Summit Receives Rare Pediatric Disease Designation From US FDA for Ezutromid in Treatment of Duchenne Muscular Dystrophy
September 27 2016 - 7:00AM
Summit Therapeutics plc (NASDAQ:SMMT) (AIM:SUMM), the drug
discovery and development company advancing therapies for Duchenne
muscular dystrophy (‘DMD’) and Clostridium difficile infection,
today announces it has received Rare Pediatric Disease designation
from the US Food and Drug Administration (‘FDA’) for ezutromid in
the treatment of DMD. As a utrophin modulator, ezutromid has
potential as a disease-modifying treatment for all patients with
the fatal muscle wasting disease DMD, regardless of their
underlying dystrophin gene mutation.
“Rare Pediatric Disease designation builds upon
the Fast Track and Orphan Drug designations which the FDA has
already awarded to ezutromid, recognizing a significant unmet
medical need in the treatment of DMD,” said Glyn Edwards,
Chief Executive Officer of Summit. “We plan to leverage
these regulatory advantages in the continued clinical development
of ezutromid, which is currently in a Phase 2 clinical trial called
PhaseOut DMD, to bring ezutromid to patients in need as quickly as
possible.”
The FDA defines ’Rare Pediatric Disease’ as a
disease that primarily affects individuals aged from birth to 18
years and affects fewer than 200,000 persons in the US. Under the
Rare Pediatric Disease Priority Review Voucher programme, a sponsor
who receives an approval for a drug or biologic for a ‘rare
pediatric disease’ may qualify for a voucher which can be redeemed
to receive a priority review of a subsequent marketing application
for a different product. The Priority Review Voucher is requested
at the time of the marketing application and awarded upon approval
of the product. The voucher may only be used once, but may be sold
or transferred an unlimited number of times. The Rare Pediatric
Designation follows on the recently granted Fast Track designation
by the FDA for ezutromid. The Rare Pediatric Priority Review
Voucher programme is set to expire on 1 October 2016, although the
US Congress is currently considering legislation to extend the
programme. If the programme is not renewed, the FDA will no longer
award pediatric vouchers to otherwise eligible sponsors.
About Utrophin Modulation in
DMD DMD is a progressive muscle wasting disease that
affects around 50,000 boys and young men in the developed world.
The disease is caused by different genetic faults in the gene that
encodes dystrophin, a protein that is essential for the healthy
function of all muscles. There is currently no cure for DMD and
life expectancy is into the late twenties. Utrophin protein is
functionally and structurally similar to dystrophin. In preclinical
studies, the continued expression of utrophin has a meaningful,
positive effect on muscle performance. Summit believes that
utrophin modulation has the potential to slow down or even stop the
progression of DMD, regardless of the underlying dystrophin gene
mutation. Summit also believes that utrophin modulation could
potentially be complementary to other therapeutic approaches for
DMD. The Company’s lead utrophin modulator, ezutromid, is an orally
administered, small molecule. DMD is an orphan disease, and the US
Food and Drug Administration and the European Medicines Agency have
granted Orphan Drug designation to ezutromid. Orphan drugs receive
a number of benefits including additional regulatory support and a
period of market exclusivity following approval. In addition,
ezutromid has been granted Fast Track designation and Rare
Pediatric Disease designation by the FDA.
About Summit Therapeutics
Summit is a biopharmaceutical company focused on the discovery,
development and commercialisation of novel medicines for
indications for which there are no existing or only inadequate
therapies. Summit is conducting clinical programs focused on the
genetic disease Duchenne muscular dystrophy and the infectious
disease C. difficile infection. Further information is available at
www.summitplc.com and Summit can be followed on Twitter
(@summitplc).
Forward-looking StatementsAny
statements in this press release about Summit’s future
expectations, plans and prospects, including but not limited to,
statements about the clinical and preclinical development of
Summit’s product candidates, the therapeutic potential of Summit’s
product candidates, and the timing of initiation, completion and
availability of data from clinical trials, and other statements
containing the words "anticipate," "believe," "continue," "could,"
"estimate," "expect," "intend," "may," "plan," "potential,"
"predict," "project," "should," "target," "would," and similar
expressions, constitute forward looking statements within the
meaning of The Private Securities Litigation Reform Act of 1995.
Actual results may differ materially from those indicated by such
forward-looking statements as a result of various important
factors, including: the uncertainties inherent in the initiation of
future clinical trials, availability and timing of data from
ongoing and future clinical trials and the results of such trials,
whether preliminary results from a clinical trial will be
predictive of the final results of that trial or whether results of
early clinical trials or preclinical studies will be indicative of
the results of later clinical trials, expectations for regulatory
approvals, availability of funding sufficient for Summit’s
foreseeable and unforeseeable operating expenses and capital
expenditure requirements and other factors discussed in the "Risk
Factors" section of filings that Summit makes with the Securities
and Exchange Commission including Summit’s Annual Report on Form
20-F for the fiscal year ended January 31, 2016. Accordingly
readers should not place undue reliance on forward-looking
statements or information. In addition, any forward-looking
statements included in this press release represent Summit’s views
only as of the date of this release and should not be relied upon
as representing Summit’s views as of any subsequent date. Summit
specifically disclaims any obligation to update any forward-looking
statements included in this press release.
-END-
For more information, please contact:
Summit Therapeutics
Glyn Edwards / Richard Pye (UK office)
Erik Ostrowski / Michelle Avery (US office)
Tel: +44 (0)1235 443 951
+1 617 225 4455
Cairn Financial Advisers LLP
(Nominated Adviser)
Liam Murray / Tony Rawlinson
Tel: +44 (0)20 7148 7900
N+1 Singer
(Broker)
Aubrey Powell / Jen Boorer
Tel: +44 (0)20 7496 3000
MacDougall Biomedical Communications
(US media contact)
Chris Erdman / Karen Sharma
Tel: +1 781 235 3060
cerdman@macbiocom.com
ksharma@macbiocom.com
Consilium Strategic Communications
(Financial public relations, UK)
Mary-Jane Elliott / Sue Stuart /
Jessica Hodgson / Lindsey Neville
Tel: +44 (0)20 3709 5700
summit@consilium-comms.com
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