RARE Daily

FDA Expands Approved Use of CSL Behring’s HAE Therapy to include Kids Age 6 and Older

September 29, 2020

Rare Daily Staff

The U.S. Food and Drug Administration approved expanded use for CSL Behring’s Haegarda to prevent attacks from hereditary angioedema attacks in patients 6 years of age and older.

Hereditary angioedema (HAE) is a rare, genetic, and potentially life-threatening condition. HAE causes painful, debilitating, and unpredictable episodes of swelling of the abdomen, larynx, face and extremities, among other areas of the body. HAE is caused by deficient or dysfunctional C1-INH, a key protein in the body that controls swelling. The defect with C1-INH lies within a person’s genetic code, which is why HAE runs in families. HAE is classified as either type I, type II or HAE with normal C1-INH levels.

Haegarda is a self-administered, plasma-derived concentrate of C1-esterase inhibitor and the only subcutaneous therapy approved in the United States for routine prophylaxis to prevent HAE attack in patients 6 years of age and older. Haegarda targets the root cause of HAE by replacing deficient or dysfunctional C1-INH, restoring functional C1-INH levels to above 40 percent of normal levels, which is proposed to reduce the risk of HAE attacks. Haegarda is dosed individually based on body weight so that each patient can achieve functional C1-INH levels.

The latest FDA approval was based on results from two CSL Behring-sponsored studies: COMPACT Pivotal Study and COMPACT Open Label Extension 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 percent relative to placebo. Use of rescue medication was reduced by a median of greater than 99 percent versus placebo.

COMPACT Open Label Extension 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 percent reduction in the median number of attacks/month. All subjects had less than one attack per 4-week period and four had less than one attack a 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.

“With this expanded indication,” said Debra Bensen-Kennedy, vice president of North America medical affairs for CSL Behring, “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.”


Photo: Debra Bensen-Kennedy, vice president of North America medical affairs for CSL Behring


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