- European Commission decides against adoption
of negative opinion -
- Translarna authorization remains active in
Europe -
- 2024 revenue guidance paused -
WARREN,
N.J., May 20, 2024 /PRNewswire/ -- PTC
Therapeutics, Inc. (NASDAQ: PTCT) announced today that the European
Commission (EC) has decided not to adopt the CHMP's negative
opinion of January 24, 2024 on the
annual renewal of the conditional marketing authorization of
Translarna™ (ataluren) and has returned the opinion to the CHMP for
re-evaluation. As a result, Translarna remains on the market and
available for patients in Europe
consistent with its current marketing authorization. The EC has
asked the CHMP to further consider the totality of evidence,
including data from patient registries and real-world evidence, in
a revised opinion.
"The maintenance of the current authorization of Translarna is a
big win for boys and young men with nonsense mutation Duchenne
muscular dystrophy in Europe,"
said Matthew B. Klein, M.D., Chief
Executive Officer, PTC Therapeutics. "The efficacy and safety
demonstrated in clinical trials and the long-term STRIDE registry
support that Translarna fills an otherwise unmet need for nmDMD
patients. We look forward to working collaboratively with CHMP on
next steps once they are defined."
In addition, the European Medicines Agency (EMA) has informed
PTC that the decision has been taken to consider the Scientific
Advisory Group (SAG) meeting for Translarna held on September 5, 2023, and all the procedural steps
that followed, as invalid. Furthermore, the input from the SAG
meeting held in September 2023 as
well as the meeting held in January
2024 will not be considered by the CHMP in any future
evaluation of Translarna.
Given the inability to accurately forecast the upside impact of
the continued authorization of Translarna in Europe on 2024 revenue, PTC will pause 2024
total revenue guidance at this time. Guidance will be updated at a
future date.
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; PTC's future
interactions with the Committee for Medicinal Products for Human
Use (CHMP) of the European Medicines Agency (EMA); the outcome of
any regulatory procedures; 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 (EEA) and
other regions; 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 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.
Acronyms:
CHMP: Committee for Medicinal Products for
Human Use
nmDMD: nonsense mutation Duchenne muscular dystrophy
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SOURCE PTC Therapeutics, Inc.