Sarepta Receives Negative CHMP Re-examination Opinion for Eteplirsen
September 21 2018 - 6:30AM
-- Relying upon Scientific Advisory Group input,
Sarepta will seek further scientific advice from European Medicines
Agency on a possible path to bring eteplirsen to patients in Europe
--
Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a commercial-stage
biopharmaceutical company focused on the discovery and development
of precision genetic medicine to treat rare neuromuscular diseases,
announced that the Committee for Medicinal Products for Human Use
(CHMP) of the European Medicines Agency (EMA), has confirmed its 31
May 2018 negative opinion for a Conditional Marketing Application
for eteplirsen. Eteplirsen is designed to treat approximately 13%
of the Duchenne muscular dystrophy community who have genetic
mutations amenable to exon 51 skipping.
“While largely anticipated, we are disappointed
with the outcome of the CHMP re-examination and firmly believe that
eteplirsen should be made available to patients in Europe, as it is
in the United States,” stated Doug Ingram, president and chief
executive officer, Sarepta Therapeutics.
Mr. Ingram continued, “We were, however,
encouraged by the openness of discussion with the SAG and CHMP and
their willingness to engage on different approaches to provide
additional data to support an eventual approval in Europe. Based on
those discussions, Sarepta will work to explore a potential path
forward that balances the needs of patients and their families to
avoid lengthy and unnecessarily burdensome trials with those of
European Regulators for additional supportive data consistent with
existing European regulations. We will be seeking follow up
Scientific Advice in 2019 in order to explore the approach to bring
eteplirsen to Europe.”
The Company expects the European Commission (EC)
to adopt the CHMP opinion by year-end 2018.
About Sarepta
TherapeuticsSarepta Therapeutics is a
commercial-stage biopharmaceutical company focused on the discovery
and development of precision genetic medicine to treat rare
neuromuscular diseases. The Company is primarily focused on rapidly
advancing the development of its potentially disease-modifying
Duchenne muscular dystrophy (DMD) drug candidates. For more
information, please visit www.sarepta.com.
Forward-Looking
StatementsThis press release contains
"forward-looking statements." Any statements contained in this
press release that are not statements of historical fact may be
deemed to be forward-looking statements. Words such as "believes,"
"anticipates," "plans," "expects," "will," "intends," "potential,"
"possible" and similar expressions are intended to identify
forward-looking statements. These forward-looking statements
include statements regarding Sarepta’s plan to seek further
scientific advice from EMA on a possible path to bring eteplirsen
to patients in Europe; eteplirsen’s potential to treat
approximately 13% of the DMD community who have genetic mutations
amenable to exon 51 skipping; Sarepta’s belief that eteplirsen
should be made available to patients in Europe; CHMP’s willingness
to engage on different approaches to provide additional data to
support an eventual approval in Europe; Sarepta’s plan to work to
explore a potential path forward that balances the needs of
patients and their families to avoid lengthy and unnecessarily
burdensome trials with those of European Regulators for additional
supportive data consistent with existing European regulations;
Sarepta’s intention to seek follow up Scientific Advice in 2019 in
order to explore the approach to bring eteplirsen to Europe; and
Sarepta’s expectation that the EC will adopt the CHMP opinion by
year-end 2018.
These forward-looking statements involve risks
and uncertainties, many of which are beyond Sarepta’s control.
Known risk factors include, among others: Sarepta may not be able
to eventually obtain regulatory approval for eteplirsen, or any
other product candidates, from EMA; Sarepta may not be able to
execute on its business plans, including meeting its expectations
with respect to EXONDYS 51 sales, meeting its expected or planned
regulatory milestones and timelines, research and clinical
development plans, and bringing its product candidates to market,
for various reasons including possible limitations of Company
financial and other resources, manufacturing limitations that may
not be anticipated or resolved for in a timely manner, and
regulatory, court or agency decisions, such as decisions by the
CHMP on eteplirsen or the United States Patent and Trademark Office
with respect to patents that cover our product candidates; and
those risks identified under the heading “Risk Factors” in
Sarepta’s most recent Annual Report on Form 10-K for the year ended
December 31, 2017 and most recent Quarterly Report on Form 10-Q
filed with the Securities and Exchange Commission (SEC) as well as
other SEC filings made by the Company which you are encouraged to
review.
Any of the foregoing risks could materially and
adversely affect the Company’s business, results of operations and
the trading price of Sarepta’s common stock. For a detailed
description of risks and uncertainties Sarepta faces, you are
encouraged to review Sarepta's 2017 Annual Report on Form 10-K and
most recent Quarterly Report on Form 10-Q filed with the SEC as
well as other SEC filings made by Sarepta. We caution investors not
to place considerable reliance on the forward-looking statements
contained in this press release. Sarepta does not undertake any
obligation to publicly update its forward-looking statements based
on events or circumstances after the date hereof.
Internet Posting of Information
We routinely post information that may be important to investors in
the 'For Investors' section of our website at www.sarepta.com. We
encourage investors and potential investors to consult our website
regularly for important information about us.
Source: Sarepta Therapeutics, Inc.
Media and Investors: Sarepta Therapeutics, Inc. Ian Estepan,
617-274-4052 iestepan@sarepta.com or W2O GroupRachel Hutman,
301-801-5540rhutman@w2ogroup.com
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