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.
The case-control analysis found no significant difference in the angioedema risk for aliskiren monotherapy and fixed-dose combination compared to BBs with adjusted odds ratio of 0.99 (95% CI, 0.45-2.20) and 1.06 (0.40-2.76), respectively.
The human plasma-derived, purified, pasteurized, lyophilized concentrate is derived from large pools of human plasma from U.S. donors.
Longhurst, Hilary, et al. "Prevention of Hereditary Angioedema Attacks with a Subcutaneous C1 Inhibitor"
CSL Behring announced that the Food and Drug Administration (FDA) has approved the use of Berinert (C1 esterase inhibitor [human]) for the treatment of hereditary angioedema (HAE) attacks in pediatric patients.
For the management of angiotensin-converting enzyme inhibitor (ACEI)-induced angioedema, use of fresh frozen plasma and complement 1 esterase (C1) inhibitor appear to be safe and effective whereas ecallantide should be avoided, a study published in the American Journal of Health-System Pharmacy concluded.
The Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for the investigation of DX-2930 (Dyax) for hereditary angioedema (HAE).
Salix and Pharming Group announced the launch of Ruconest (C1 esterase inhibitor [recombinant]) injection for the treatment of acute angioedema attacks in patients with hereditary angioedema (HAE).
Salix and Pharming Group NV announced that the FDA has approved Ruconest (C1 Esterase Inhibitor [Recombinant]) for the treatment of acute angioedema attacks in adult and adolescent patients with hereditary angioedema (HAE).
The FDA has accepted for review Santarus and Pharming's Biologics License Application for Ruconest (INN conestat alfa) 50 IU/KG, a recombinant human C1 esterase inhibitor for the treatment of acute angioedema attacks in patients with hereditary angioedema.
For patients with hereditary angioedema (HAE), weekly administration of recombinant C1INH (rhC1INH) is well tolerated and is associated with a reduction in attack frequency.
Santarus and Pharming Group NV announced that their Phase 3 clinical study of Ruconest (recombinant human C1 esterase inhibitor) for the treatment of acute attacks of angioedema in patients with Hereditary Angioedema (HAE) met its primary endpoint.