Dyax Corp. announced that the US Food and Drug Administration's (FDA) Pulmonary-Allergy Advisory Committee voted (6 yes, 5 no, 2 abstentions) in favour of approval of DX-88 (ecallantide) for the treatment of acute attacks of hereditary angioedema (HAE). If approved, DX-88 will be the first drug available in the US for treating acute attacks of HAE and the first subcutaneously administered HAE therapy. HAE is a rare, potentially fatal genetic disorder characterized by spontaneous episodes of severe, debilitating and often painful swelling. The Committee's findings will be weighed by the FDA in determining whether DX-88 is to be approved for marketing.
"Dyax is grateful to the Committee for their review, and we will consider their recommendations in our ongoing discussions with the FDA," said Gustav A. Christensen, president and chief executive officer of Dyax. "We are committed to establishing safe use conditions for DX-88, if approved, and will work with the FDA to ensure our post-marketing program achieves this goal. We look forward to bringing DX-88 to HAE patients living with this devastating disease."
According to a recent Burden of Illness study sponsored by the United States Hereditary Angioedema Association and Dyax1, the average HAE patient experiences more than 26 acute attacks per year. Approximately half of the study respondents reported missing school or work as a result of any given acute attack, with a typical attack lasting an average of more than two and a half days. Attacks can occur in any part of the body, becoming life-threatening when affecting the larynx. Study respondents reported experiencing considerable long-term burden in addition to the immediate pain and suffering associated with acute attacks, including depression, missed opportunities, deterioration of mental and physical health and overall decreased productivity.
The BLA submission was based primarily on data from two placebo-controlled Phase 3 clinical studies, known as EDEMA3 and EDEMA4, which, taken together, represent the largest placebo-controlled evaluation of any therapy used in the treatment of HAE.
The recombinant, small protein, DX-88 (ecallantide), was discovered utilizing Dyax's proprietary phage display technology. DX-88 is a potent and selective plasma kallikrein inhibitor, a key mediator of inflammation in angioedema, and is being evaluated as a subcutaneous therapy for treating acute HAE attacks.
Hereditary angioedema (HAE) is an acute inflammatory condition characterized by episodes of severe, often painful swelling affecting the extremities, the gastrointestinal tract, the genitalia, and in potentially life-threatening cases, the larynx. HAE is caused by low or dysfunctional levels of C1 esterase inhibitor (C1-INH), a naturally occurring molecule that inhibits plasma kallikrein, a key mediator of inflammation, and other serine proteases in the blood.
Dyax is focused on advancing novel biotherapeutics for unmet medical needs, with an emphasis on oncology and inflammatory indications. Dyax utilizes its proprietary drug discovery technology to identify antibody, small protein and peptide compounds for clinical development.