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 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, 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 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.
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
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 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, 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 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.
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
