SOUTH PLAINFIELD, N.J.,
Nov. 11, 2016 /PRNewswire/
-- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that
the Committee for Medicinal Products for Human Use (CHMP) of the
European Medicines Agency (EMA) has recommended the renewal of the
conditional marketing authorization of Translarna™ (ataluren) for
the treatment of nonsense mutation Duchenne muscular dystrophy
(nmDMD) in ambulatory patients five years and older. In connection
with the renewal, the marketing authorization will include a
specific obligation to conduct an additional long-term
post-authorization trial.
"We are pleased with this outcome which took into account all
available data for Translarna," said Stuart
W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics,
Inc. "This decision reflects the benefit that Translarna is having
for patients suffering from nonsense mutation Duchenne muscular
dystrophy."
The CHMP opinion forms the basis for a European Commission
decision on the renewal of the marketing authorization. The
European Commission generally delivers its decision within three
months.
"Translarna has shown clinically meaningful benefits for
patients," said Eugenio Mercuri,
M.D., Professor of Pediatric Neurology at the
Catholic University, Rome,
Italy. "Duchenne is a devastating disease with a progressive
loss of function. Maintaining function is of the utmost importance
to patients."
"The consistency of Translarna's benefit shown across key
endpoints is impressive for a dystrophin replacement therapy," said
Craig McDonald, M.D., Professor of
Pediatrics and Chair of the Department of Physical Medicine &
Rehabilitation at University of
California. "I am encouraged for the DMD community by the
CHMP's recommendation."
The CHMP has requested that PTC conduct a new 18-month
randomized, placebo-controlled study in patients with nonsense
mutation Duchenne muscular dystrophy, as a specific
post-authorization obligation, with results expected to be
available in the first quarter of 2021. This study will be followed
by an 18-month open-label extension period where all patients will
be switched to Translarna. PTC has proposed a trial similar in size
to ACT DMD and details of the protocol are expected to be finalized
in future interactions with the EMA. Conditional marketing
authorizations are subject to annual reassessment and renewal.
"For boys with Duchenne, every day matters and functional loss
cannot be regained. Patients need access to innovative new
therapies like Translarna," stated Filippo
Buccella, founder of the Italian Parent Project, a patient
advocacy group for Duchenne Muscular Dystrophy.
About Translarna™ (ataluren)
Translarna, discovered
and developed by PTC Therapeutics, Inc., 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 muscular dystrophy. Translarna is licensed
in the European Economic Area for the treatment of nonsense
mutation Duchenne muscular dystrophy in ambulatory patients aged
five years and older. Translarna is an investigational new drug in
the United States. The development
of Translarna has been supported by grants from Cystic Fibrosis
Foundation Therapeutics Inc. (the nonprofit affiliate of the Cystic
Fibrosis Foundation); Muscular Dystrophy Association; FDA's Office
of Orphan Products Development; National Center for Research
Resources; National Heart, Lung, and Blood Institute; and Parent
Project Muscular Dystrophy.
About Duchenne Muscular Dystrophy
Primarily affecting
males, Duchenne muscular dystrophy (DMD) is a progressive muscle
disorder caused by the lack of functional dystrophin protein.
Dystrophin is critical to the structural stability of skeletal,
diaphragm, and heart muscles. Patients with DMD, the more severe
form of the disorder, lose the ability to walk as early as age 10
and experience life-threatening lung and heart complications in
their late teens and twenties. It is estimated that a nonsense
mutation is the cause of DMD in approximately 13 percent of
patients.
About PTC Therapeutics
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 the
final determination by the European Commission with respect to
renewal of the marketing authorization in the European Economic
Area (EEA) for Translarna for the treatment of nmDMD; the final
design, enrollment, timing, conduct, cost, evaluation and results
of the clinical trial of Translarna for the treatment of nmDMD that
PTC will undertake pursuant to the specific obligation associated
with the Translarna marketing authorization (if renewal is granted
by the European Commission); the clinical utility and potential
advantages of Translarna; PTC's ability to continue to supply
Translarna to patients across Europe and in other territories; 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," "target," "anticipate,"
"believe," "estimate," "expect," "intend," "may," "potential,"
"project," "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
Commission determines to approve the renewal of such authorization
and whether the EMA 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 (following renewal) and
PTC's ability to enroll, fund and conduct such trial; the outcome
of future interactions PTC has with the FDA with respect to
Translarna for the treatment of nmDMD, 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 (NDA) submission; the EMA's
determinations with respect to PTC's variation submission which
seeks to add Translarna for the treatment of nonsense mutation
cystic fibrosis to PTC's marketing authorization in the EEA; the
scope of regulatory approvals or authorizations for Translarna (if
any), including labeling and other matters that could affect the
availability or commercial potential of Translarna; the outcome of
ongoing or future clinical trials or studies in Translarna,
including ACT CF and the Phase 2 study of Translarna for nmDMD in
pediatric patients; 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, including its
ability to establish and maintain arrangements with manufacturers,
suppliers, distributors and production and collaboration partners
on favorable terms; the outcome of pricing and reimbursement
negotiations in those territories in which PTC is authorized to
sell Translarna; whether patients and healthcare professionals may
be able to access Translarna through alternative means if pricing
and reimbursement negotiations in the applicable territory do not
have a positive outcome; 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; PTC's ability to
fulfill any additional obligations, including with respect to
further trials or studies relating to cost-effectiveness, obtaining
licenses or satisfying requirements for labor and business
practices, in the territories in which it may obtain regulatory
approval, including the United
States, EEA and other territories; PTC's scientific approach
and general development progress; the sufficiency of PTC's cash
resources and PTC's ability to obtain adequate financing in the
future for PTC's 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.