Shire’s Firazyr gains US approval after initial setback
pharmafile | September 1, 2011 | News story | Sales and Marketing | FDA, Firazyr, Shire
After three years of waiting Shire’s Firazyr has been approved by the FDA for sudden attacks of swelling.
The US reguator licensed Firazyr (icatibant) injection for the treatment of acute attacks of a hereditary angioedema (HAE) in people ages 18 years and older.
HAE is caused by low levels, or the improper function, of a protein called C1 inhibitor, which is involved in regulating how certain immune system and blood clotting pathways function.
Decreased function of this protein can lead to rapid and serious swelling of the face or other parts of the body, which may result in permanent disfigurement, disability or death.
The US regulator had denied the drug this licence three years ago, saying the Ireland-based biotech had not produced sufficient data for Firazyr.
But new data showing the drug’s efficacy and safety was enough to persuade the FDA to approve it this time around.
Although it failed in its first attempt to convince the FDA of Firazyr’s merits, Shire did manage to persuade the EMA of its efficacy, and it has been on the European market as an orphan drug for HAE since 2008.
But because it has not enjoyed a presence in the US – the world’s largest pharma market – the drug has struggled to make major sales, bringing in only $11 million last year.
It is estimated US approval will lift sales by $100 million in additional peak sales, and analysts have touted the drug as one to watch out for in the future.
Timothy Craig, professor of medicine and pediatrics at Penn State Hershey Medical Center, said: “Until now, HAE patients faced challenges gaining rapid access to acute treatment such as the need to travel to the physician’s office or hospital.
“Firazyr is a treatment with demonstrated efficacy that can be carried and stored at room temperature and self-injected by the patient. [The drug] addresses this important unmet need by providing HAE patients with fast access to acute treatment,” he concluded.
The safety and efficacy of drug was shown in three controlled clinical trials in which 225 patients received over one thousand doses of 30mg Firazyr.
The median time for patients treated with Firazyr to report onset of symptom relief was two hours compared with almost 20 hours with placebo.
Firazyr is the third drug approved in the US to treat HAE attacks – in October 2009 the FDA approved CSL Behring’s Berinert to treat facial and abdominal attacks of HAE.
Two months later it approved Dyax Corp’s Kalbitor to treat acute attacks of HAE in patients ages 16 years and older.
Even though it is third to market in the US, because it can be administered by the patient it could gain an edge over these pre-existing treatments, which are not yet licensed in the country to be injected at home by patients.
HAE’s rarity – fewer than 30,000 people in the US suffer from the disease – has seen the FDA grant Firazyr orphan status, extending the time Shire can hold on to its patent.
Ben Adams
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