whose
epiphyses (growth plates) are not closed. The diagnosis of
achondroplasia should be confirmed by appropriate genetic testing.
A final approval decision, typically consistent with the CHMP
recommendation, is expected from the European Commission in the
fourth quarter of 2023. VOXZOGO is currently approved in
Europe in children with
achondroplasia who are 2 years of age and older with open growth
plates.
The U.S. Food and Drug Administration (FDA) has also 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 United
States to include children with achondroplasia under the age
of 5 years. VOXZOGO is currently approved in the U.S. in children
with achondroplasia who are 5 years of age and older with open
growth plates.
"The expansion of VOXZOGO to allow physicians to begin treatment
earlier offers the possibility of increased growth and other
potential important long-term benefits," said Hank Fuchs, M.D., president of Worldwide
Research and Development at BioMarin. "We look forward to the final
approval decision from the European Commission, as well as a
decision from the FDA, later this year. These approvals have the
potential to further advance care by reaching very young children
with achondroplasia."
Since the introduction of VOXZOGO in 2021, the company has
seen strong demand for the medicine worldwide. Over the last few
weeks, BioMarin has increased fill-finish commitments and is
continuing to work with the company's fill-finish provider to meet
additional demand.
VOXZOGO is the only approved treatment that addresses the
underlying cause of achondroplasia. The CHMP based its opinion on
data and outcomes from the randomized, double-blind,
placebo-controlled Phase 2 clinical trial (111-206) in children
aged 4 months to 5 years that showed an improvement in height
Z-score of approximately 0.3 standard deviations (SDS) across all
age groups after one year of treatment. The application also
included longer-term data from the ongoing extension study
(111-208).
"For the first time, very young children with achondroplasia may
have access to a treatment that has the potential to address the
root cause of the condition," said Klaus
Mohnike, professor of pediatrics at Magdeburg University
Hospital in Germany and
investigator for the VOXZOGO clinical program. "This is a
remarkable step forward in our approach to treating
achondroplasia."
VOXZOGO Safety
The safety profile was generally
consistent with that seen in older participants in the Phase 3
VOXZOGO 301 study (the current label population in the U.S.). The
most common adverse events were mild and self-limiting injection
site reactions. See the U.S. Important Safety Information below and
full Prescribing Information and the EU (European Union) Summary of
Product Characteristics for additional safety information.
Description of Phase 2 Study in Infants and
Toddlers
The 52-week phase 2 study enrolled 75 infants and
young children with achondroplasia, aged zero to less than five
years old (60 months). The study consisted of three cohorts by age
(24 months to less than 5 years, 6 months to less than 24 months,
less than 6 months) and was followed by a subsequent open-label
extension trial where all children received active treatment. The
objectives of the study were to evaluate safety, tolerability, and
the effect of VOXZOGO on growth. The study also evaluated
proportionality, functionality, quality of life, sleep apnea, and
foramen magnum dimension, as well as the advent of major illnesses
and surgeries which frequently occur in children with
achondroplasia.
About VOXZOGO® (vosoritide) for
Injection
In individuals 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 currently approved in Europe in children with achondroplasia who are
2 years of age and older with open growth plates.
VOXZOGO is also approved in the U.S. and indicated to increase
linear growth in children with achondroplasia who are 5 years of
age and older with open growth plates. 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.
U.S. FDA-Approved Indication
VOXZOGO is a C type
natriuretic peptide (CNP) analog 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).
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 the 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 development of BioMarin's VOXZOGO program,
including (i) BioMarin's expectations regarding timing and the
ability to obtain final approval decision from the European
Commission for the marketing authorization to expand the
indication for VOXZOGO, and (ii) BioMarin's expectations regarding
the PDUFA Target Action Date with respect to its sNDA for VOXZOGO
to expand treatment in the United
States to include children with achondroplasia under the age
of 5 years; the potential impact of obtaining final approvals for
expanded indications from the European Commission and the FDA,
including BioMarin's expectations regarding the number of
additional children with achondroplasia that could be prescribed
with VOXZOGO and related potential benefits; the potential benefits
of VOXZOGO for children with achondroplasia, including the duration
of such benefits and potential improvement in proportionality; 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:
|
|
Investors
|
Media
|
Traci
McCarty
|
Andrew
Villani
|
BioMarin
Pharmaceutical Inc.
|
BioMarin
Pharmaceutical Inc.
|
(415)
455-7558
|
(628)
269-7393
|
View original content to download
multimedia:https://www.prnewswire.com/news-releases/biomarin-receives-positive-chmp-opinion-in-europe-to-expand-use-of-voxzogo-vosoritide-to-treat-children-aged-4-months-and-older-with-achondroplasia-301929188.html
SOURCE BioMarin Pharmaceutical Inc.