The U.S. FDA approved the company’s Investigational New Drug application for initiation of two Phase 3 clinical trials of dasiglucagon in treating congenital hyperinsulinism.
Zealand Pharma has announced that the U.S. Food and Drug Administration (FDA) has approved the company’s Investigational New Drug (IND) application for initiation of two Phase 3 clinical trials of dasiglucagon in treating congenital hyperinsulinism (CHI).
Britt Meelby Jensen, President and CEO of Zealand, wrote in a press release: "Congenital hyperinsulinism is a rare and severe disease, which affects newborns and children. I am very pleased with FDA's decision to approve our Phase 3 trials, as it allows us to make fast progression towards a potential treatment that can help these children and possibly save them from surgery with severe lifelong implications.”
CHI is the result of a genetic defect developed in neonates and children in which the beta cells in the pancreas produce abnormally high levels of insulin. These patients frequently experience episodes of low blood sugar, or hypoglycemia, that cause a lack of energy in the patient, irritability, rapid heart rate and difficulty feeding.
Low blood sugar episodes most commonly begin in a patient’s first month of life and be very severe; however, some patients do not develop CHI until well into childhood and are often milder versions of the condition.
As the brain’s access to sugar is continuously depleted, frequent episodes of low blood sugar create a series of complications such as breathing difficulties, seizures, intellectual disability, vision loss, brain damage, and coma. The treatment is being developed to assist in patients avoiding potential brain damage.
In 2017, the FDA and European Commission (EC) granted dasiglucagon orphan drug designation as a therapy for CHI. In the coming months, Zealand intends to evaluate the potential of chronic dasiglucagon infusions delivered through a pump to prevent hypoglycemia in 50 children diagnosed with CHI in a Phase 3 clinical trial. Researchers hope to reduce morbidities associated with hypoglycemia, neurodevelopmental defects and the need for pancreatectomy.
Dasiglucagon is being researched in a pair of other indications, including the ready-to-use rescue pen treatment of severe hypoglycemia and a Type 1 Diabetes artificial pancreas pump containing insulin and dasiglucagon. The results from these trials are expected in 2018.
According to the Children’s Hospital of Philadelphia (CHOP), 1 in 25,000 to 50,000 babies will be diagnosed with CHI. While 50% of children will respondto medical therapy, other patients must undergo surgery to partially — sometimes entirely – remove the pancreas during the first month of life to control their blood glucose levels.
Zealand expects that fast development of their CHI therapy will offer an effective non-surgical treatment for future patients.