There was good news from speciality pharmaceuticals group Shire yesterday in the form of positive Phase III data for its hereditary angioedema (HAE) drug Firazyr and Canadian approval of its Vpriv.
The Dublin, Ireland-headquartered group said headline results from the FAST-3 (For Angioedema Subcutaneous Treatment) trial showed that patients with the rare genetic disease HAE given Firazyr (icatibant) experienced a significantly faster time until the start of symptom relief compared to those taking a placebo.
Data from the trial showed that Shire’s drug achieved its primary endpoint by inducing a 50% reduction in a composite symptom score assessment by the patient in just 2.0 hours (median value) compared 19.8 hours for the placebo arm, a highly stastistically difference that clearly demonstrates its potential benefit for those with the condition.
HAE is characterised by recurrent sudden attacks of swelling of the skin (hands, arms, feet, legs, thighs, face, genitals) or the mucous membranes (gastrointestinal tract, larynx or voicebox), with symptoms disfiguring and painful at best and potentially fatal at worst (swelling of the larynx can cause suffocation).
Firazyr is already approved for the treatment of acute HAE attacks in 37 countries, including within the European Union, but was barred entry to the all important US market in 2008 following a not approvable letter, so Shire agreed to carry out this additional clinical study to support its marketing application.
“These very positive and clinically meaningful results represent a significant milestone in the US clinical development program for Firazyr,” noted Sylvie Gregoire, president of Shire HGT. “We are currently preparing our complete response and we look forward to working with the FDA to provide Type 1 and Type 2 HAE patients in the US with an additional treatment option that holds the potential to offer quick access to effective treatment," she added.
Shire said it now expects to file a complete response to the FDA's not approvable letter early in 2011, pushing the drug closer to US clearance.
Canada OKs Vpriv
Meanwhile, in other good news for the firm, Candian health regulators issued a stamp of approval for Vpriv (velaglucerase alfa), an enzyme replacement therapy (ERT) for long-term use in paediatric and adult patients with type 1 Gaucher disease.
Gaucher disease is a rare and often debilitating lysosomal storage disorder caused by a hereditary deficiency of the enzyme glucocerebrosidase, which can lead to the build up of fatty substances in organs including the spleen, liver, kidney and brain, resulting in a whole host of complications such as liver malfunction, skeletal disorders anaemia.
As the term suggests, ERT involves treating a patient with the specific enzyme they are lacking; Vpriv has the same amino acid sequence as the naturally-occurring human enzyme glucocerebrosidase, and thereby offer patients with Gaucher’s hope for relief from some of the symptoms of the disease.