Amicus Therapeutics Receives European Medicines Agency PRIME Designation for CLN6 Batten Disease Gene Therapy
September 24 2020 - 6:00AM
Amicus Therapeutics (Nasdaq: FOLD), a global, patient-dedicated
biotechnology company focused on discovering, developing and
delivering novel medicines for rare diseases, today announced the
European Medicines Agency (EMA) has granted Priority Medicines
(PRIME) designation to AT-GTX-501, the Company’s investigational
gene therapy for children living with variant late infantile
neuronal ceroid lipofuscinosis 6 (vLINCL6) disease, also known as
CLN6 Batten disease.
The PRIME initiative provides enhanced support
and increased interaction to developers of promising medicines with
the goal of optimizing development plans and speeding regulatory
evaluations. The goal of EMA’s PRIME is to help patients benefit as
early as possible from innovative new therapies that have
demonstrated the potential to significantly address an unmet
medical need.
The PRIME designation is based on data from the
ongoing Phase 1/2 clinical trial evaluating a single dose of
AT-GTX-501 for the treatment of children with CLN6 Batten disease.
Additional information about the trial is available at
ClinicalTrials.gov (NCT02725580).
“We are very pleased that the EMA has recognized
the potential of our CLN6 gene therapy. Based on our preliminary
clinical data, we believe AT-GTX-501 could potentially be a
transformative treatment option for children living with CLN6
Batten disease, an ultra-rare, debilitating condition that presents
in early childhood and is often associated with childhood death,”
said John F. Crowley, Chairman and Chief Executive Officer. “We
look forward to continuing to work closely with the EMA to
accelerate development of this first potential treatment option for
children living with CLN6 Batten disease.”
Additional data from the ongoing Phase 1/2
clinical study will be presented at the Child Neurology Society
Annual Meeting in October. Regulatory interactions are ongoing and
the Company expects to provide feedback on the path forward in
early 2021.
In the U.S., AT-GTX-501 previously was granted
Rare Pediatric Disease and Orphan Drug designations by the United
States Food and Drug Administration. In the EU, the Company now
holds PRIME and orphan medicinal product designations.
About
AT-GTX-501AT-GTX-501 is a novel gene therapy in
Phase 1/2 development for CLN6 Batten disease, a rare, fatal,
inherited lysosomal disorder with no approved treatment that
primarily affects the nervous system. AT-GTX-501 is dosed in a
one-time intrathecal infusion to deliver a functional copy of the
CLN6 gene to cells of the central nervous system. The therapy is
designed to address the underlying enzyme deficiency that results
in progressive cell damage and neurodevelopmental and physical
decline.
About Batten
DiseaseBatten disease is the common name for a broad class
of rare, fatal, inherited disorders of the nervous system also
known as neuronal ceroid lipofuscinoses, or NCLs. In these
disorders, a defect in a specific gene triggers a cascade of
problems that interferes with a cell’s ability to recycle certain
molecules. Each gene is called CLN (ceroid lipofuscinosis,
neuronal) and given a different number designation as its subtype.
There are 13 known forms of Batten disease often referred to as
CLN1-8; 10-14. The various types of Batten disease have similar
features and symptoms but vary in severity and age of onset.
Most forms of Batten disease/NCLs usually begin
during childhood. The clinical course often involves progressive
loss of independent adaptive skills such as mobility, feeding and
communication. Patients may also experience vision loss,
personality changes, behavioral problems, learning impairment and
seizures. Patients typically experience progressive loss of motor
function and eventually become wheelchair-bound, are then bedridden
and die prematurely.
About Amicus TherapeuticsAmicus
Therapeutics (Nasdaq: FOLD) is a global, patient-dedicated
biotechnology company focused on discovering, developing and
delivering novel high-quality medicines for people living with rare
metabolic diseases. With extraordinary patient focus, Amicus
Therapeutics is committed to advancing and expanding a robust
pipeline of cutting-edge, first- or best-in-class medicines for
rare metabolic diseases. For more information please visit the
company’s website at www.amicusrx.com, and follow on Twitter and
LinkedIn.
Forward-Looking StatementsThis
press release contains "forward-looking statements" within the
meaning of the Private Securities Litigation Reform Act of 1995
relating to preclinical and clinical development of our product
candidates, the timing and reporting of results from preclinical
studies and clinical trials and the prospects and timing of the
potential regulatory approval of our product candidates. In
particular, this press release relates to interim data from an
ongoing Phase 1/2 study to investigate intrathecal administration
of AAV-CLN6 gene therapy. The inclusion of forward-looking
statements arising from this interim data, ongoing study and
natural history preliminary data should not be regarded as a
representation by us that any of our plans will be achieved. Any or
all of the forward-looking statements in this press release may
turn out to be wrong and can be affected by inaccurate assumptions
we might make or by known or unknown risks and uncertainties. For
example, with respect to statements regarding the goals, progress,
timing, and outcomes of discussions with regulatory authorities,
and in particular the potential goals, progress, timing, and
results of preclinical studies and clinical trials, actual results
may differ materially from those set forth in this release due to
the risks and uncertainties inherent in our business, including,
without limitation: the potential that results of clinical or
preclinical studies indicate that the product candidates are unsafe
or ineffective; the potential that it may be difficult to enroll
patients in our clinical trials; the potential that regulatory
authorities, including the FDA, EMA, and PMDA, may not grant or may
delay approval for our product candidates; the potential that
preclinical and clinical studies could be delayed because we
identify serious side effects or other safety issues; and the
potential that we will need additional funding to complete all of
our studies. Further, the results of earlier preclinical studies
and/or clinical trials may not be predictive of future results. The
interim data and Phase 1/2 study discussed herein is inherently
preliminary and early in the study, derived from a limited patient
set, and later trial results with this patient set or others may
not be consistent with these preliminary results. In addition, all
forward-looking statements are subject to other risks detailed in
our Annual Report on Form 10-K for the year ended December 31, 2019
and Quarterly Report on Form 10-Q for the quarter ended June 30,
2020. You are cautioned not to place undue reliance on these
forward-looking statements, which speak only as of the date hereof.
All forward-looking statements are qualified in their entirety by
this cautionary statement, and we undertake no obligation to revise
or update this news release to reflect events or circumstances
after the date hereof.
CONTACTS:
Investors:Amicus TherapeuticsAndrew
FaughnanDirector, Investor Relationsafaughnan@amicusrx.com(609)
662-3809
Media:Amicus TherapeuticsDiana MooreHead of
Global Corporate Communicationsdmoore@amicusrx.com(609)
662-5079
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