- PTC to submit a request for re-examination
-
WARREN,
N.J., June 28, 2024 /PRNewswire/ -- PTC
Therapeutics, Inc. (NASDAQ: PTCT) announced today that the CHMP has
issued a negative opinion on the renewal of the conditional
marketing authorization of Translarna™ (ataluren) for the treatment
of nmDMD. This opinion follows the return of the previously issued
negative opinion by the European Commission (EC) for re-review.
PTC plans to request re-examination of the opinion. The
marketing authorization for Translarna remains in effect, pending
the outcome of the re-examination procedure and subsequent EC
ratification. Based on the timeline of these procedures, PTC
expects Translarna to remain on the market through the end of 2024
even if the negative opinion is maintained and ratified.
"We are disappointed that the CHMP has maintained its negative
opinion on Translarna despite the request of the European
Commission to consider the totality of evidence including the
STRIDE real world evidence in a revised opinion," said Matthew B. Klein, M.D., Chief Executive Officer,
PTC Therapeutics. "The Scientific Advisory Group convened as part
of this most recent review procedure concluded that the evidence of
significant efficacy provided by STRIDE 'should not be ignored.'
This view of the CHMP expert committee is consistent with the view
of DMD key opinion leaders worldwide. We will request
re-examination of the CHMP opinion to ensure that we make every
effort to maintain availability of Translarna on the market for as
long as possible for boys and young men with nmDMD."
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 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); the outcome of any re-examination process;
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 clinical data from
its international drug registry study and real-world evidence
concerning Translarna's benefits to support a continued marketing
authorization for Translarna for the treatment of nmDMD in the EEA;
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 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.
Acronyms:
CHMP: Committee for Medicinal
Products for Human Use
nmDMD: nonsense mutation Duchenne muscular dystrophy
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SOURCE PTC Therapeutics, Inc.