The U.S. Food and Drug Administration (FDA) has approved an expanded indication for HAEGARDA for routine prophylaxis to prevent HAE attacks in patients 6 years of age and older.
HAEGARDA, the HAE therapy that reduces attacks by a median of 95%, is now the first and only subcutaneous treatment option for prevention of HAE attacks in patients 6 years of age and older. In addition to the expanded pediatric indication, the updated label now includes clinical safety data regarding HAEGARDA use in pregnant women.
“Since 2017, HAEGARDA has been a trusted and effective option for patients seeking to prevent HAE attacks, but until now preventative options for younger children living with the condition have been limited,” said Debra Bensen-Kennedy, MD, Vice President, North America Medical Affairs, CSL Behring. “With this expanded indication, we are able to offer pediatric patients as young as 6 years of age, an effective, preventative subcutaneous solution and deliver on our promise of addressing the unmet needs of people living with HAE.”
The latest FDA approval was based on results from two CSL Behring-sponsored COMPACT (Clinical Study for Optimal Management of Preventing Angioedema with Low-Volume Subcutaneous C1-Inhibitor Replacement Therapy) trials: COMPACT Pivotal Study and COMPACT Open Label Extension (OLE) Study.
COMPACT, an international, prospective multi-center, randomized, double-blind, placebo-controlled Phase 3 pivotal study, included six subjects aged 17 years or younger with symptomatic HAE. In the COMPACT pivotal study, HAEGARDA, at the FDA approved dose of 60 IU/kg, reduced the number of HAE attacks by a median of 95% relative to placebo. Use of rescue medication was reduced by a median of greater than 99% versus placebo.
COMPACT OLE featured 126 subjects, including nine patients ages 17 years or younger. In this trial, all nine pediatric subjects experienced greater than 50 percent reduction in number of attacks per month versus the pre-study period, with a median of 97% reduction in the median number of attacks/month (0.11). All subjects had less than one attack/4-week period and four had less than one attack/year (one subject was attack free). No subject discontinued treatment due to a treatment-related adverse event. Safety and effectiveness results of subgroup analysis by age was consistent with overall study results.
“The FDA’s approval of HAEGARDA for preventing HAE attacks in children six years of age and older represents a major stride in filling an unmet medical need. The U.S. Hereditary Angioedema Association is excited that there is now a subcutaneous prophylaxis option for the younger members of our HAE community and the families that care for them,” said Anthony J. Castaldo, U.S. HAEA President & Chief Executive Officer.
The new label now includes results from the randomized, open-label, active treatment controlled study regarding four patients who became pregnant during the study, and received treatment until pregnancy was identified. These patients ranged in age from 19 to 32 and received C1-INH (S.C. administration). Patients received HAEGARDA for 4 – 8 weeks (9 – 15 doses) during the first trimester. As noted, these women reported no complications during delivery and all women delivered healthy babies.
“As the mother of a young patient and an active member of the HAE community, this news brings tears to my eyes as I am reminded of our struggles and strides over the years,” says Lisa Chacon, HAE patient advocate. “It warms my heart to know that so many children will now have access to this convenient, preventative therapy.”
(Source: CSL Behring)