As expected, advisors to the US Food and Drug Administration have recommended approval for Shire's Firazyr as a treatment for hereditary angioedema.
The agency's Pulmonary-Allergy Drugs Advisory Committee has recommended, by a vote of 12-1, that efficacy and safety data provides substantial evidence to back Firazyr (icatibant) for the treatment of acute attacks of HAE in patients 18 years and older. In addition, the panel voted 11-1 (with one abstention) to recommend self-administration of the drug, echoing the backing given for the treatment in papers released by FDA staff.
The vote was based on data from three late-stage trials including FAST-3, which showed that the median time to onset of symptom relief for Firazyr by this measure was 2 hours, compared with 19.8 hours for placebo. That trial was necessary to satisfy the FDA which originally rejected the drug(acquired by Shire through its purchase of Jerini in 2008) because of variations in clinical data.
HAE is a rare genetic disorder characterised by recurring and sudden attacks of swelling in the skin (including hands, feet, face and genitals) or the mucous membranes (gastrointestinal tract, larynx or voicebox).
Sylvie Gregoire, president of the Shire HGT unit, said that "due to the unpredictable and potentially life-threatening nature of HAE attacks, we believe that self-administration of Firazyr can represent an effective and convenient treatment". The drug is already available in 37 countries, including those of the European Union, and the FDA is expected to make a final decision by August 25.