Takhzyro™ (Lanadelumab-flyo) – 2018 FDA Approval

FDA Approval: 8/23/2018

Hereditary angioedema (HAE) is a disease characterized by recurrent and debilitating attacks of angioedema, or severe swelling of the limbs, airway, face, or intestinal tract. HAE affects about one in 10,000 to 50,000 individuals worldwide and can have a significant impact on quality of life. There are currently six FDA-approved therapies available for individuals with HAE; however, there is still an unmet need for this condition as new medications with sustained efficacy and less treatment burden are being sought out.

Lanadelumab-flyo is a fully human monoclonal antibody developed by Shire Plc (previously Dyax Corp, acquired by Shire in 2016). Lanadelumab-flyo received Priority Review designation from the FDA in February of 2018 with a PDUFA action date of August 26, 2018. FDA approval for Prophylaxis of Hereditary Angioedema (HAE) Attacks for patients 12 years and over occurred on August 23, 2018.

For our complete review of Lanadelumab-flyo (Takhzyro™):

Executive Summary of Evaluation:


Lanadelumab (Shire Plc)







  Orphan Drug Designation


  FDA Fast Track Designation


  Breakthrough Therapy Designation


  Priority Review Designation


  FDA Approval


  Patent Expiration


Indications Hereditary Angioedema (HAE)

Approved (BLA)

Efficacy Hereditary Angioedema (HAE)

Efficacy reported in Phase III Trials


Clinical Trial data obtained at https://clinicaltrials.gov/ct2/home Accessed: June 28, 2018.

“FDA Approved Treatments”. US Hereditary Angioedema Association (HAEA). https://www.haea.org/ApprovedTreatments.php. Accessed: June 29, 2018.

Global Data, Pharma Intelligence Center https://pharma.globaldata.com Accessed: August 27, 2018.

“Hereditary angioedema”. NIH U.S. National Library of Medicine: Genetics Home Reference. https://ghr.nlm.nih.gov/condition/hereditary-angioedema#resources. Accessed: June 28, 2018.

“Shire Reports Positive Topline Phase 3 Results for Lanadelumab (SHP643) in Patients With Hereditary Angioedema (HAE)”. Shire. http://investors.shire.com/~/media/Files/S/Shire-IR/presentations-webcast/year-2017/shire-investor-presentation-2017-05-18.pdf. Accessed: June 28, 2018.

U.S. Food & Drug Administration Drugs@FDA: FDA Approved Drug Products Product Labeling https://www.accessdata.fda.gov/drugsatfda_docs/label/2018/761090s000lbl.pdf Accessed: August 27, 2018

U.S. Food & Drug Administration, FDA News & Events, FDA News Release: “FDA approves new treatment for rare hereditary disease” https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm616176.htm  Accessed: August 27, 2018

“What is HAE?” HAEi.org. http://haei.org/hae/what-is-hae/. Accessed: June 28, 2018.

Additional 2018 Pipeline Reviews are available in our pipeline library.

Please see the July 2018 FDA Actions Newsletter for additional information on recent FDA approvals

Coauthored by: Kristen Ciampi, PharmD

Kristen recently graduated with a PharmD degree from the University of Rhode Island in May of 2018, and is a Pharmacy Analytics Intern at RJ Health Systems.