- Following ratification by the European
Commission, Evrysdi will be available to treat all spinal muscular
atrophy patients, including babies from birth -
SOUTH
PLAINFIELD, N.J., July 21,
2023 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:
PTCT) today announced that the Committee for Medicinal Products for
Human Use (CHMP) has adopted a positive opinion for the extension
of the Evrysdi® (risdiplam) marketing authorization to
include infants under two months of age in the European Union (EU).
A final decision regarding the approval is expected from the
European Commission later this year.
"The CHMP opinion represents another important milestone in the
development of Evrysdi," said Matthew B.
Klein, M.D., Chief Executive Officer, PTC Therapeutics.
"Presymptomatic babies are an important patient group that deserve
access to oral, at-home administered treatments early in the
disease."
The CHMP opinion is based on the RAINBOWFISH interim analysis
(n=18), which included six babies with two or three copies of the
SMN2 gene who completed at least one year of study
assessments. Of these, 100% (6/6) were able to sit after one year
of treatment with Evrysdi, 67% (4/6) could stand and 50% (3/6)
could walk independently. All infants were alive at 12 months
without permanent ventilation.
Evrysdi was based on PTC's splicing platform. Evrysdi is
marketed by Roche and in the United
States by Genentech, a member of the Roche Group. Roche
leads the clinical development of Evrysdi as part of a
collaboration with the SMA Foundation and PTC Therapeutics.
About Spinal Muscular Atrophy (SMA)
Spinal muscular
atrophy (SMA) is a severe, progressive neuromuscular disease that
can be fatal. It affects approximately 1 in 10,000 babies and when
untreated is the leading genetic cause of infant mortality. SMA is
caused by a mutation of the survival motor neuron 1 (SMN1) gene,
which leads to a deficiency of SMN protein. This protein is found
throughout the body and is essential to the function of nerves that
control muscles and movement. Without it, nerve cells cannot
function correctly, leading to progressive muscle weakness over
time. Depending on the type of SMA, an individual's physical
strength and their ability to walk, eat or breathe can be
significantly diminished or lost.
About Evrysdi® (risdiplam)
Evrysdi is
a survival motor neuron 2 (SMN2)-directed RNA splicing modifier
designed to treat SMA caused by mutations in chromosome 5q that
lead to SMN protein deficiency. Evrysdi is designed to distribute
evenly to all parts of the body, including the central nervous
system (CNS), and it is administered daily at home in liquid form
by mouth or feeding tube.
Evrysdi is designed to treat SMA by increasing and sustaining
the production of the survival motor neuron (SMN) protein in the
central nervous system (CNS) and peripheral tissues. SMN protein is
found throughout the body and is critical for maintaining healthy
motor neurons and movement.
Evrysdi was granted PRIME designation by the European
Medicines Agency (EMA) in 2018 and Orphan Drug Designation by
the FDA in 2017. In 2021, Evrysdi was awarded Drug Discovery of the
Year by the British Pharmacological Society, as well as
the Society for Medicines Research Award for Drug
Discovery. Evrysdi is currently approved in 100 countries. Evrysdi
is marketed in the United States by Genentech, a
member of the Roche Group, and marketed in rest of world by
Roche.
About RAINBOWFISH
RAINBOWFISH (NCT03779334) is an
open-label, single-arm, multicenter study, investigating the
efficacy, safety, pharmacokinetics, and pharmacodynamics of Evrysdi
in babies (~n=25), from birth to six weeks of age (at first dose)
with genetically diagnosed SMA who are not yet presenting with
symptoms. The study is fully enrolled.
About PTC Therapeutics, Inc.
PTC is a global
biopharmaceutical company focused on the discovery, development and
commercialization of clinically differentiated medicines that
provide benefits to patients with rare disorders. PTC's ability to
innovate to identify new therapies and to globally commercialize
products is the foundation that drives investment in a robust and
diversified pipeline of transformative medicines. PTC's mission is
to provide access to best-in-class treatments for patients who have
little to no treatment options. PTC's strategy is to leverage its
strong scientific and clinical expertise and global commercial
infrastructure to bring therapies to patients. PTC believes this
allows it to maximize value for all its stakeholders. To learn more
about PTC, please visit us at www.ptcbio.com and follow us on
Facebook, Instagram, LinkedIn and Twitter at @PTCBio.
For More Information:
Investors:
Kylie
O'Keefe
+1 (908) 300-0691
kokeefe@ptcbio.com
Media:
Jeanine
Clemente
+1 (908) 912-9406
jclemente@ptcbio.com
Forward-Looking Statement
This press release contains
forward-looking statements within the meaning of The Private
Securities Litigation Reform Act of 1995. All statements contained
in this release, other than statements of historic fact, are
forward-looking statements, including statements regarding:
advancement of PTC's joint collaboration program in SMA, including
the commercialization of any products therein or royalty or
milestone payments; the future expectations, plans and prospects
for PTC, including with respect to the expected timing of clinical
trials and studies, availability of data, regulatory submissions
and responses, licensing or commercialization of its products and
products candidates and other matters; PTC's strategy, future
operations, future financial position, future revenues, projected
costs; and the objectives of management. Other forward-looking
statements may be identified by the words, "guidance", "plan,"
"anticipate," "believe," "estimate," "expect," "intend," "may,"
"target," "potential," "will," "would," "could," "should,"
"continue," and similar expressions.
PTC's actual results, performance or achievements could differ
materially from those expressed or implied by forward-looking
statements it makes as a result of a variety of risks and
uncertainties, including those related to: the outcome of pricing,
coverage and reimbursement negotiations with third party payors for
PTC's products or product candidates that PTC commercializes or may
commercialize in the future; expectations with respect to the
commercialization of Evrysdi under our SMA collaboration;
significant business effects, including the effects of industry,
market, economic, political or regulatory conditions; changes in
tax and other laws, regulations, rates and policies; the eligible
patient base and commercial potential of PTC's products and product
candidates; PTC's scientific approach and general development
progress; and the factors discussed in the "Risk Factors" section
of PTC's most recent Annual Report on Form 10-K, as well as any
updates to these risk factors filed from time to time in PTC's
other filings with the SEC. You are urged to carefully consider all
such factors.
As with any pharmaceutical under development, there are
significant risks in the development, regulatory approval and
commercialization of new products. There are no guarantees that any
product will receive or maintain regulatory approval in any
territory, or prove to be commercially successful, including
Evrysdi.
The forward-looking statements contained herein represent PTC's
views only as of the date of this press release and PTC does not
undertake or plan to update or revise any such forward-looking
statements to reflect actual results or changes in plans,
prospects, assumptions, estimates or projections, or other
circumstances occurring after the date of this press release except
as required by law.
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SOURCE PTC Therapeutics, Inc.