The US Food and Drug Administration approved in July Ruconest (C1 esterase inhibitor) in adults and adolescents with acute attacks of hereditary angioedema (HAE). The product, the first approved C1-esterase inhibitor manufactured in recombinant form, is purified from the milk of transgenic rabbits and restores levels of functional C1-esterase inhibitor in the plasma of patients, thereby reducing swelling often seen in the abdomens, extremities and faces of HAE sufferers. The drug is produced by Leiden, the Netherlands–based Pharming Group partnered with Salix Pharmaceuticals of Raleigh, North Carolina.
“'The Broad Gets $650 Million For Psychiatric Research'. That seems an awful lot of money for one woman.” One person's reaction to the gift of $650 million from the Stanley Foundation to the Cambridge, Massachusetts Broad Institute. (In the Pipeline, 22 July 2014)
“More people are studying orphan diseases than have orphan diseases.” Michael S. Kinch, of Yale Center for Molecular Discovery, commenting on pharma's disinterest in developing antibiotics. According to drug benefits manager Express Script, 70% of drugs approved by the FDA in 2013 were specialty drugs, used by less than 1% of the population. (The New York Times, 22 July 2014)