FDA rejects Zealand Pharma's hypoglycemia treatment for infants

The US Food and Drug Administration (FDA) has issued a complete response letter (CRL) to Zealand Pharma, denying the approval of dasiglucagon for treating and preventing hypoglycaemia in infants with congenital hyperinsulinism (CHI). This decision is related to Part 1 of the new drug application (NDA) which pertains to patients aged seven days and older. The CRL highlights the necessity for a reinspection at a third-party manufacturing facility.

The regulatory decision hinges on the timing of a reinspection at a contract manufacturing site, which concluded in August/September 2024. The facility, responsible for producing dasiglucagon, has not yet received its inspection classification following the reinspection. Although a prior inspection identified deficiencies, none were related to dasiglucagon, and these issues had been addressed by the time of the latest inspection.

The CRL does not raise any concerns regarding the clinical data or the safety profile of dasiglucagon. Zealand Pharma’s NDA for dasiglucagon is being reviewed in two parts. While Part 1 involved dosing for up to three weeks, Part 2 will evaluate usage beyond this period. The FDA has requested additional analyses from existing continuous glucose monitoring data collected during Phase III clinical trials to support the prolonged use of dasiglucagon in CHI.

Zealand Pharma plans to submit the required data by the end of 2024. David Kendall, Zealand Pharma’s chief medical officer, emphasized the company's awareness of the significant unmet medical need for newborns and children with congenital hyperinsulinism, who currently have very limited treatment options. He affirmed Zealand Pharma's commitment to working with the FDA and their third-party manufacturing partner to bring dasiglucagon to patients in need of this important treatment in the coming months.