SOUTH
PLAINFIELD, N.J., Jan. 25,
2024 /PRNewswire/ -- PTC Therapeutics, Inc.
(NASDAQ: PTCT) announced today that the Committee for Medicinal
Products for Human Use (CHMP) of the European Medicines Agency
(EMA) issued a negative opinion following the re-examination
procedure for the conditional marketing authorization of
Translarna™ (ataluren).
"We are disappointed that the CHMP has maintained its negative
opinion on the Translarna authorization which will result in the
withdrawal of a therapy for patients in Europe with nonsense mutation Duchenne
Muscular Dystrophy that the data support is safe and effective,"
said Matthew B. Klein, M.D., Chief
Executive Officer of PTC Therapeutics. "The CHMP's decision, which
is against the stated wishes of the patient community and expert
physicians, will be devastating for children and young men in
Europe for whom no other
disease-modifying therapies are available."
Per European regulations, the European Commission has 67 days to
adopt the opinion.
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 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-20's 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 press release, 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; the European Commission's potential adoption of
the CHMP's negative opinion for Translarna (ataluren); the clinical
utility and potential advantages of Translarna; 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 and other
regions; whether investigators agree with PTC's interpretation of
the results of clinical trials and the totality of clinical data
from PTC's 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.
View original
content:https://www.prnewswire.com/news-releases/chmp-issues-negative-opinion-for-renewal-of-conditional-marketing-authorization-for-translarna-ataluren-following-re-examination-procedure-302045154.html
SOURCE PTC Therapeutics, Inc.