TIDMAMYT
RNS Number : 9628Y
Amryt Pharma PLC
15 September 2020
AMRYT PHARMA PLC
("Amryt" or the "Company")
AMRYT RECEIVES POSITIVE OPINION FROM EMA ON ORPHAN DRUG
DESIGNATION FOR AP103
DUBLIN, Ireland, and Boston MA, September 15 2020, Amryt
(Nasdaq: AMYT, AIM: AMYT), a global, commercial-stage
biopharmaceutical company dedicated to developing and
commercializing novel therapeutics to treat patients suffering from
serious and life-threatening rare diseases , is pleased to announce
that the European Medicines Agency ("EMA") Committee for Orphan
Medicinal Products ("COMP") has adopted a positive opinion for
orphan designation for the use of AP103 in Epidermolysis Bullosa
("EB") . AP103 is based on Amryt's gene-therapy platform technology
and offers a potential treatment for patients with Dystrophic
Epidermolysis Bullosa ("DEB"), a subset of EB.
Orphan drug designation in the EU is granted by the European
Commission within 30 days of a positive opinion being issued by the
EMA COMP. This designation provides certain regulatory and
financial incentives including but not limited to product market
exclusivity for ten years in the EU following regulatory approval.
Orphan designation is available to companies developing products
intended to treat a life-threatening or chronically debilitating
condition affecting no more than five in 10,000 persons in the
European Union, and where the treatment provides a significant
benefit to those affected by the condition or no satisfactory
treatment is available. Amryt plans to submit an orphan drug
designation request for AP103 to the US Food and Drug
Administration ("FDA") in the near future.
Dr Joe Wiley, CEO of Amryt Pharma, commented: "Receiving this
positive opinion from the EMA for our gene-therapy candidate,
AP103, is a significant development for patients suffering from
Epidermolysis Bullosa and provides additional momentum to our
development pipeline. Unlike other gene therapies that rely on
viral vectors, AP103 is based on a novel polymer-based topical
delivery platform, which we believe offers potential advantages in
the gene-therapy field and has potential to be used in other
genetic skin conditions. Alongside our recent positive top line
readout from our Phase 3 EASE trial in FILSUVEZ (R) , today's news
further illustrates the exciting clinical opportunities in Amryt's
portfolio. These milestones relating to our efforts in
Epidermolysis Bullosa are significant for all Amryt stakeholders as
we build a global Epidermolysis Bullosa franchise and become a
leader in rare and orphan diseases."
About AP103
AP103 is currently in preclinical development for the treatment
of patients with DEB, a subset of EB. AP103 is the first gene
therapy product candidate based on our novel polymer-based topical
gene-therapy delivery platform, which also has potential use for
the treatment of other rare genetic diseases. Amryt intends to
initiate clinical development of AP103 in the first half of
2022.
About Epidermolysis Bullosa
EB is a rare, chronic and distressing genetic skin disorder that
causes the skin layers and internal body linings to separate and
affects infants, children and adults. The global incidence of EB is
estimated to be approximately 1 in 20,000, which implies that there
are as many as 30,000 affected individuals in the US and over
500,000 worldwide. There are currently no approved treatments. The
global market opportunity for EB is estimated by the Company to be
in excess of $1.0 billion.
Enquiries :
Amryt Pharma plc +353 (1) 518 0200
Joe Wiley, CEO
Rory Nealon, CFO/COO
LifeSci Advisors, LLC +1 (212) 915 2564
Tim McCarthy
Consilium Strategic Communications +44 (0) 20 3709 5700
Amber Fennell, Matthew Neal, Ashley Tapp
About Amryt
Amryt is a biopharmaceutical company focused on developing and
delivering innovative new treatments to help improve the lives of
patients with rare and orphan diseases. Amryt comprises a strong
and growing portfolio of commercial and development assets.
Amryt's commercial business comprises two orphan disease
products.
Juxtapid(R)/ Lojuxta(R) (lomitapide) is approved as an adjunct
to a low-fat diet and other lipid-lowering medicinal products for
adults with the rare cholesterol disorder, Homozygous Familial
Hypercholesterolaemia ("HoFH") in the US, Canada, Columbia,
Argentina and Japan (under the trade name Juxtapid(R)) and in the
EU (under the trade name Lojuxta(R)). HoFH is a rare genetic
disorder which impairs the body's ability to remove low density
lipoprotein ("LDL") cholesterol ("bad" cholesterol) from the blood,
typically leading to abnormally high blood LDL cholesterol levels
in the body from before birth - often ten times more than people
without HoFH - and subsequent aggressive and premature
cardiovascular disease.
Myalept(R) / Myalepta(R) (metreleptin) is approved in the US
(under the trade name Myalept(R)) as an adjunct to diet as
replacement therapy to treat the complications of leptin deficiency
in patients with congenital or acquired generalized lipodystrophy
(GL) and in the EU (under the trade name Myalepta(R)) for the
treatment of leptin deficiency in patients with congenital or
acquired GL in adults and children two years of age and above and
familial or acquired partial lipodystrophy (PL) in adults and
children 12 years of age and above for whom standard treatments
have failed to achieve adequate metabolic control. Metreleptin is
also approved for lipodystrophy in Japan. Generalised and partial
lipodystrophy are rare disorders characterised by loss or lack of
adipose tissue resulting in the deficiency of the hormone leptin,
produced by fat cells and are associated with severe metabolic
abnormalities including severe insulin resistance, diabetes,
hypertriglyceridemia and fatty liver disease.
Amryt's lead development candidate, FILSUVEZ (R) is a potential
treatment for the cutaneous manifestations of EB, a rare and
distressing genetic skin disorder affecting young children and
adults for which there is currently no approved treatment. In
September 2020, Amryt reported positive top line results from its
pivotal global phase 3 trial of FILSUVEZ(R) in EB. The primary
endpoint of the trial was met (p-value = 0.013). FILSUVEZ (R) has
been granted Rare Pediatric Disease Designation and has also
received a Fast Track Designation from the U.S. Food and Drug
Administration. The global market opportunity for EB is estimated
by the Company to be in excess of $1.0 billion.
In March 2018, Amryt in-licenced a preclinical gene-therapy
platform technology, AP103, which offers a potential treatment for
patients with Dystrophic Epidermolysis Bullosa, a subset of EB, and
is also potentially relevant to other genetic disorders.
For more information on Amryt, including products, please visit
www.amrytpharma.com .
This announcement contains inside information for the purposes
of article 7 of the Market Abuse Regulation (EU) 596/2014.
The person making this notification on behalf of Amryt is Rory
Nealon, CFO/COO and Company Secretary.
Financial Advisors
Shore Capital ( Edward Mansfield, Daniel Bush, John More) are
NOMAD and Joint Broker to Amryt in the UK. Stifel (Ben Maddison)
are Joint Broker to the company in the UK. Davy (John Frain, Daragh
O'Reilly) act as Joint Broker to the company.
Forward-Looking Statements
Statements in this announcement with respect to Amryt's
business, strategies, timing for completion of and announcing
results from the EASE trial, the potential impact of closing
enrollment in the EASE trial, as well as other statements that are
not historical facts are forward-looking statements involving risks
and uncertainties which could cause the actual results to differ
materially from such statements. Statements containing the words
"expect", "anticipate", "intends", "plan", "estimate", "aim",
"forecast", "project" and similar expressions (or their negative)
identify certain of these forward-looking statements. The
forward-looking statements in this announcement are based on
numerous assumptions and Amryt's present and future business
strategies and the environment in which Amryt expects to operate in
the future. Forward-looking statements involve inherent known and
unknown risks, uncertainties and contingencies because they relate
to events and depend on circumstances that may or may not occur in
the future and may cause the actual results, performance or
achievements to be materially different from those expressed or
implied by such forward-looking statements. These statements are
not guarantees of future performance or the ability to identify and
consummate investments. Many of these risks and uncertainties
relate to factors that are beyond each of Amryt's ability to
control or estimate precisely, such as future market conditions,
the course of the COVID-19 pandemic, currency fluctuations, the
behaviour of other market participants, the outcome of clinical
trials, the actions of regulators and other factors such as Amryt's
ability to obtain financing, changes in the political, social and
regulatory framework in which Amryt operates or in economic,
technological or consumer trends or conditions. Past performance
should not be taken as an indication or guarantee of future
results, and no representation or warranty, express or implied, is
made regarding future performance. No person is under any
obligation to update or keep current the information contained in
this announcement or to provide the recipient of it with access to
any additional relevant information that may arise in connection
with it. Such forward-looking statements reflect the Company's
current beliefs and assumptions and are based on information
currently available to management.
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END
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