- Conference call to be held at 8:45 am
EDT -
SOUTH PLAINFIELD,
N.J., Sept. 15, 2023 /PRNewswire/ -- PTC
Therapeutics, Inc. (NASDAQ: PTCT) announced today that the
Committee for Medicinal Products for Human Use (CHMP) of the
European Medicines Agency (EMA) has given a negative opinion on the
conversion of the conditional marketing authorization to full
marketing authorization of Translarna™ (ataluren) for the treatment
of nonsense mutation Duchenne muscular dystrophy (nmDMD). The
negative opinion also applies to the renewal of the existing
conditional authorization. PTC plans to submit a request for
re-examination per EMA guidelines. Translarna will remain on the
market and available to patients with nmDMD until the
re-examination process is completed. Based on CHMP procedural
guidance, the opinion following the re-examination process would be
expected to occur in January 2024,
with EC ratification of the opinion within the following 67
days.
"We are surprised and extremely disappointed by the CHMP
decision, given the well-established and favorable safety and
efficacy profile of Translarna," said Matthew B. Klein, M.D., Chief Executive Officer,
PTC Therapeutics. "Of course, this decision is most devastating for
the hundreds of boys and young men in Europe with nonsense mutation DMD, for whom no
other approved therapies are available. We will be submitting a
request for re-examination to the CHMP to reverse this opinion, as
we have done previously in the regulatory history of Translarna in
Europe."
Translarna received conditional marketing authorization in
Europe in 2014 based on the
results of Study 007, and the conditional authorization was renewed
in 2017. As part of the renewal of the conditional marketing
authorization, PTC agreed to a specific obligation to conduct a
third placebo-controlled trial, Study 041. PTC shared the results
of Study 041, which included nominally statistically significant
results on several key endpoints in the overall enrolled
Intent-to-Treat population of 359 boys even though it did not meet
statistical significance in the primary analysis subgroup.
The data from Study 041, as well as data from the two previous
placebo-controlled trials—Studies 007 and 020 — formed the basis of
the CHMPs review and decision. Additional supportive evidence of
efficacy included robust meta-analyses demonstrating highly
statistically significant benefit on several key disease endpoints
capturing different aspects of DMD, including the Six-Minute Walk
Distance test (6MWD), North Star Ambulatory Assessment, and Timed
Function Tests. In addition, analyses of the real-world STRIDE
registry, which includes 300 boys with an average treatment
duration of over 5.5 years, demonstrating that long-term Translarna
therapy delays the time to loss of ambulation by 3.5 years were
included as part of the data package to confirm long-term
meaningful treatment benefit. In addition, Translarna has shown a
favorable safety profile, with over 3,000 patients treated to
date.
In the re-examination, PTC plans to focus on the clear and
consistent evidence of benefit recorded in the overall population
of 700 boys included in all studies (007, 020 and 041). In
addition, PTC will address the decision to designate a different
primary analysis subgroup in Study 041 than the subgroup previously
demonstrated to be most responsive to a treatment effect on 6MWD in
Studies 007 and 020. PTC will also address concerns raised
regarding the robustness of the STRIDE data in supporting long-term
treatment benefit.
Today's Conference Call
PTC will hold a conference call at 8:45 am
EDT today to discuss this news. To access the call by phone,
please click here to register and you will be provided with dial-in
details. To avoid delays, we recommend participants dial in to the
conference call 15 minutes prior to the start of the call. The
webcast conference call can be accessed on the Investor section of
the PTC website at https://ir.ptcbio.com/events-presentations. A
replay of the call will be available approximately two hours after
completion of the call and will be archived on the company's
website for 30 days following the call.
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
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 or its conversion to a full approval; PTC's
ability to complete 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 is 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 and a conversion
to a standard marketing authorization in the EEA; 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 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