This article is part of MPR‘s coverage of the American Academy of Allergy, Asthma & Immunology annual meeting, taking place in San Francisco, California. Our staff will report on medical research related to asthma, allergy, and other respiratory conditions, conducted by experts in the field. Check back regularly for more news from AAAAI 2019. Researchers sought to examine…
During the treatment period, the mean number of attacks per month was 1.97 for placebo and 0.48, 0.53, and 0.26 for the lanadelumab groups, respectively. The mean differences in the attack rate per month compared with placebo were −1.49, −1.44, and −1.71, respectively.
Patients with hereditary angioedema who received lanadelumab exhibited few cases of hypersensitivity and had low immunogenicity.
The Company filed the sBLA last November following FDA feedback on two completed Phase 2 trials of Ruconest for prophylaxis of HAE attacks.
ADVM-053 is being developed as a single‑administration treatment that could potentially provide sustained levels of the C1 esterase inhibitor protein, which may prevent breakthrough angioedema attacks.
The approval was based on data from 4 clinical trials, including the 26-week Phase 3 HELP (Hereditary Angioedema Long-Term Prophylaxis) study that enrolled 125 patients with HAE aged ≥12 years.
The Company is currently conducting a Phase 3 trial (APeX-2) and a long-term safety study (APeX-S) evaluating 2 doses of the treatment.
The most commonly reported adverse events were gastrointestinal adverse events, mainly grade 1, especially in the 2 highest BCX7353-dose groups.
The approval was based on data from a randomized, single-blind, multicenter, dose-ranging, crossover study which evaluated the safety and efficacy of Cinryze in 12 pediatric patients (7 to 11 years old).
Results found that those who received a subcutaneous administration of 300mg lanadelumab once every 2 weeks had an 87% reduction in mean frequency of HAE attacks.
