- The FDA designates Dyax's (NASDAQ:DYAX) investigational fully human monoclonal antibody inhibitor of plasma kallikrein (pKAL), DX-2930, a Breakthrough Therapy for the treatment of hereditary angioedema (HAE). Uncontrolled pKAL activity leads to excessive generation of bradykinin, a vasodilator believed to play a key role in localized swelling, inflammation and pain associated with HAE.
- HAE is a rare acute inflammatory condition characterized by episodes of severe, painful swelling of the extremities, GI tract, genitalia and larynx. It is caused by abnormally low levels of C1 esterase inhibitor (C1-INH), a naturally occurring molecule that inhibits pKAL. HAE affects ~1 in 50,000 people.
- Breakthrough Therapy status provides for more intensive guidance from the FDA, the involvement of more senior agency staff and a rolling review of the New Drug Application (NDA).