Dutch biotechnology company Pharming has filed for approval of its lead product, recombinant human C1 inhibitor, in the European Union, keeping it on track to move into profit later this year or early in 2007.
Pharming submitted rhC1INH to the European Medicines Agency (EMEA) for the treatment of acute attacks of hereditary angioedema, a human genetic disorder that affects roughly one in 30,000 people. Sufferers have an average of seven acute attacks per year, characterised by painful swelling of soft tissues, which can be life-threatening if the throat is involved.
Untreated attacks can last up to five days but, in February, Pharming reported data from a 25-patient study showing rapid time to relief of symptoms, starting between fifteen minutes to two hours.
Pharming said it anticipates responding to questions from EMEA and coordinating inspections in 2006.
Currently, the only approved product for the treatment of HAE attacks is plasma derived C1 inhibitor, which is available in a limited number of European countries.
Other companies developing treatments for HAE include Germany's Jerini, whose icatibant is in Phase III testing and was recently licensed to Kos Pharmaceuticals in the USA, as well as Lev Pharmaceuticals' C1-esterase inhibitor C1-INH (in Phase III) and Dyax Corp's DX-88 (in Phase II).