U.S. Food and Drug Administration issues Complete Response Letter
for the glepaglutide New Drug Application for the treatment of
short bowel syndrome
Company announcement – No. 51 / 2024
U.S. Food and Drug Administration issues Complete Response
Letter for the glepaglutide New Drug Application for the treatment
of short bowel syndrome
- The FDA concluded
that Zealand Pharma’s application did not meet the full
requirements for substantial evidence to establish the efficacy and
safety of the to-be-marketed dose of glepaglutide.
- Zealand Pharma
will continue the dialogue with the FDA to align on the path toward
obtaining regulatory approval in the U.S.
- Zealand Pharma
expects to proceed with its current plans for a European Marketing
Authorization Application submission in 2025.
Copenhagen, Denmark, December 19, 2024 –
Zealand Pharma A/S (Nasdaq: ZEAL) (“Zealand”) (CVR-no. 20045078), a
biotechnology company focused on the discovery and development of
innovative peptide-based medicines, today announced that the U.S.
Food and Drug Administration (FDA) has issued a Complete Response
Letter (CRL) for the company’s New Drug Application (NDA) for
glepaglutide, a long-acting GLP-2 analog, under development for the
treatment of adult patients with short bowel syndrome (SBS) with
intestinal failure (IF) who are dependent on parenteral
support.
The submitted NDA included a single randomized,
placebo-controlled Phase 3 registration trial, which is common for
a rare disease indication such as SBS. The trial consisted of two
active treatment arms, a once-weekly and twice-weekly dosing arm,
respectively. Treatment with glepaglutide twice-weekly demonstrated
significant and superior effects in reducing parenteral support
requirements in patients with SBS-IF compared to placebo.
Once-weekly glepaglutide treatment resulted in a reduction in
parenteral support, but did not achieve statistical significance.
In the CRL, the FDA recommended an additional clinical trial to
provide further evidence to confirm the efficacy and safety of
glepaglutide at the to-be-marketed dose.
“While we are certainly disappointed in the FDA’s decision,
we remain confident that the data showed robust and compelling
evidence of both efficacy and safety for glepaglutide treatment. We
remain firm in our belief that glepaglutide provides a significant
advance in GLP-2-based therapies for the potential treatment of SBS
patients who are dependent on parenteral
support,” said David Kendall, MD, Chief Medical Officer
of Zealand Pharma. “We are committed to working with the agency
to align on the path toward a regulatory approval, so that we can
bring glepaglutide to patients in the U.S. In parallel, we expect
to proceed with our current plans for a European Marketing
Authorization Application submission in 2025.”
Zealand Pharma expects to initiate a single Phase 3 trial in
2025 that is anticipated to support marketing authorizations for
glepaglutide in geographies outside the U.S. and the EU and provide
further confirmatory evidence for a regulatory resubmission in the
U.S.
About glepaglutide
Glepaglutide is a long-acting GLP-2 analog in development as a
potential treatment option for short bowel syndrome (SBS).
Glepaglutide is being developed as a liquid product in an
autoinjector designed for subcutaneous administration, aimed to
reduce, or eliminate, the need for parenteral support in people
living with SBS. The U.S. Food and Drug Administration (FDA) has
granted orphan drug designation for glepaglutide for the treatment
of SBS.
About the EASE clinical program
The Phase 3 program, named EASE, includes four clinical trials
evaluating the potential for glepaglutide to reduce or eliminate
the need for parenteral support in SBS patients with intestinal
failure.
EASE-1 (NCT03690206) is a randomized, double-blind Phase 3 trial
that enrolled a total of 106 SBS patients with intestinal failure
who were dependent on parenteral support for at least three days
per week. Patients were evenly randomized to receive treatment with
10 mg glepaglutide administered either once or twice weekly, or
placebo. The primary endpoint in the trial was the absolute change
in weekly parenteral support volume from baseline at 24 weeks.
In total, 102 of 106 participating patients completed EASE-1, of
which 96 continued into the ongoing two-year, long-term safety and
efficacy extension trial, EASE-2. EASE-2 (NCT03905707) is a
randomized, double-blind trial in which SBS patients continued
their assigned treatment from EASE-1 with glepaglutide 10 mg once
or twice weekly. Patients who received placebo in EASE-1 were
re-randomized to treatment with either glepaglutide 10 mg once or
twice weekly. Patients who complete EASE-2 are eligible to
participate in EASE-3 (NCT04881825), evaluating glepaglutide
administered once weekly using an auto-injector.
EASE-4 (NCT04991311) is a Phase 3b trial to assess long-term
effects of glepaglutide on intestinal fluid and energy uptake.
About short bowel syndrome
Short bowel syndrome (SBS) with intestinal failure is a complex
chronic and severe condition associated with reduced or complete
loss of intestinal function in which individuals are dependent on
receiving fluids and nutrition parenterally. While life-sustaining,
parenteral support poses significant restrictions on daily life and
carries a risk of serious and life-threatening complications such
as sepsis, blood clots, liver damage and renal impairment.
About Zealand Pharma A/S
Zealand Pharma A/S (Nasdaq: ZEAL) ("Zealand") is a biotechnology
company focused on the discovery and development of peptide-based
medicines. More than 10 drug candidates invented by Zealand have
advanced into clinical development, of which two have reached the
market and three candidates are in late-stage development. The
company has development partnerships with a number of pharma
companies as well as commercial partnerships for its marketed
products.
Zealand was founded in 1998 and is headquartered in Copenhagen,
Denmark, with a presence in the U.S. For more information about
Zealand’s business and activities, please visit
www.zealandpharma.com.
Forward-Looking Statements
This company announcement contains “forward-looking statements”, as
that term is defined in the Private Securities Litigation Reform
Act of 1995 in the United States, as amended, even though no longer
listed in the United States this is used as a definition to provide
Zealand Pharma’s expectations or forecasts of future events
regarding the research, development, and commercialization of
pharmaceutical products, the timing of the company’s clinical
trials and the reporting of data therefrom. These forward-looking
statements may be identified by words such as “aim,” “anticipate,”
“believe,” “could,” “estimate,” “expect,” “forecast,” “goal,”
“intend,” “may,” “plan,” “possible,” “potential,” “will,” “would”,
and other words and terms of similar meaning. You should not place
undue reliance on these statements, or the scientific data
presented. The reader is cautioned not to rely on these
forward-looking statements. Such forward-looking statements are
subject to risks, uncertainties and inaccurate assumptions, which
may cause actual results to differ materially from expectations set
forth herein and may cause any or all of such forward-looking
statements to be incorrect, and which include, but are not limited
to, unexpected costs or delays in clinical trials and other
development activities due to adverse safety events or otherwise;
unexpected concerns that may arise from additional data, analysis
or results obtained during clinical trials; our ability to
successfully market both new and existing products; changes in
reimbursement rules and governmental laws and related
interpretation thereof; government-mandated or market-driven price
decreases for our products; introduction of competing products;
production problems; unexpected growth in costs and expenses; our
ability to effect the strategic reorganization of our businesses in
the manner planned; failure to protect and enforce our data,
intellectual property and other proprietary rights and
uncertainties relating to intellectual property claims and
challenges; regulatory authorities may require additional
information or further studies, or may reject, fail to approve or
may delay approval of our drug candidates or expansion of product
labeling; failure to obtain regulatory approvals in other
jurisdictions; exposure to product liability and other claims;
interest rate and currency exchange rate fluctuations; unexpected
contract breaches or terminations; inflationary pressures on the
global economy; and political uncertainty, including due to the
ongoing military conflict in Ukraine. If any or all of such
forward-looking statements prove to be incorrect, our actual
results could differ materially and adversely from those
anticipated or implied by such statements. The foregoing sets forth
many, but not all, of the factors that could cause actual results
to differ from our expectations in any forward-looking statement.
All such forward-looking statements speak only as of the date of
this press release/company announcement and are based on
information available to Zealand Pharma as of the date of this
release/announcement. We do not undertake to update any of these
forward-looking statements to reflect events or circumstances that
occur after the date hereof. Information concerning pharmaceuticals
(including compounds under development) contained within this
material is not intended as advertising or medical advice.
Contacts
Neshat Ahmadi (Investors)
Investor Relations Manager
Email: neahmadi@zealandpharma.com
Adam Lange (Investors)
Investor Relations Officer
Email: alange@zealandpharma.com
Anna Krassowska, PhD (Media and Investors)
Vice President, Investor Relations & Corporate
Communications
Email: akrassowska@zealandpharma.com
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