This follows the EMA’s acceptance of Shire’s marketing authorization application (MAA) for the potential therapy, which reduces the number of days required for evaluation from 210 to 150.
Shire recently reported similar regulatory milestones in the U.S. and Canada. In March, the U.S. Food and Drug Administration (FDA) granted priority review to lanadelumab. A final decision is expected by August 26. In February, Health Canada accepted Shire’s New Drug Submission for the potential medication, which also means it will receive accelerated review. Similar to the U.S., a decision in Canada is planned for the second half of 2018.
Shire also received priority review and orphan drug designation for lanadelumab from Australia’s Therapeutic Goods Administration.
Lanadelumab was granted orphan drug status from the FDA in 2013 and from EMA in 2015. The treatment candidate also had received breakthrough therapy designation from the FDA.
Lanadelumab (SHP643) is an investigational, under-the-skin treatment for the prevention of HAE attacks in patients 12 years and older. It is an antibody designed to target and inhibit plasma kallikrein. High levels of this protein are the underlying cause of HAE.
“HAE presents a significant burden on the lives of patients whose recurring attacks of swelling can be debilitating and painful,” Andreas Busch, PhD, executive vice president, head of research and development at Shire, said in a press release.
“Lanadelumab is the first monoclonal antibody under evaluation to prevent HAE attacks and has the potential to change the treatment paradigm for this rare disease, if approved. We look forward to working with regulatory bodies to bring a new treatment option to HAE patients,” Busch added.
The company’s submissions are supported by results of four clinical trials, including the Phase 3 HELP trial (NCT02586805), which assessed the effectiveness and safety of under-the-skin lanadelumab. HELP included a total of 125 patients aged 12 and older with type I/II HAE and was the largest-ever prevention study in HAE, according to Shire.
Results showed that 300 mg lanadelumab given once every two weeks led to an 87% decrease in the mean frequency of HAE attacks and better health-related quality-of-life. Further analysis demonstrated that during the steady state stage of the trial (days 70-182), treatment with lanadelumab reduced HAE attacks by 91% and approximately eight out of 10 patients achieved an attack-free state.
No treatment-related serious adverse events or deaths were observed. The most frequent adverse event was pain at the injection site.
In addition to results from HELP, Shire also is including preliminary data of its extension study (NCT02741596), which covered 96% of patients who completed the Phase 3 trial and evaluated the long-term safety of lanadelumab over 14 months.
“As a physician treating patients with HAE, I would welcome new treatment options to help prevent attacks, as it is important to recognize the impact HAE can have on the quality of life of these individuals,” said Marcus Maurer, MD, an investigator in the HELP clinical trial.
“I am pleased to see the progress in the review of lanadelumab that if approved, would offer a targeted mechanism of action inhibiting plasma kallikrein,” he said.