Shire has been granted approval by the EMA for rare disease drug Takhzyro, a blockbuster-in-waiting for the rare disease hereditary angioedema (HAE).
The EU regulator has cleared Takhzyro (lanadelumab) for the preventive treatment of HAE in patients aged over 12, based on clinical data showing that the drug can reduce HAE attacks – typically swelling in areas of the body such as the abdomen, face, feet, genitals, hands and throat – by 87% versus placebo.
Analysts have predicted that the drug could make up to $2bn in peak sales, making it one of the brightest prospects in Shire’s pipeline. EU approval follows a green light in the US in August as well as authorisation in Canada, and is big boost to Shire as well as Japan’s Takeda which is in the process of acquiring the Irish company in a $62bn takeover.
Takhzyro is the first antibody to be approved for HAE and targets plasma kallikrein, an enzyme whose activity is uncontrolled in people with the painful, debilitating, and potentially life-threatening disease. Sire acquired the drug through its $5.9bn buyout of Dyax Corp in 2016.
There are only a handful of treatments available for HAE, which affects around one in 50,000 people, and Shire is already a major provider of drug treatment with three other products on the market (Cinryze, Firazyr and Kalbitor) that collectively made more than $1bn sales in the first nine months of the year. Almost all sales come from the US market, mainly because of supply constraints for Cinryze in recent years.
The company maintains that Takhzyro has the potential to expand the HAE prevention market, particularly in international markets, as it is easier to administer than other drugs. It’s given as a subcutaneous injection that takes less than a minute to deliver every two weeks, around 75% fewer injections than other products for HAE prevention.
It’s early US take-up is encouraging, according to Shire, with third quarter sales coming in at $51m. On the company’s third-quarter results call chief executive Flemming Ornskov said that the prescriptions are coming for new patients as well as switches from other drugs.
The switchovers includes some patients on Cinryze but also competitors’ products, he said. At the moment Shire’s big competitor in HAE is CSL with its Haegarda product, a subcutaneous medicine that launched last year and has been trying to steal market share from Cinryze, which is administered intravenously twice a week.
Analysts at Bernstein recently predicted that Takhzyro will eventually dominate the HAE prophylaxis market with a 70% share.