Irish giant Shire welcomes ‘important’ clinical trial result

Experimental drug led to ‘significantly fewer’ attacks of life-threatening genetic condition

Shire head of research and development Andreas Busch said results were an important development for patients

Shire head of research and development Andreas Busch said results were an important development for patients


Dublin-based pharma giant Shire has received test results which show patients treated with its experimental drug lanadelumab experienced “significantly fewer” attacks of a rare and potentially life-threatening genetic condition.

Hereditary angioedema (HAE) occurs in about one in 10,000 to one in 50,000 people. Its symptoms include swelling in various body parts, including the hands, feet, face and airway that can be debilitating and painful. Laryngeal attacks that obstruct the airways are potentially life-threatening due to the risk of asphyxiation.

Aside from the burden of the disease itself, patients can suffer anxiety, fatigue and depression between attacks.

Shire announced additional data from the phase three HELP study evaluating the efficacy and safety of subcutaneously administered lanadelumab in HAE on Friday. The results will be presented at the American College of Allergy, Asthma & Immunology 2018 annual scientific meeting.

“The data showed that patients treated with lanadelumab 300mg every two weeks experienced significantly fewer HAE attacks,” Shire said.

The patients were also “less likely to have moderate or severe attacks or use rescue medication [primary and secondary endpoints], and were more likely to be HAE attack-free than those treated with placebo.”

The results were noted during the entire 26-week treatment period, and, according to a post hoc sensitivity analysis, were greatest for patients during the 16-week steady state period (days 70-182).


Shire head of research and development Andreas Busch said the results were an important development for patients suffering from HAE.

“For those living with this chronic and unpredictable disease it is important that they can reduce the frequency of their HAE attacks. Additional analysis of the HELP study continues to support the use of lanadelumab as a preventive subcutaneous treatment option for HAE in appropriate patients 12 years of age and older.”

There were no serious side effects or deaths reported in the study’s participants. The most commonly reported adverse events were injection site pain (42.9 per cent), followed by upper respiratory infection (23.8 per cent), and headache (20.2 per cent).

Some 212 adolescent and adult patients received at least one dose of lanadelumab as part of the study. Of these, 109 patients were rollover participants from the study and 103 were new participants who had a confirmed diagnosis of HAE and a historical baseline attack rate of more than one attack per 12 weeks.

It was announced on Monday that Japan’s Takeda Pharmaceutical will hold an investor vote on its $62 billion acquisition of Shire next month, and aims to close the deal on January 8th, signalling its confidence in securing the required support.