Sangamo Therapeutics Receives U.S. FDA Fast Track Designation for Isaralgagene Civaparvovec for the Treatment of Fabry Disease
May 22 2023 - 8:05AM
Business Wire
Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine
company, today announced that the U.S. Food and Drug Administration
(FDA) has granted Fast Track Designation to isaralgagene
civaparvovec, or ST-920, a wholly owned gene therapy product
candidate for the treatment of Fabry disease.
Fast Track designation aims to facilitate the development and
expedite the review of new therapeutics that are intended to treat
serious or life-threatening conditions and that demonstrate the
potential to address unmet medical needs. Companies granted this
designation are given the opportunity for more frequent
interactions with the FDA. These clinical programs may also be
eligible to apply for Accelerated Approval and Priority Review if
relevant criteria are met. The FDA has previously granted ST-920
Orphan Drug Designation.
“We are thrilled with the FDA’s decision to grant Fast Track
Designation for ST-920. Fabry is a debilitating disease with
life-long impact,” said Nathalie Dubois-Stringfellow, Ph.D,
Sangamo’s Senior Vice President, Chief Development Officer. “This
decision from the FDA underscores the potential for ST-920 to
address a serious unmet need and serve as a meaningful therapeutic
option for patients with Fabry disease. We are highly encouraged by
this promising development and look forward to our expected meeting
with the FDA on Phase 3 trial design in the summer.”
ST-920 is currently being evaluated in the Phase 1/2 STAAR
study, with a total of 20 patients dosed to date. In February 2023,
Sangamo announced promising results from the STAAR study via an
oral presentation at the 19th Annual WORLDSymposium, showing
sustained, elevated expression of alpha-galactosidase A (α-Gal A)
activity in the 13 dosed patients as of the data cutoff, 78%
globotriaosylceramide (Gb3) substrate clearance at 6-months and 77%
reduction in urine podocyte loss in one of the first kidney
biopsies, and a clinically meaningful and statistically significant
increase in mean general health scores, as measured by the SF-36
General Health survey. A copy of the presentation is available in
the Presentations section of the Sangamo website. Sangamo is
currently preparing for a potential Phase 3 trial and plans to meet
with the FDA on the proposed Phase 3 study design in the summer,
with a trial start anticipated by the end of 2023, depending on
regulatory interactions.
About the STAAR Study
The Phase 1/2 STAAR study is a global open-label, single-dose,
dose-ranging, multicenter clinical study designed to evaluate the
safety and tolerability of isaralgagene civaparvovec, or ST-920, a
gene therapy product candidate in patients with Fabry disease.
Isaralgagene civaparvovec requires a one-time infusion without
preconditioning. The STAAR study is enrolling patients who are on
ERT, are ERT pseudo-naïve (defined as having been off ERT for six
or more months), or who are ERT-naïve. The U.S. Food and Drug
Administration has granted Orphan Drug and Fast Track designation
to isaralgagene civaparvovec, which has also received Orphan
Medicinal Product designation from the European Medicines
Agency.
About Fabry Disease
Fabry disease is a lysosomal storage disorder caused by
mutations in the galactosidase alpha gene (GLA), which leads to
deficient alpha-galactosidase A (α-Gal A) enzyme activity, which is
necessary for metabolizing globotriaosylceramide (Gb3). The buildup
of Gb3 in the cells can cause serious damage to vital organs,
including the kidney, heart, nerves, eyes, gut and skin. Symptoms
of Fabry disease can include decreased or absent sweat production,
heat intolerance, angiokeratoma (skin blemishes), vision problems,
kidney disease, heart failure, gastrointestinal disturbance, mood
disorders, neuropathic pain and tingling in the extremities.
About Sangamo Therapeutics
Sangamo Therapeutics is a clinical-stage biopharmaceutical
company with a robust genomic medicines pipeline. Using
ground-breaking science, including our proprietary zinc finger
genome engineering technology and manufacturing expertise, Sangamo
aims to create new genomic medicines for patients suffering from
diseases for which existing treatment options are inadequate or
currently don’t exist. To learn more, visit www.sangamo.com and
connect with us on LinkedIn and Twitter.
Sangamo Forward Looking Statements
This press release contains forward-looking statements regarding
Sangamo's current expectations. These forward-looking statements
include, without limitation, statements relating to the ability of
the Fast Track designation to facilitate the development and
expedite the review of isaralgagene civaparvovec (ST-920), the
ability to meet relevant criteria in order to be eligible to apply
for Accelerated Approval and Priority Review, the therapeutic
potential of isaralgagene civaparvovec, including its potential to
improve the lives of patients without the need for ERT and to have
a favorable effect on Fabry nephropathy, Sangamo’s expectation for,
and timelines related to, the completion of dosing in the Phase 1/2
STAAR study, plans to meet with the FDA on the proposed Phase 3
study design, the planning for and the anticipated commencement of
and dosing in a potential Phase 3 trial, depending on regulatory
interactions, and the anticipated timing thereof, and other
statements that are not historical fact. These statements are not
guarantees of future performance and are subject to risks and
uncertainties that are difficult to predict. Sangamo’s actual
results may differ materially and adversely from those expressed.
Factors that could cause actual results to differ include, but are
not limited to, risks and uncertainties related to: the uncertain
timing and unpredictable nature of clinical trials and clinical
trial results, including the risks that therapeutic effects
observed in preliminary clinical trial results, including data from
kidney biopsies, will not be durable in patients and that final
Phase 1/2 STAAR study data will not validate the potential safety
and efficacy of isaralgagene civaparvovec and that the patients
withdrawn from ERT will remain off ERT; reliance on results of
early clinical trials, such as the Phase 1/2 STAAR study, which
results are not necessarily predictive of future clinical trial
results, including the results of any Phase 3 trial of isaralgagene
civaparvovec; the research and development process, including the
enrollment, operation and results of clinical trials and the
presentation of clinical data; the effects of the COVID-19 pandemic
and the impacts of the pandemic on the global business environment,
healthcare systems and business and operations of Sangamo,
including the initiation and operation of clinical trials; the
unpredictable regulatory approval process for product candidates
across multiple regulatory authorities; the manufacturing of
products and product candidates; the commercialization of approved
products; the potential for technological developments that obviate
technologies used by Sangamo in isaralgagene civaparvovec;
Sangamo’s lack of resources to fully develop, obtain regulatory
approval for and commercialize its product candidates; and those
risks and uncertainties described in Sangamo's filings with the
U.S. Securities and Exchange Commission, or the SEC, including its
Annual Report on Form 10-K for the year ended December 31, 2022, as
supplemented by its Quarterly Report on Form 10-Q for the quarter
ended March 31, 2023, and future filings and reports that Sangamo
makes from time to time with the SEC. Forward-looking statements
contained in this announcement are made as of this date, and
Sangamo undertakes no duty to update such information except as
required under applicable law.
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Investor Relations & Media Inquiries Louise Wilkie
ir@sangamo.com media@sangamo.com
Sangamo Therapeutics (NASDAQ:SGMO)
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