Zealand Pharma provides U.S. regulatory update on dasiglucagon in
congenital hyperinsulinism
Company announcement – No. 48 / 2024
Zealand Pharma provides U.S. regulatory
update on dasiglucagon in congenital hyperinsulinism
- U.S. Food and Drug Administration (FDA) issues Complete
Response Letter (CRL) for dasiglucagon in CHI for up to three weeks
of dosing due to the timing of a third-party manufacturing facility
reinspection
- The reinspection of the facility was completed in
August/September 2024 for which a new inspection classification is
pending
- Zealand remains committed to working with the FDA and the
third-party manufacturer to bring dasiglucagon to patients in the
U.S. as soon as possible
Copenhagen, Denmark, October 9,
2024 – Zealand Pharma A/S (Nasdaq: ZEAL) (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 Part 1 of the New Drug
Application (NDA) for dasiglucagon for the prevention and treatment
of hypoglycemia in pediatric patients 7 days of age and older with
congenital hyperinsulinism (CHI) for up to 3 weeks of dosing.
The CRL is related to the timing of a reinspection
at a third-party contract manufacturing facility that was completed
in August/September 2024. The third-party manufacturer has not yet
received its inspection classification following the reinspection.
A prior inspection of the facility had identified deficiencies that
did not involve dasiglucagon. These prior deficiencies had been
resolved as of this reinspection. The CRL did not state any
concerns about the clinical data package or safety of
dasiglucagon.
“We at Zealand Pharma are acutely aware of the
significant unmet medical need for newborns and children with
congenital hyperinsulinism who have either no or very limited
treatment options today,” said David Kendall, M.D., Chief Medical
Officer of Zealand Pharma. “We are committed to working with the
FDA and our third-party manufacturing partner to bring dasiglucagon
to patients living with this devastating disease in the months
ahead.”
The regulatory review of dasiglucagon is being
conducted in two parts under the same NDA. Part 1 relates to dosing
of up to 3 weeks and Part 2 relates to the use beyond 3 weeks.
Supporting the use of dasiglucagon in CHI beyond 3 weeks, the FDA
requested additional analyses from existing continuous glucose
monitoring (CGM) datasets from the Phase 3 clinical program.
Zealand expects to submit these data by the end of 2024.
About congenital
hyperinsulinism
Congenital hyperinsulinism (CHI) is a severe, ultra-rare genetic
disease, primarily affecting infants and children, in which the
pancreatic beta cells dysfunction and secrete too much insulin,
leading to frequent, recurrent, and often severe episodes of
hypoglycemia. Persistent episodes of hypoglycemia can result in
seizure, brain damage and death.1,2 It is estimated that
CHI develops in one out of 50,000 (or more) children, corresponding
to 180-300 newborns being diagnosed with the disease in the US and
Europe every year.3,4
CHI has a significant impact on patient quality of
life. Complex care requirements, including continuous intravenous
infusion of glucose, can result in lengthy and frequent
hospitalizations and make daily social activities difficult for
both patients and their families. The only currently approved
medical treatment for hyperinsulinism is diazoxide, which can be
associated with increased risk of fluid retention, pulmonary
hypertension, and congestive heart failure.5 Glucagon
and the somatostatin analog octreotide may be used but are not
approved therapies. It is estimated that more than 50% of CHI
patients do not respond adequately to the medical treatment options
currently available, so there remains a significant unmet medical
need for more and better treatment options.6
About dasiglucagon
Dasiglucagon is a glucagon receptor agonist that works by causing
the liver to release stored sugar to the blood and is being
evaluated for the prevention and treatment of hypoglycemia in
infants and children with congenital hyperinsulinism. Dasiglucagon
is designed to be administered by continuous subcutaneous infusion
using a wearable pump system. Zealand Pharma has a collaborative
development and supply agreement with DEKA Research &
Development Corporation and affiliates for the wearable
subcutaneous infusion pump system.
About Zealand Pharma
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 pre-clinical
and 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,
patient recruitment 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 at third party
manufacturers; dependency on third parties, for instance contract
research or development organizations; 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 the ongoing
military conflict in Ukraine and the uncertainty surrounding
upcoming elections in the US. 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
company announcement and are based on information available to
Zealand Pharma as of the date of this 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
Adam Lange (Investors)
Investor Relations Officer
Zealand Pharma
Email: alange@zealandpharma.com
Anna Krassowska, PhD (Investors and Media)
Vice President, Investor Relations & Corporate
Communications
Zealand Pharma
Email: akrassowska@zealandpharma.com
References
1) Thornton PS et al. (2015) Recommendations from the Pediatric
Endocrine Society for Evaluation and Management of Persistent
Hypoglycemia in Neonates, Infants, and Children, J Pediatr.
2015;167(2):238-45.
2) Banerjee I et al. (2022) Correction to: Congenital
hyperinsulinism in infancy and childhood: challenges, unmet needs
and the perspective of patients and families, Orphanet J Rare Dis.
2022;17:61.
3) Arnoux JB et al. (2011) Congenital hyperinsulinism: current
trends in diagnosis and therapy, Orphanet J Rare Dis. 2011;
6:63.
4) Yau et al. (2020) Using referral rates for genetic testing to
determine the incidence of a rare disease: The minimal incidence of
congenital hyperinsulinism in the UK is 1 in 28,389, Plos One.
2020;15(2).
5) PROGLYCEM® (diazoxide) oral suspension package insert
(07/18/2024)
https://www.accessdata.fda.gov/drugsatfda_docs/label/2024/017453s024lbl.pdf
(accessed October 7, 2024)
6) Yorifuji et al. (2017) Clinical practice guidelines for
congenital hyperinsulinism, Clin Pediatr Endocrinol.
2017;26(3):127-152.
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