SOUTH
PLAINFIELD, N.J., Dec. 5, 2023
/PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced
today an update on Translarna™ (ataluren) regulatory activities in
Europe and the United States.
As planned, PTC has submitted the briefing document as part of
the re-examination of the initial Committee for Medicinal Products
for Human Use (CHMP) negative opinion on the renewal of the
conditional marketing authorization of Translarna in Europe. The re-examination procedure will
focus only on the renewal of the existing conditional marketing
authorization. The procedure is expected to last until late
January 2024, when an opinion will be
provided by CHMP. This opinion will then be sent for adoption by
the European Commission, within the following 67 days.
In the United States, PTC
recently participated in a Type C meeting with the U.S. Food and
Drug Administration (FDA) to discuss a potential path to a
resubmission of a New Drug Application (NDA) for Translarna. The
discussion focused on the totality of evidence collected to date
from the Translarna clinical trials and the STRIDE registry. Based
on the discussion, FDA suggested that PTC request a follow-up
meeting to align on the specific contents that could support a
potential NDA filing. PTC expects this meeting will occur in the
first quarter of 2024.
"PTC continues to work to secure global registrations that will
enable access to Translarna for patients with nmDMD worldwide,"
said Matthew B. Klein, M.D., Chief
Executive Officer of PTC. "The data collected to date demonstrate
the short-and long-term benefits of Translarna on key functional
aspects of disease, including ambulatory and neuromuscular
function. In Europe, we believe we
can address the concerns raised by the CHMP in its initial negative
opinion on the renewal of Translarna conditional authorization. In
the U.S., we look forward to continuing to work collaboratively
with FDA on the contents of a potential NDA resubmission."
About Translarna™ (ataluren)
Translarna (ataluren),
discovered and developed by PTC Therapeutics, is a protein
restoration therapy designed to enable the formation of a
functioning protein in patients with genetic disorders caused by a
nonsense mutation. A nonsense mutation is an alteration in the
genetic code that prematurely halts the synthesis of an essential
protein. The resulting disorder is determined by which protein
cannot be expressed in its entirety and is no longer functional,
such as dystrophin in Duchenne. Translarna, the tradename of
ataluren, is licensed in multiple countries including Great Britain, Northern Ireland and the European Economic
Area for the treatment of nonsense mutation Duchenne muscular
dystrophy (nmDMD) in ambulatory patients aged 2 years and older.
Ataluren is an investigational new drug in the United States.
About Duchenne Muscular Dystrophy (Duchenne)
Primarily
affecting males, Duchenne is a rare and fatal genetic disorder that
results in progressive muscle weakness from early childhood and
leads to premature death in the mid-20s due to heart and
respiratory failure. It is a progressive muscle disorder caused by
the lack of functional dystrophin protein. Dystrophin is critical
to the structural stability of all muscles, including skeletal,
diaphragm, and heart muscles. Patients with Duchenne can lose the
ability to walk (loss of ambulation) as early as 10 years old,
followed by loss of the use of their arms. Duchenne patients
subsequently experience life-threatening lung complications,
requiring the need for ventilation support, and heart complications
in their late teens and 20s.
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 presentation, other than statements of historic fact, are
forward-looking statements, including statements regarding: the
future expectations, plans and prospects for PTC, including with
respect to the commercialization of its products and product
candidates; PTC's plans for interactions with the European
Medicines Agency (EMA) and US Food and Drug Administration (FDA);
the outcome of any re-examination process or meetings with
regulatory authorities; the clinical utility and potential
advantages of Translarna (ataluren); 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; PTC's ability to maintain its
marketing authorization of Translarna for the treatment of nmDMD in
Brazil, Russia, the European Economic Area (EEA) and
other regions, including whether the European Medicines Agency
(EMA) determines in the re-examination process that the
benefit-risk balance of Translarna authorization supports renewal
of such authorization; PTC's ability to use the results of Study
041, a randomized, 18-month, placebo-controlled clinical trial of
Translarna for the treatment of nmDMD followed by an 18-month
open-label extension, which was a specific obligation to continued
marketing authorization in the EEA; PTC's ability to utilize
results from Study 041 to support a marketing approval for
Translarna for the treatment of nmDMD in the United States; whether investigators agree
with PTC's interpretation of the results of clinical trials and the
totality of clinical data from our trials in Translarna;
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 Quarterly Report on Form 10-Q and 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
Translarna.
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.