Therapy for congenital hyperinsulinism not approved due to manufacturing issue
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Key takeaways:
- The FDA issued a complete response letter for dasiglucagon to treat congenital hyperinsulinism on Oct. 9.
- The problem was lack of an inspection classification at a manufacturing facility, unrelated to the drug.
The FDA declined to approve a glucagon receptor agonist to treat congenital hyperinsulinism among infants and children, according to an industry press release.
Dasiglucagon (Zegalogue, Zealand Pharma) is a subcutaneous medication designed to treat severe hypoglycemia. As Healio previously reported, the FDA approved the agent as an auto injector or prefilled syringe to treat severe hypoglycemia for children and adults aged 6 years and older with diabetes in 2021.
The medication is currently under investigation for the prevention and treatment of hypoglycemia for children with congenital hyperinsulinism, a rare genetic disease in which pancreatic beta cells secrete too much insulin. The agent would be delivered subcutaneously through a wearable pump system.
On Oct. 9, Zealand Pharma announced it received a complete response letter from the FDA regarding part one of its new drug application for the medication, which would allow for the use for dasiglucagon for up to 3 weeks of dosing in infants and children aged 7 days and older with congenital hyperinsulinism. The FDA rejected the medication due to timing of a reinspection at a third-party contract manufacturing facility, according to Zealand Pharma’s press release. The company stated the facility previously had deficiencies unrelated to dasiglucagon. It was reinspected in August and September after the deficiencies were resolved, but the facility has yet to receive its inspection classification, according to the press release.
“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,” David Kendall, MD, chief medical officer of Zealand Pharma, said in the press release. “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.”
Zealand Pharma said the FDA’s complete response letter did not include any concerns about data or safety regarding dasiglucagon. As Healio previously reported, topline results from two phase 3 trials revealed dasiglucagon reduced hypoglycemia by 40% to 50% compared with standard of care among children with more than three hypoglycemic events per week despite near-total pancreatectomy. In a second phase 3 trial, dasiglucagon was tied to a significantly lower IV glucose infusion rate for infants aged 7 days to 12 months compared with placebo.
Part two of the new drug application would allow for the use of dasiglucagon for longer than 3 weeks. According to the press release, the FDA has requested additional continuous glucose monitoring data supporting the use of the medication for more than 3 weeks. Zealand Pharma said it plans to submit the data by the end of the year.
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