SOUTH PLAINFIELD, N.J.,
Jan. 9, 2017 /PRNewswire/ -- PTC
Therapeutics, Inc. (NASDAQ: PTCT) announced that it received
notification today from the European Commission (EC) of its
adoption of a positive decision granting annual renewal of the
conditional marketing authorization for Translarna™
(ataluren). The positive decision is based on the recent renewal
recommendation by the Committee for Medicinal Products for Human
Use (CHMP) of the European Medicines Agency (EMA). The
authorization renewal allows the Company to market Translarna for
the treatment of nonsense mutation Duchenne muscular dystrophy
(nmDMD) in ambulatory patients aged five years and older in the 28
countries that are Member States of the European Union, as well as
European Economic Area members Iceland, Liechtenstein and Norway. As a specific obligation of the
renewal, the Company will conduct an additional trial of Translarna
in nmDMD.
"As we continue towards our goal of providing Translarna to all
who may benefit, we are pleased by the EC ratification of the
recent CHMP positive opinion," said Stuart
W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics.
"This important regulatory milestone supports the continued growth
of our sustainable ex-US business in both the European Union and
countries that reference the authorization. Duchenne is a rapidly
progressive disease and physicians need access to medicines, like
Translarna, that have the potential to slow the devastating
progression of this disorder."
About PTC Therapeutics, Inc.
PTC is a global
biopharmaceutical company focused on the discovery, development and
commercialization of orally administered, proprietary small
molecule drugs targeting an area of RNA biology we refer to as
post-transcriptional control. Post-transcriptional control
processes are the regulatory events that occur in cells during and
after a messenger RNA, or mRNA, molecule is copied from DNA through
the transcription process. PTC's internally discovered pipeline
addresses multiple therapeutic areas, including rare disorders and
oncology. PTC has discovered all of its compounds currently under
development using its proprietary technologies. PTC plans to
continue to develop these compounds both on its own and through
selective collaboration arrangements with leading pharmaceutical
and biotechnology companies. For more information on the company,
please visit our website www.ptcbio.com.
For More Information:
Investors:
Emily
Hill
+1 (908) 912-9327
ehill@ptcbio.com
Media:
Jane
Baj
+1 (908) 912-9167
jbaj@ptcbio.com
Forward Looking Statements:
All statements, other than those of historical fact, contained
in this press release, are forward-looking statements, including
statements regarding: the future expectations, plans and prospects
for PTC; the timing and outcome of PTC's regulatory process,
including with respect to PTC's plan to conduct an additional trial
of Translarna in nmDMD; our ability to continue commercial growth
in the European Union and countries that may reference the
Translarna marketing authorization in the European Economic Area;
the clinical utility and potential advantages of Translarna; PTC's
strategy, future operations, future financial position, future
revenues or projected costs; and the objectives of management.
Other forward-looking statements may be identified by the words
"will," "plan," "expect," "target," "anticipate," "believe,"
"estimate," "intend," "may," "possible," "potential," "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: PTC's ability to
maintain its marketing authorization of Translarna for the
treatment of nmDMD in the EEA, including whether the European
Medicines Agency determines in future annual renewal cycles that
the benefit-risk balance of Translarna authorization supports
renewal of such authorization; the final design of the new nmDMD
trial that PTC will undertake pursuant to the specific obligation
associated with the marketing authorization and PTC's ability to
enroll, fund and conduct such trial; the timing and outcome
of future interactions PTC has with the FDA with respect to
Translarna for the treatment of nmDMD, including PTC's ability to
resolve the matters set forth in the Refuse to File letter from the
FDA or otherwise advance Translarna for the treatment of nmDMD in
the United States (whether
pursuant to the file over protest process or otherwise), including
whether PTC is required to perform additional clinical and
non-clinical trials at significant cost and whether such trials, if
successful, may enable FDA review of a new drug application;
the outcome of ongoing or future clinical trials or studies in
Translarna, in particular ACT CF; the eligible patient base
and commercial potential of Translarna and PTC's other product
candidates; PTC's ability to commercialize and commercially
manufacture Translarna in general and specifically as a treatment
for nmDMD; the outcome of pricing and reimbursement negotiations in
those territories in which PTC is authorized to sell Translarna;
expectations for regulatory approvals, including PTC's ability to
make regulatory submissions in a timely manner (or at all), the
period during which the outcome of regulatory reviews will become
available, adverse decisions by regulatory authorities (or other
delay or deceleration of the regulatory process), and PTC's ability
to meet existing or future regulatory standards with respect to
Translarna; the sufficiency of PTC's cash resources and its ability
to obtain adequate financing in the future for its foreseeable and
unforeseeable operating expenses and capital expenditures; and the
factors discussed in the "Risk Factors" section of PTC's most
recent Quarterly Report on Form 10-Q 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
Translarna will receive full regulatory approval in any territory
or maintain its current marketing authorization in the EEA, or
prove to be commercially successful in general, or specifically
with respect to the treatment of nmDMD.
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.