Pharming of the Netherlands has reported encouraging data from a Phase II/III of its candidate drug for hereditary angioedema, a debilitating genetic disease that affects around 10,000 patients in the USA and Europe.
Pharming said that its product, a recombinant human C1 inhibitor (rhC1INH), was able to relieve the symptoms of HAE rapidly – generally between fifteen minutes to two hours after treatment. Patients typically reported minimal symptoms within twelve hours of dosing.
HAE is characterized by acute attacks of painful swelling of soft tissues (oedema), including regions of the skin, the intestine, and the mouth and throat. If the soft tissue of the throat is involved, an attack of angioedema can be fatal.
None of the patients treated with rhC1INH showed any related serious adverse events, allergic reactions or antibody responses to study medication, nor did any patient experience a relapse of the initial HAE attack. rhC1NH is Pharming’s lead pharmaceutical development programme.
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).