U.S. Food and Drug Administration issues Complete Response Letter for dasiglucagon in congenital hyperinsulinism for up to three weeks of dosing due to inspection findings at third-party manufacturing facility
23 December 2023 - 8:47PM
U.S. Food and Drug Administration issues Complete Response Letter
for dasiglucagon in congenital hyperinsulinism for up to three
weeks of dosing due to inspection findings at third-party
manufacturing facility
Company announcement – No. 45 / 2023
U.S. Food and Drug Administration issues
Complete Response Letter for dasiglucagon in congenital
hyperinsulinism for up to three weeks of dosing due to inspection
findings at third-party manufacturing facility
- The CRL is related to deficiencies identified at a third-party
manufacturing facility following an inspection but are not specific
to dasiglucagon.
- The CRL did not state any concerns about the clinical data
package or safety of dasiglucagon.
Copenhagen, Denmark, December 23,
2023 – 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 deficiencies identified following an inspection
at a third-party contract manufacturing facility. These
deficiencies are not specific to dasiglucagon. The CRL did not
state any concerns about the clinical data package or safety of
dasiglucagon.
“We are committed to working closely with the FDA
and the third-party manufacturer to resolve these issues so that we
can bring dasiglucagon to patients living with this devastating
disease who are in need of new treatment options,” said David
Kendall, M.D., Chief Medical Officer of Zealand Pharma. “Based on
our understanding of the FDA’s position, we remain confident in the
potential for dasiglucagon to help children and their families
affected by congenital hyperinsulinism.”
Zealand expects to resubmit the NDA for
dasiglucagon for CHI for up to three weeks of dosing in the first
half of 2024 contingent on successful reinspection of the
third-party manufacturing facility.
The FDA granted dasiglucagon Priority Review on
August 30, 2023 for up to three weeks of dosing. The regulatory
review of dasiglucagon is planned to be 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, which Zealand still expects to submit in the first half
of 2024. CGM was included as a secondary outcome measure in the
Phase 3 clinical program.
About congenital
hyperinsulinismCongenital 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, hypertension and
acute heart failure. 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.5
About DasiglucagonDasiglucagon 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 PharmaZealand 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 statementsThis
company announcement contains forward-looking statements that
provide Zealand Pharma’s expectations or forecasts of future events
regarding the research, development and commercialization of
pharmaceutical products. 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, the occurrence of adverse safety
events; risks of unexpected costs or delays; unexpected concerns
that may arise from additional data, analysis or results obtained
during clinical trials; 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 fail to approve or may delay
approval of our drug candidates or expansion of product labelling;
failure to obtain regulatory approvals in other jurisdictions; and
product liability claims. 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 OfficerZealand PharmaEmail:
alange@zealandpharma.com |
|
Anna Krassowska,
PhD (Investors and Media)Vice President, Investor Relations &
Corporate CommunicationsZealand PharmaEmail:
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) Yorifuji et al. (2017) Clinical practice guidelines
for congenital hyperinsulinism, Clin Pediatr Endocrinol.
2017;26(3):127-152.
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