Summaries of Must-Read Clinical Literature, Guidelines, and FDA Actions
FDA Approves Haegarda for Hereditary Angioedema
FDA news release; 2017 Jun 22
The FDA has approved Haegarda (C1 esterase inhibitor subcutaneous [human]) to prevent hereditary angioedema (HAE) attacks.
Indications: Haegarda is a plasma-derived concentrate of C1-INH indicated for routine prophylaxis to prevent HAE attacks in adolescent and adult patients.
Dosage and Administration:
- Administer 60 international units per kg body weight twice weekly.
- Reconstitute prior to use using sterile water for injection, USP.
- Use a silicone-free syringe for reconstitution and administration.
- Administer at room temperature within 8 hours after reconstitution.
Efficacy and safety: Approval is based on study results showing that participants receiving either 40 or 60 IU/kg doses experienced a significantly reduced number of HAE attacks compared to those taking placebo.
Side effects/risks: The most common adverse reactions are injection site reaction, hypersensitivity, nasopharyngitis, and dizziness.
FDA approves first subcutaneous C1 Esterase Inhibitor to treat rare genetic disease [news release]. Silver Spring, MD: FDA. June 22, 2017. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm564332.htm. Accessed July 2, 2017.
Haegarda [package insert]. Kankakee, IL: CSL Behring LLC, Inc. 2017. https://www.fda.gov/downloads/BiologicsBloodVaccines/BloodBloodProducts/ApprovedProducts/LicensedProductsBLAs/FractionatedPlasmaProducts/UCM564335.pdf. Accessed July 2, 2017.