Sarepta Therapeutics Receives Complete Response Letter from the US Food and Drug Administration for Golodirsen New Drug Appli...
August 19 2019 - 5:48PM
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision
genetic medicine for rare diseases, today announced it had received
a Complete Response Letter (CRL) from the U.S. Food and Drug
Administration (FDA) regarding the New Drug Application (NDA)
seeking accelerated approval of golodirsen injection for the
treatment of Duchenne muscular dystrophy (DMD) in patients with a
confirmed mutation amenable to exon 53 skipping.
The CRL generally cites two concerns: the risk
of infections related to intravenous infusion ports and renal
toxicity seen in pre-clinical models of golodirsen and observed
following administration of other antisense oligonucleotides. Renal
toxicity with golodirsen was observed in pre-clinical models at
doses that were ten-fold higher than the dose used in clinical
studies. Renal toxicity was not observed in Study 4053-101, on
which the application for golodirsen was based.
“We are very surprised to have received the
complete response letter this afternoon. Over the entire course of
its review, the Agency did not raise any issues suggesting the
non-approvability of golodirsen, including the issues that formed
the basis of the complete response letter,” said Doug Ingram,
president and chief executive officer, Sarepta. “We will work with
the Division to address the issues raised in the letter and, to the
fullest extent possible, find an expeditious pathway forward for
the approval of golodirsen. We know that the patient community is
waiting.”
Sarepta will immediately request a meeting with
the FDA to determine next steps.
The ESSENCE study (4045-301), a global,
randomized double-blind, placebo-controlled study assessing the
efficacy and safety of golodirsen and casimersen, our exon-45
skipping agent, is ongoing.
Forward-Looking StatementThis
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 our ability
to work with the Division to address the issues raised in the
letter and find an expeditious pathway forward for the approval of
golodirsen, and our immediate request for a meeting with the
FDA.
These forward-looking statements involve risks
and uncertainties, many of which are beyond Sarepta’s control.
Known risk factors include, among others: we may not be able to
obtain FDA approval of golodirsen; we may not be able to complete
clinical trials required by the FDA or other regulatory authorities
for approval of our product candidates; the results of our ongoing
research and development efforts and clinical trials for our
products and product candidates may not be positive or consistent
with prior results or demonstrate a safe treatment benefit or
support an NDA or a BLA filing, positive advisory committee
recommendation or marketing approval by the FDA or other regulatory
authority; we may not be able to execute on our business plans
including meeting our expected or planned regulatory milestones and
timelines, clinical development plans and bringing our product
candidates to market, including the commercialization of VYONDYS
53, for various reasons, including factors outside of our control,
such as possible limitations of company financial and other
resources, manufacturing limitations that may not be anticipated or
resolved for in a timely manner or at all, and regulatory, court or
agency decisions, such as decisions by 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, 2018 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 2018 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.
Sarepta Therapeutics, Inc.
Investors: Ian Estepan,
617-274-4052iestepan@sarepta.com Media:Tracy Sorrentino,
617-301-8566tsorrentino@sarepta.com
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