Amryt Pharma PLC FDA grants Pediatric Rare Disease designation (5544W)
02 August 2018 - 4:00PM
UK Regulatory
TIDMAMYT
RNS Number : 5544W
Amryt Pharma PLC
02 August 2018
2 August 2018
AIM: AMYT
ESM: AYP
Amryt Pharma plc
("Amryt" or the "Company")
FDA grants Pediatric Rare Disease designation for AP101 for
patients with Epidermolysis Bullosa
Amryt, a biopharmaceutical company focused on rare and orphan
diseases, today announces that the US Food and Drug Administration
("FDA") has granted a Rare Pediatric Disease designation for AP101,
the Company's lead development asset, for the treatment of
Epidermolysis Bullosa ("EB").
Rare Pediatric Disease designations are granted for diseases
that predominantly affect children aged 18 or younger and which
affect a total of fewer than 200,000 people in the US.(1) The
designation means if a New Drug Application ("NDA") for AP101 is
approved, Amryt will be eligible to receive a priority review
voucher that can be used, sold or transferred.
EB is a rare genetic skin disorder that can cause skin to
blister and tear from the slightest friction or trauma and can, in
some cases, cause blistering and erosion of the epithelial lining
of internal organs. EB is chronic, potentially disfiguring and in
some cases fatal. There are approximately 500,000 people living
with EB worldwide and there are currently no approved
treatments.(2)
AP101 is currently in a Phase III clinical trial (EASE), the
largest ever global Phase III study for EB. An interim efficacy
data readout is due later this year and the top-line data readout
is expected in Q2 2019.
Joe Wiley, CEO of Amryt Pharma, commented: "We are pleased to
have been granted this Pediatric Rare Disease designation by the
FDA, which recognises the rare and serious nature of EB and its
impact on the lives of children suffering with this condition. With
the interim efficacy readout for our Phase III trial of AP101 in EB
due later this year, we look forward to continuing to demonstrate
progress with our lead development asset."
(1) https://www.fda.gov/downloads/RegulatoryInformation/Guidances/UCM423325.pdf (2) https://www.debra.org.uk/what-is-eb/what-is-eb
- Ends -
Enquiries:
Amryt Pharma plc +353 (1) 518 0200
Joe Wiley, CEO
Rory Nealon, CFO/COO
Shore Capital +44 (0) 20 7408 4090
NOMAD and Joint Broker
Edward Mansfield, Mark Percy, Daniel
Bush
Stifel +44 (0) 20 7710 7600
Joint Broker
Jonathan Senior, Ben Maddison
Davy +353 (1) 679 6363
ESM Adviser and Joint Broker
John Frain, Anthony Farrell
Consilium Strategic Communications +44 (0) 20 3709 5700
Amber Fennell, Matthew Neal, Nicholas
Brown
About Amryt
Amryt is a biopharmaceutical company focused on developing and
delivering innovative new treatments to help improve the lives of
patients with rare or orphan diseases.
Lojuxta is an approved treatment for adult patients with the
rare cholesterol disorder - Homozygous Familial
Hypercholesterolaemia ("HoFH"). This disorder 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 narrowing and blocking of blood vessels,
heart attacks and strokes, even at a very young age if not properly
diagnosed or receiving adequate treatment. Lojuxta is indicated as
an adjunct to a low-fat diet and other lipid-lowering medicinal
products with or without LDL apheresis in adult patients with
HoFH.
Amryt holds an exclusive licence to sell Lojuxta (lomitapide)
across the European Economic Area, Middle East and North Africa,
Switzerland, Turkey, Israel, Russia, the Commonwealth of
Independent States and the non-EU Balkan states.
Amryt's lead drug candidate, AP101, is a potential treatment for
Epidermolysis Bullosa ("EB"), a rare and distressing genetic skin
disorder affecting young children for which there is currently no
treatment. It is currently in Phase III clinical trials. The
European and US market opportunity for EB is estimated to be in
excess of EUR1 billion.
Amryt's earlier stage product, AP102, is focused on developing
novel, next generation somatostatin analogue ("SSA") peptide
medicines for patients with rare neuroendocrine diseases, where
there is a high unmet medical need, including Acromegaly and
Cushing's disease.
In March 2018, Amryt in-licenced a pre-clinical gene-therapy
platform technology, AP103, which offers a potential treatment for
patients with Recessive Dystrophic Epidermolysis Bullosa, a subset
of EB, and is also potentially relevant to other genetic
disorders.
For more information on Amryt, please visit amrytpharma.com
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END
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