The European Medicines Agency’s CHMP committee has recommended Shire’s Takhzyro (lanadelumab), a new rare disease treatment which has blockbuster potential.
Takhzyro has been recommended for prevention of recurrent attacks of hereditary angioedema (HAE) a rare, genetic disorder that results in recurring attacks of oedema in parts of the body, including the abdomen, face, feet, genitals, hands and throat. Laryngeal attacks that obstruct the airways are potentially life-threatening due to the risk of asphyxiation.
Approved in the US in August, the drug is important for Shire, which is in the midst of a $62bn acquisition by Takeda.
Analysts think the drug can achieve around $1.8bn in peak sales, though this will depend on many currently undiagnosed patients being identified and treated in the US, Europe and beyond.
The drug isn’t the first HAE treatment on the market, however: CSL’s subcutaneous therapy Haegarda gained approval in 2017 – despite Shire’s legal efforts to block it with claims it infringed one of its own patents.
CSL’s drug has showed a median reduction in monthly attacks of between 89% and 95% versus placebo, a very similar rate to that achieved by Shire’s drug in its phase 3 HELP trial.
However Takhzyro has an advantage in its dosing regimen – patients need only take it once or twice a month, compared with the twice a week administration of Haegarda.
“This positive opinion marks an important step towards providing adults and adolescents living with HAE in Europe a first-of-its-kind monoclonal antibody treatment option to help prevent attacks,” said Andreas Busch, executive vice president, head of research and development at Shire.
“We are excited about the future potential of lanadelumab in helping to address the needs of those living with this chronic and unpredictable disease.”
Lanadelumab had been granted accelerated assessment by the EMA, which cut the evaluation period from 210 to 150 days, with a final EU approval expected by early 2019.
The CHMP decision is good news for Takeda, as there has been talk of revolt among some of its Japanese shareholders against the acquisition, largely because the amount of debt Takeda will accrue. The news will help bolster Takeda CEO Christophe Weber’s argument that the deal makes sense.
Shire acquired Takhzyro through another acquisition deal, when it bought Dyax Corp. in January 2016 for $5.9 billion.