Dublin-based biotechnology company Shire is gearing up to submit a biologics license application (BLA) to the FDA after releasing positive data from its Phase III clinical trial of lanadelumab. The drug was able to reduce the monthly attack rate in patients with hereditary angioedema (HAE) by 87 percent.
The placebo-controlled HELP clinical trial involved 125 patients 12 years or older with type I/II HAE. The rare genetic disease is characterized by intermittent angioedema attacks, which result in swelling of the gastrointestinal tract, upper airways and the extremities.
“In the US, available treatment options include either injections for acute attacks or short-acting intravenous infusions administered twice a week,” said Dr. Aleena Banerji of Massachusetts General Hospital, and clinical trial investigator. “If approved, lanadelumab may offer patients a long-acting treatment option that significantly reduces HAE attacks when administered subcutaneously as infrequently as every four weeks.” the placebo. The drug was administered via a subcutaneous injection on a biweekly basis, and the statistically significant reduction in HAE attack frequency was consistently observed regardless of a patient’s baseline
The HELP study met its primary and secondary endpoints, compared to the placebo. The drug was administered via a subcutaneous injection on a biweekly basis, and the statistically significant reduction in HAE attack frequency was consistently observed regardless of a patient’s baseline attack rate.
Shire reported that patients enrolled in the study showed a range of disease pathology; 65 percent had a history of laryngeal attacks, 52 percent experienced three or more attacks before starting treatment, and 56 percent were on long-term prophylaxis to prevent attacks.
“The possibility of a new way to address the underlying cause of HAE to prevent attacks could transform how we treat the disease in the future,” said Dr. Marcus Maurer of Charité –Universitätsmedizin in Germany, and clinical trial investigator. “Patients with HAE want to live independently and without fear of an angioedema attack.”
Between 1 in 10,000 and 1 in 50,000 people around the world are affected by HAE. The rare genetic disorder causes localized edema, which can be debilitating, painful, and in the case of throat swelling, life-threatening.
“We are extremely encouraged by these topline Phase 3 results,” said Dr. Flemming Ornskov, CEO of Shire. “We have nearly a decade of experience and a strong portfolio and pipeline in HAE and believe these data demonstrate high potential for transforming the way patients living with this condition are treated.”