SAN RAFAEL, Calif.,
March 3, 2021 /PRNewswire/
-- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced today
that the company has completed full enrollment in a global Phase 2
randomized, placebo-controlled study of vosoritide, an
investigational, once daily injection analog of C-type Natriuretic
Peptide (CNP) for children with achondroplasia, the most common
form of disproportionate short stature in humans. The 52-week study
consists of approximately 70 infants and young children with
achondroplasia, aged zero to less than five years old (60
months). The study will be followed by a subsequent open-label
extension trial where all children receive active treatment.
Children in this study will have completed a minimum three- or
six-month baseline study to determine their respective baseline
growth prior to entering the Phase 2 study. The objectives of the
study are to evaluate safety, tolerability, and the effect of
vosoritide on growth. The company also plans to augment the
height data with assessments including proportionality,
functionality, quality of life, sleep apnea, and foramen magnum
dimension, as well as the advent of major illnesses and
surgeries.
There are currently no approved pharmacological treatments for
achondroplasia, with existing treatments mainly limited to surgical
interventions to address a variety of symptoms. This treatment gap
presents a significant unmet need. Vosoritide is an investigational
therapy that seeks to directly target the root cause of
achondroplasia by interrupting the pathway that slows bone growth
due to the causative mutation in achondroplasia. Beyond
disproportionate short stature, people with achondroplasia can
experience serious health complications, such as foramen magnum
compression, sleep apnea, and spinal stenosis. Some of these
complications can result in the need for invasive surgeries. In
addition, studies show increased mortality at every age.
"This milestone is an important building block of a
comprehensive clinical program that is methodically and responsibly
studying this potential first pharmacological treatment choice for
achondroplasia with the goal of further understanding the safety
and efficacy in the youngest children," said Hank Fuchs, M.D., President, Worldwide Research
and Development at BioMarin. "In this trial, we are studying the
effects of vosoritide during the most productive time of
growth. We are grateful to the children and families enrolled
in this placebo-controlled study and are committed to the long-term
follow up of the children in these studies."
"This is an exciting milestone for children and families,
who are interested in a treatment choice to address the
basic cause of the irregular bone growth seen in
achondroplasia. It represents a potential medical
breakthrough that would be the first non-surgical treatment
for children with achondroplasia," said John A. Phillips, III, M.D., Vanderbilt University Medical Center (David T
Karzon Professor of Pediatrics) and investigator for the vosoritide
clinical program. "As a treating physician, I see an urgent demand
from families for a treatment option that addresses bone growth and
potentially the serious complications associated with
achondroplasia, especially during infancy."
"This milestone is a giant step towards improving the quality of
medical care and options available to individuals with
achondroplasia and to their families," said Munira Shamim, co-founder of Growing
Stronger. Many families are eagerly awaiting a drug treatment
option that could possibly decrease the risk of health issues
associated with achondroplasia and increase the quality of
life. We would like to recognize the committed families and
participants in the placebo-controlled studies for
collaborating with dedicated scientists to further scientific
learning that can potentially change the lives of thousands of
families."
Regulatory Status
In 2020, the European Medicines Agency (EMA) and U.S. Food and
Drug Administration (FDA) accepted and validated the marketing
authorization application for vosoritide for achondroplasia. The
Committee for Medicinal Products for Human Use (CHMP) opinion is
expected in Europe in June of
2021. The U.S. New Drug Application (NDA) for vosoritide is under
review by the FDA with a Prescription Drug User Fee Act (PDUFA)
target action date of August 20,
2021. In the United States, the Company has chosen to provide
the 2-year outcomes from the Phase 3 extension study to the FDA as
additional data to convey the vosoritide treatment effect and
long-term durability. The Company believes that supplying this
additional data could result in a major amendment, resetting the
current PDUFA target action date out three months to
November.
In January 2021, the Company
received notice from the FDA that the NDA for vosoritide had been
granted Priority Review Designation based on the serious pediatric
indication it addresses, and the lack of treatment options
currently available. Consistent with FDA's policy on changes to
review classification for an ongoing application review, the PDUFA
action date is not affected by this designation. If approved, the
vosoritide NDA may qualify for a Priority Review Voucher
(PRV). A PRV confers priority review to a subsequent drug
application that would not otherwise qualify for that designation.
The rare pediatric disease review voucher program is designed to
encourage development of new drugs and biologics for the prevention
or treatment of rare pediatric diseases.
Upon the acceptance of the regulatory submission for vosoritide,
the Agency reiterated a position raised during the Pediatric
Advisory Committee (PAC) and Endocrinologic and Metabolic Drugs
Advisory Committee (EMDAC) held on May 11,
2018 recommending two-year controlled trials in different
age groups. BioMarin believes the highly persuasive outcomes from
the one-year randomized, double-blind, placebo-controlled Phase 3
trial, coupled with data from the Phase 2 program with up to 5
years of long-term follow-up that has been compared to robust
natural history data on growth and the updated 2-year data from the
Phase 3 study, offers a rigorous and reliable method to assess
whether vosoritide has a durable impact on the rate of endochondral
bone growth that ultimately increases final adult height.
Vosoritide has also received orphan drug designation from the
FDA and EMA for the treatment of children with achondroplasia. The
Orphan Drug Designation program is intended to advance the
evaluation and development of products that demonstrate promise for
the diagnosis and/or treatment of rare diseases or conditions.
Description of Phase 2 Study in Infants and Young Children
Ages 0 to 5 Years
This is a Phase 2 randomized, placebo-controlled study of
vosoritide. The 52-week study consists of approximately 70 infants
and young children with achondroplasia, aged zero to less than five
years old (60 months). The study will be followed by a subsequent
open-label extension trial when all subjects receive active
treatment. Children in this study will have completed a
three-to-six-month baseline study to determine their respective
baseline growth prior to entering the Phase 2 study. The primary
objectives of the study are to evaluate safety, tolerability, and
the effect of vosoritide on height. The company also plans to
augment the height data with other analyses of effects on growth
and assessments including proportionality, functionality, quality
of life, sleep apnea, and foramen magnum dimension, as well as the
advent of major illnesses and surgeries.
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. Beyond disproportionate short stature, people with
achondroplasia can experience serious health complications,
including foramen magnum compression, sleep apnea, bowed legs,
mid-face hypoplasia, permanent sway of the lower back, spinal
stenosis and recurrent ear infections. Some of these complications
can result in the need for invasive surgeries such as spinal cord
decompression and straightening of bowed legs. In addition, studies
show increased mortality at every age.
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. Vosoritide
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. In the
U.S., Europe, Latin America, the Middle East, and
most of Asia Pacific, there are currently no licensed
medicines for achondroplasia.
About BioMarin
BioMarin is a global biotechnology company that develops and
commercializes innovative therapies for patients with serious and
life-threatening rare and ultra-rare genetic diseases. The
company's portfolio consists of six commercialized products and
multiple clinical and pre-clinical product candidates. For
additional information, please visit www.biomarin.com. Information
on such website is not incorporated by reference into this press
release.
Forward-Looking Statement
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 vosoritide program generally; the potential benefits of
vosoritide for infants and young children; the continued clinical
development of vosoritide; the timing, design and conduct of the
planned Phase 2 study in infants and young children and the
expectation that topline results from this Phase 2 study will be
released in mid-2022; the timing, design and conduct of other
ongoing and possible future studies of vosoritide; the expected
results of such studies, the ability to use the primary objectives
of the Phase 2 study to support the use of vosoritide in infants
and young children; the timing of actions by regulatory authorities
including the expectation of the CHMP opinion for vosoritide in
Europe in June of 2021; the
potential for the vosoritide NDA, if approved, to qualify for a
Priority Review Voucher; and the plan to submit the second year of
Phase 3 data to the FDA and the potential that this could result in
a major amendment, resetting the current PDUFA date out three
months to November. 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
vosoritide; our ability to enroll participants into such clinical
trials, our ability to successfully manufacture vosoritide; the
content and timing of decisions by the U.S. Food and Drug
Administration, the European Commission and other regulatory
authorities concerning vosoritide; and those other risks and
uncertainties detailed from time to time under the caption "Risk
Factors" and elsewhere in the Company's Securities and Exchange
Commission (SEC) filings, including, without limitation, BioMarin's
Annual Report on Form 10-K for the year ended December 31, 2020 as such factors may be updated
by any subsequent reports. BioMarin undertakes no duty or
obligation to update any forward-looking statements contained in
this press release as a result of new information, future events or
changes in its expectations.
BioMarin® is a registered trademark of BioMarin
Pharmaceutical Inc.
Contact:
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Traci
McCarty
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Debra
Charlesworth
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BioMarin
Pharmaceutical Inc.
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BioMarin
Pharmaceutical Inc.
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(415)
455-7558
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(415)
455-7451
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SOURCE BioMarin Pharmaceutical Inc.