New drug for hereditary angioedema (HAE)! FDA approves Haegarda: used to routinely prevent HAE ≥ 6-year-old patients in the united states.

September 28, 2020 // -- CSL Behring is a global leader in plasma protein biologics.
recently, the company announced that the U.S. Food and Drug Administration (FDA) has approved Haegarda (subsurfic injection type C1 esterase inhibitor, C1-INH) expanded adaptation as a conventional preventive treatment for patients 6 years of age and older to prevent hereditary angioedema (HAE) episodes.
HAE is a rare, hereditary, potentially life-threatening disease that affects about one in 50,000 people in the United States and can cause swelling pain, potentially debilitating and unpredictable seizures in the abdomen, throat, face, limbs and other parts of the body.
if one of the parents has the disease, there is a 50% chance that the child will inherit HAE.
Haegarda reduces HAE seizures by 95 percent, making the drug the first and only off-skin treatment for patients 6 years and older to prevent HAE seizures.
addition to expanding pediatric adaptations, the updated labels include clinical safety data on haegarda use in pregnant women.
this latest approval, based on the results of two COMACT trials sponsored by CSL, COMPACT Key Studies and COMPAC Open Label Extension (OLE) studies.
COMPACT is an international, forward-looking, multi-center, randomized, double-blind, placebo-controlled Phase 3 key study that included six patients aged ≤17 years of age with symptomatic HAE pediatrics.
In a key COMPACT study, Haegarda reduced HAE seizures by 95 percent (median) and first aid drug use by more than 99 percent (median) compared to placebos at FDA-approved doses of 60 IU/kg.
126 patients in COMPACT OLE, 9 of whom were ≤ 17 years of age.
the trial, all nine children had more than 50 percent fewer seizures per month than before the study, and the median number of monthly seizures was reduced to 97 percent.
all subjects had fewer than 1 seizure/month, and 4 subjects had fewer than 1 seizure/year (one subject had no seizures).
patients stopped treatment due to adverse events associated with treatment.
safety and effectiveness of the age grouping analysis were consistent with the overall results.
new label now includes the results of randomized, open-label, positive drug-controlled studies involving four patients who became pregnant during the study period and were treated until pregnancy was determined.
patients were between the ages of 19 and 32 and were given C1-INH (S.C.).
were treated with Haegarda for 4-8 weeks (9-15 doses) in the early stages of pregnancy (the first three months).
as mentioned earlier, these women reported no complications during childbirth and all gave birth to healthy babies.
Haegarda: The first subsultic injection drug for the preventive treatment of HAE seizures in the United States, Haegarda was approved by the FDA in June 2017 as a routine preventive drug for the prevention of hereditary angioedema (HAE) in adolescents and adult patients.
this approval, making Haegarda the first and only under-skin preventive treatment option approved for HAE.
the approved dose of Haegarda was 60 IU/kg.
is a deficiency of HAE, or supplement C1 inhibitor (C1-INH), a rare genetic disorder with a incidence rate of 1:10000-1:50000.
defects in the C1-INH gene in HAE patients, leading to the absence or dysfunction of the C1-INH protein in the body, which is involved in the control of inflammation by the body's immune system.
In clinical, HAE is characterized by repeated episodes of angioedema, typical seizures of the epithelial, mucosal edema in 24h continuous progress and aggravation, and then in 48 to 72h can be white receding, edema can occur in any part, mainly affected areas for the face, limb end, external genitalia, upper respiratory mucosa and digestive tract mucosa.
the upper respiratory tract mucosal edema (UAE) causes respiratory obstruction, which can cause asphyxiation and life-threatening injuries.
in cases of asphyxia caused by UAE, which were not properly diagnosed and treated in a timely manner, the mortality rate was as high as 30% to 50%.
given the potential risk of death from HAE, preventive treatment of the disease is particularly important.
Haegarda is a low-volume, subsulcial injection-type preventive therapy that replaces the C1-INH protein that is missing or dysfunctional in patients with defective C1-INH genes to prevent haE attacks.
() Origin: U.S. Food and Drug Administration approves HAEGARDA (C1 Esterase Addor Subcutaneous (Human)) for Prevention of Hereditary Angioedema (HAE) Attacks in Pediatric Patients.