Amicus Therapeutics Announces European Regulatory and Clinical Updates for AT-GAA in Pompe Disease
June 26 2018 - 4:05PM
Amicus Therapeutics (Nasdaq:FOLD), a global biotechnology company
focused on discovering, developing and delivering novel medicines
for rare metabolic diseases, announced today regulatory and
clinical advancements in its development program for AT-GAA for
Pompe disease following guidance from regulators in the European
Union as well as guidance on manufacturing and bio-comparability
from German regulatory authorities (BfArM).
Regulatory & Clinical:
During the second quarter, Amicus met with the
Scientific Advice Working Party (SAWP) within the Committee for
Medicinal Products for Human Use (CHMP) of the European Medicines
Agency (EMA). The purpose of the discussions were two-fold:
1) to gain alignment on the design of a pivotal study for full
approval for AT-GAA as well as other supplemental clinical studies
in Pompe patients; 2) to discuss whether Amicus may pursue a
pathway for AT-GAA that includes a conditional marketing approval
(CMA) application in Europe. Amicus has concluded this series
of interactions and has now received written guidance from the
SAWP.
With respect to the pivotal study, Amicus will
incorporate SAWP feedback on key elements of the nature and design
of the pivotal study. The pivotal study is planned to focus
on up to 80 ERT-switch patients with 6-minute walk as the primary
endpoint and a primary treatment period up to 12 months. Patients
enrolled in the current prospective observational study being
conducted by Amicus (STRIDE Study 003) would be entered into the
pivotal treatment study. This pivotal study is expected to commence
in 2H 2018, pending feedback from the Type C meeting with FDA which
is on track for 3Q18.
The SAWP was also supportive of studying
additional patient populations, including pediatric Pompe patients
and ERT-treatment naïve Pompe patients. Amicus expects to
include these patient populations in studies to initiate in 2019,
in addition to the pivotal study in ERT-switch patients.
With respect to a pathway for conditional
approval, while the SAWP specifically noted that the efficacy data
for AT-GAA to date appear “promising” the SAWP indicated that the
current clinical package is not sufficient for a Conditional
Marketing Authorization Application at this time. Amicus
intends to continue a dialogue on a potential pathway for
conditional approval with the EMA authorities in 2019 with further
data on both efficacy and safety to include:
- Data from up to 10 additional ERT-switch patients in a new
Cohort 4 as part of the ongoing Phase 1/2 study (data expected
in 2019)
- Presentation of longer-term clinical data out to 18-months for
the 19 original Phase 1/2 patients (data expected in 2H 2018)
- Completion of a retrospective natural history study in
approximately 100 ERT-treated Pompe patients (data expected in 2H
2018)
As the clinical data continue to develop, Amicus
will also be able to commence the Process Performance Qualification
(PPQ) runs at the 1,000 Liter (commercial scale). These PPQ
manufacturing runs will be essential for any marketing application
under any pathway in both the EU and United States.
Manufacturing
Comparability:
In a separate meeting with the German regulatory
authorities (BfArM), Amicus received scientific advice indicating
general agreement with the manufacturing strategy for ATB200,
including on the strategy to demonstrate comparability between drug
substance and drug product manufactured at the 1,000 liter scale
and drug substance and drug product manufactured at the 250 liter
scale.
John F. Crowley, Chairman and Chief Executive
Officer of Amicus Therapeutics, Inc., stated, “We continue to take
steps forward in this vitally important program. As we have
indicated previously, we also continue to believe that the evolving
regulatory path will include a series of further iterative
discussions with regulators as the program advances and as
additional data are collected. Our commitment remains the
same as it has always been since we initiated the development of
our Pompe cell line - to deliver this promising new treatment
regimen to as many people living with Pompe disease as soon as
possible. We look forward to the Type-C meeting with FDA on
this program in the third quarter and to incorporating their
feedback along with that of the CHMP to initiate the pivotal trial
this
year.”
About Scientific Advice:Scientific Advice is a
procedure offered by the EMA to stakeholders for clarification of
questions arising during development of medicinal products. The
scope of Scientific Advice is limited to scientific issues, i.e. to
quality, non-clinical and clinical aspects of the concerned
medicinal product not yet unequivocally covered by published
scientific guidelines. Scientific Advice is legally
non-binding and is based on the current scientific knowledge which
may be subject to future changes.
About AT-GAA (ATB200/AT2221)ATB200/AT2221 is a
novel treatment paradigm that consists of ATB200, a unique
recombinant human acid alpha-glucosidase (rhGAA) enzyme with
optimized carbohydrate structures, particularly mannose-6 phosphate
(M6P), to enhance uptake, co-administered with AT2221, a
pharmacological chaperone. In preclinical studies, ATB200 was
associated with increased tissue enzyme levels and reduced glycogen
levels in muscle, which was further improved when AT2221 was
co-administered with ATB200. Amicus Therapeutics is
currently conducting a global Phase 1/2 study (ATB200-02) to
evaluate the safety, tolerability, pharmacokinetics (PK) and
pharmacodynamics of ATB200/AT2221.
AT-GAA is an investigational product and is not
approved by regulatory authorities in any
jurisdiction.
About Pompe DiseasePompe disease is an
inherited lysosomal storage disorder caused by deficiency of the
enzyme acid alpha-glucosidase (GAA). Reduced or absent levels of
GAA leads to accumulation of glycogen in cells, which is believed
to result in the clinical manifestations of Pompe disease. Pompe
disease can be debilitating, and is characterized by severe muscle
weakness that worsens over time. Pompe disease ranges from a
rapidly fatal infantile form with significant impacts to heart
function to a more slowly progressive, late-onset form primarily
affecting skeletal muscle. It is estimated that Pompe disease
affects approximately 5,000 to 10,000 people worldwide.
About Amicus Therapeutics
Amicus 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. The cornerstone of the Amicus portfolio is
migalastat, an oral precision medicine for people living with Fabry
disease who have amenable genetic mutations. Migalastat is
currently approved under the trade name Galafold™ in the European
Union and Japan, with additional approvals granted and pending in
several geographies. The lead biologics program in the Amicus
pipeline is AT-GAA, a novel, late-stage, potential best-in-class
treatment paradigm for Pompe disease. The Company is committed to
advancing and expanding a robust pipeline of cutting-edge, first-
or best-in-class medicines for rare metabolic diseases.
Forward-Looking StatementsThis press release
contains "forward-looking statements" within the meaning of the
Private Securities Litigation Reform Act of 1995 relating to
clinical development of our product candidates, the timing and
reporting of results from clinical trials and the prospects and
timing of the potential regulatory approval of our product
candidates. The inclusion of forward-looking statements 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 and timing of clinical trials, 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 we may not
be successful in commercializing our product candidates if and when
approved; the potential that preclinical and clinical studies could
be delayed because we identify serious side effects or other safety
issues; the potential that we may not be able to manufacture or
supply sufficient clinical or commercial products; and the
potential that we will need additional funding to complete all of
our studies and manufacturing. Further, the results of earlier
preclinical studies and/or clinical trials may not be predictive of
future 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, 2017. 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/Media:Amicus
TherapeuticsAndrew FaughnanAssociate Director, Investor
Relationsafaughnan@amicusrx.com (609) 662-3809
Media:Pure CommunicationsJennifer Paganelli
jpaganelli@purecommunications.com (347) 658-8290
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