News

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…

The recently announced merger of BioCryst Pharmaceuticals and Idera Pharmaceuticals will strengthen the two companies’ focus on developing and commercializing treatments to serve patients with rare diseases, including angioedema, the companies said. The name of the new company has not yet been announced. Among BioCryst’s ongoing development programs is…

The biopharmaceutical company Shire is expected to soon submit a biologics license application to the U.S. Food and Drug Administration (FDA) seeking marketing approval of its investigational monoclonal antibody lanadelumab as therapy for patients with hereditary angioedema. The application is supported by positive results from the company’s Phase 3 HELP…

This week marks the launch of the “7,000 Mile Rare Movement,” a nationwide effort to raise money for research into the 7,000 known rare diseases that afflict at least 30 million Americans. The campaign kicks off Feb. 1 and culminates with Rare Disease Day on Feb. 28. Organized by…

An oral candidate for the treatment of hereditary angioedema (HAE) has begun a phase 1 clinical trial, KalVista Pharmaceuticals recently announced. HAE is a rare genetic disease characterized by recurrent episodes of severe swelling (angioedema) and potential pain, nausea, and vomiting. It occurs in about 1 in 10,000 to…

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…