BioMarin Enrolls First Participant in Phase 3 Trial of Vosoritide for Treatment of Children with Achondroplasia
December 12 2016 - 7:29AM
BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced today that the
company has initiated a global Phase 3 study for vosoritide, an
analog of C-type Natriuretic Peptide (CNP), in children with
achondroplasia, the most common form of dwarfism. The first child
enrolled in the study was at a site in Australia.
"Based on the positive results seen in our Phase 2 study, we
look forward to continuing to investigate vosoritide,” said Hank
Fuchs, M.D., President, Worldwide Research and Development at
BioMarin. “We are grateful to the children and their families, who
have participated in the earlier study and are participating in
this Phase 3 study.”
The Phase 3 study is a randomized, placebo-controlled study of
vosoritide in approximately 110 children with achondroplasia ages
5-14 for 52 weeks. The study will be followed by a subsequent
open-label extension. Children in this study will have completed a
minimum six-month baseline study to determine their respective
baseline growth velocity prior to entering the Phase 3 study.
Vosoritide is being tested in children whose growth plates are
still open. This is approximately 25 percent of people with
achondroplasia.
The primary endpoint of the study is the change in growth
velocity from baseline over one year in children treated compared
to placebo. The company also plans to augment the growth
velocity data with assessments of proportionality and
functionality.
In addition, BioMarin is planning a separate Phase 2 study
evaluating the effect of vosoritide in infants and toddlers.
Vosoritide has been granted orphan drug designation in both the
United States and Europe.
About Achondroplasia Achondroplasia, the most
common form of human dwarfism, is characterized by failure of
normal conversion of cartilage into bone, which results in
disproportionate short stature.
Beyond 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 invasive surgeries such as spinal
cord decompression and straightening of bowed legs. In addition,
studies show increased mortality at every age.[i], [ii]
More than 80 percent 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.[iii]
About BioMarin
BioMarin is a global biotechnology company that develops and
commercializes innovative therapies for people with serious and
life-threatening rare disorders. The company's portfolio consists
of five commercialized products and multiple clinical and
pre-clinical product candidates.
For additional information, please visit www.BMRN.com.
Information on BioMarin's 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., including,
without limitation, statements about: the development of
vosoritide; the continued clinical development of vosoritide; the
timing, design and conduct of the planned Phase 3 and Phase 2
studies; the expected results of such studies, the ability to use
the primary endpoint to support registration of vosoritide; and
actions by regulatory authorities. 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 patients 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 factors detailed in
BioMarin's filings with the Securities and Exchange Commission,
including, without limitation, the factors contained under the
caption "Risk Factors" in BioMarin's 2015 Annual Report on Form
10-K, and the factors contained in BioMarin's reports on Form 10-Q.
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® is a registered trademark of BioMarin Pharmaceutical
Inc.
[i] Hecht JT, Francomano CA, Horton WA, Annegers JF. Am J Hum
Genet. 1987; 41: 454-464.
[ii] Wynn J, King TM, Gabello MJ, Waller DK, Hecht JT. Am J
Med Genet A. 2007; 143A: 2502-2511.
[iii] Waller DK, Correa A, Vo TM, Wang Y, Hobbs C, Langlois PH,
Pearson K, Romitti PA, Shaw GM, Hecht JT. 2008. The
population-based prevalence of achondroplasia and thanatophoric
dysplasia in selected regions of the US. Am J Med Genet Part A
146A:2385–2389.
Contact:
Investors:
Traci McCarty
BioMarin Pharmaceutical Inc.
(415) 455-7558
Media:
Debra Charlesworth
BioMarin Pharmaceutical Inc.
(415) 455-7451
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