Expanded Indication in the U.S. Now Includes
Children of all ages with Achondroplasia
SAN
RAFAEL, Calif., Oct. 20,
2023 /PRNewswire/ -- BioMarin Pharmaceutical
Inc. (Nasdaq: BMRN), a global biotechnology company dedicated to
transforming lives through genetic discovery, today announced that
the U.S. Food and Drug Administration (FDA) has approved the
supplemental New Drug Application (sNDA) for VOXZOGO®
(vosoritide) to increase linear growth in pediatric patients with
achondroplasia with open epiphyses (growth plates). This indication
is approved under accelerated approval based on an improvement in
annualized growth velocity. Previously, VOXZOGO was indicated for
children who were 5 years of age and older. This expanded
indication now includes children of all ages with open growth
plates.
"We are pleased that VOXZOGO is now available for children of
all ages with achondroplasia," said Hank
Fuchs, M.D., president of Worldwide Research and Development
at BioMarin. "We are grateful for the collaboration of the
achondroplasia community, physicians, and the children and their
families who have played a crucial role in advancing this clinical
program. We also look forward to further understanding the
potential role of VOXZOGO in other genetic short stature
conditions, including hypochondroplasia."
"VOXZOGO is the only approved treatment for children with
achondroplasia. Until now, it has only been approved in the U.S.
for children aged 5 and older with open growth plates," said
William Wilcox, M.D., professor of
human genetics at Emory University. "I
am delighted that VOXZOGO is now approved for younger children
where we hope to have potentially greater impact by starting
treatment earlier and, as a result, a much longer treatment
window."
BioMarin conducted a randomized, double-blind,
placebo-controlled Phase 2 clinical trial evaluating the safety and
efficacy of VOXZOGO in children aged 5 and under (Study 111-206).
Based on the results of this trial, together with evidence from the
adequate and well controlled Phase 3 study in pediatric patients
aged 5 years and older (Study 111-301), safety and effectiveness of
VOXZOGO have been established in pediatric patients of all ages for
the improvement in linear growth in children with achondroplasia
with open epiphyses. The overall safety profile of VOXZOGO in
children under 5 years of age was similar to that seen in older
children.
Data from an open-label, long-term Phase 2 extension study was
recently presented at the 2023 European Society for Paediatric
Endocrinology Meeting in September. Over a four-year period,
children aged 2 years and above who received VOXZOGO exhibited a
mean (average) height Z-score improvement of 1.1 to 1.4 standard
deviations (95% CI limits from 0.46 to 1.93) and a mean height gain
of 6.3 to 7.8 centimeters (cm) (95% CI limits from 2.98 to 10.40
cm) when compared to untreated children with achondroplasia of the
same age and sex. In addition, children under the age of 2 years,
treated with VOXZOGO for three years, had a mean height Z-score
improvement of 0.8 to 1.0 standard deviations (95% CI limits from
0.37 to 1.59) and a height gain between 3.5 and 3.9 cm (95% CI
limits from 1.57 to 6.16 cm).
Since the introduction of VOXZOGO in 2021, the company has seen
strong patient demand for the medicine worldwide. BioMarin has
recently been able to secure increased fill-finish commitments in
2024 and beyond to meet this additional demand. There are
approximately 800 children under 5 with achondroplasia in the
U.S.
VOXZOGO is currently approved in Europe in children with achondroplasia who are
2 years of age and older with open growth plates. In September, 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 are not closed. A final approval decision,
typically consistent with the CHMP recommendation, is expected from
the European Commission later this year.
VOXZOGO is also approved in Japan in children from birth who have
achondroplasia with open growth plates. In addition, it is approved
in Brazil in children who are 6
months and older with open growth plates as well as in
Australia in children with
achondroplasia who are 2 years of age and older with open growth
plates.
Orphan Drug Designation in Hypochondroplasia
VOXZOGO also recently received orphan drug designation from the
FDA for the treatment of hypochondroplasia, a genetic condition
caused by a mutation in the fibroblast growth factor receptor 3
(FGFR3) gene and characterized by impaired bone growth. While
similar to achondroplasia, people with hypochondroplasia
typically present with milder disproportionality and less severe
short stature compared to achondroplasia.
BioMarin plans to initiate a pivotal development program in
hypochondroplasia later this year.
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 FGFR3. VOXZOGO, a
C-type natriuretic peptide (CNP) analog, acts as a positive
regulator of the signaling pathway downstream of FGFR3 to promote
endochondral bone growth.
VOXZOGO is approved in the U.S. and indicated to increase linear
growth in children with achondroplasia with open epiphyses. This
indication 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 trial(s). To fulfill this post-marketing
requirement, BioMarin intends to use the ongoing open-label
extension studies compared to available natural history.
Patient Support Accessing VOXZOGO
To reach a BioMarin RareConnections® Case Manager,
please call, toll-free, 1-833-VOXZOGO (1-833-869-9646) or e-mail
VOXZOGOSupport@biomarin-rareconnections.com. For more information
about VOXZOGO, please visit www.voxzogo.com. For additional
information regarding this product, please contact BioMarin Medical
Information at medinfo@bmrn.com.
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 FGFR3 gene, 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 and open growth plates
(epiphyses).
- 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), high levels of blood
alkaline phosphatase shown in blood tests, 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 potential
impact and role of VOXZOGO in children with achondroplasia,
including younger children, as well as in the treatment of
hypochondroplasia and other genetic short stature conditions; the
development of BioMarin's VOXZOGO program, including 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; BioMarin's
plans for the continued clinical development of VOXZOGO, including
ability to maintain continued approval in the U.S. based on ongoing
label-extension studies and confirmatory trials; BioMarin's plans
to initiate a pivotal development program in hypochondroplasia; and
BioMarin's expectations regarding the number of children with
achondroplasia that could be prescribed with VOXZOGO and related
potential benefits. 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 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
|
Media
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Traci
McCarty
|
Andrew Villani
|
BioMarin
Pharmaceutical Inc.
|
BioMarin
Pharmaceutical Inc.
|
(415)
455-7558
|
(628)
269-7393
|
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SOURCE BioMarin Pharmaceutical Inc.