FDA Grants Orphan Drug Status to Gene Therapy Candidate ADVM-053 for HAE

A new gene therapy candidate, ADVM-053, developed by Adverum Biotechnologies, was granted orphan drug status by the U.S. Food and Drug Administration for the treatment of hereditary angioedema (HAE). The FDA’s orphan drug designation program grants orphan status to investigational therapies being developed for the diagnosis or treatment of…

New Model Predicts Correlation Between Doses of Subcutaneous Medication and Risk for HAE Attacks

A new model estimates the association between doses of a subcutaneous medication and risk for an attack in hereditary angioedema (HAE) patients, a recent study shows. The research, “Exposure-Response Model of Subcutaneous C1-Inhibitor Concentrate to Estimate the Risk of Attacks in Patients With Hereditary Angioedema,” was published…

FDA to Review Pharming’s Application for Ruconest as HAE Swelling Prevention

The U.S. Food and Drug Administration (FDA) has agreed to review Pharming’s application to get Ruconest (conestat alfa) approved for the routine prevention of swelling attacks caused by hereditary angioedema (HAE). The therapy already is approved for the acute treatment of swelling attacks in adults and adolescents with…