News

The U.S. Food and Drug Administration (FDA) will review Shire’s application requesting that Cinryze (C1 esterase inhibitor [human]) be approved for the prevention of hereditary angioedema attacks in children 6  years and older. The supplemental biologics license application (sBLA) received priority review, meaning the process will be shortened from the standard 12 months…

A rock-painting contest in Las Vegas. A fashion show in New York. A 7,000-meter race around the Washington Monument that’ll coincide with a similar #Racefor7 event in Bengaluru and Mumbai, India. From Athens to Atlanta, from San Diego to Sydney, people across the globe will mark World Rare Disease…

  The application requesting that lanadelumab be reviewed with priority as a new treatment for the prevention of angioedema attacks has been accepted by Health Canada, the medicine’s manufacturer, Shire, announced. The New Drug Submission (NDS) is requesting the lanadelumab’s approval for patients older than 12 years, who live with hereditary angioedema (HAE). In…

Long-term treatment with human plasma-derived C1 inhibitor may help women with hereditary angioedema get pregnant and carry out the pregnancy to full term without risking complications, a case report suggests. The study, from a hospital in Valencia, Spain, titled, “Successful long-term prophylaxis with human plasma-derived C1 inhibitor in…

David Curtis Glebe, a retired 64-year-old public prosecutor now living in Millsboro, Delaware, knows he’s lucky to be alive. In mid-2013, while in Arizona, Glebe was diagnosed with pancreatic neuroendocrine cancer (PNET) — the same disease that killed Apple’s founder and CEO Steve Jobs. After three years of progress…

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 BCX7353, a…

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 1…