Shire is getting closer to regulatory approval for its potential blockbuster drug to treat Hereditary Angioedema (HAE).
The Dublin, Ireland-based drug maker on Thursday reported results from a global, multi-center, randomized, double-blind placebo study evaluating the efficacy and safety of investigational treatment lanadelumab in preventing angioedema attacks in patients 12 years of age or older.
Over a 26-week period, investigators gave 125 patients 150 or 300 mg subcutaneously administered dosages every four weeks or 300 mg every two weeks.
While the data showed all three treatment regimens produced a significantly higher proportion of patients who were attack free throughout the entire study period versus placebo, the 300 mg dose every two weeks elicited the most effective results with a reduced monthly HAE attack frequency of 87 percent compared to placebo. The results were consistent regardless of baseline attack rate.
“In the U.S., available treatment options include either injections for acute attacks or short-acting intravenous infusions administered twice a week,” said Dr. Aleena Banerji, M.D., Massachusetts General Hospital, Boston, MA and clinical trial investigator, in a statement. “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.”
About one in 50,000 people are diagnosed with HAE. Patients with this condition deal with recurrent episodes of severe swelling in extremities, upper airways, and the gastrointestinal tract, according to Reuters.
Analysts predict this drug could have blockbuster potential by generating more $2 billion in peak sales, which will help Shire protect and grow its HAE franchise.
The company’s next step is to submit a U.S. marketing application to the U.S. Food and Drug Administration later this year or early 2018 followed by an EU application in the first half of 2018 as well.
Filed Under: Drug Discovery
