FDA Drug Approvals: Pediatrics — Year in Review 2018

Mary L Windle, PharmD


January 15, 2019

New Drugs in Immunology

Revcovi (elapegademase)

Elapegademase is indicated for adults and children with adenosine deaminase severe combined immune deficiency (ADA-SCID). Elapegademase provides an exogenous source of ADA enzyme that catalyzes the hydrolytic deamination of adenosine or deoxyadenosine to inosine or deoxyinosine. Maintaining a low level of 2'-deoxyadenosine and adenosine is crucial for proper number and function of immune cells, as well as decreasing the frequency of opportunistic infections.

For years, patients with ADA-SCID have been treated with pegademase (Adagen), which is bovine derived. Elapegademase is a new pegylated recombinant adenosine deaminase (rADA) that does not need to be sourced from animals.

Takhzyro (lanadelumab)

Lanadelumab is indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE). It is a human monoclonal antibody (IgG1 kappa light chain) that targets plasma kallikrein and inhibits proteolytic activity to control excess bradykinin generated with HAE. In patients with HAE caused by C1-inhibitor (C1-INH) deficiency or dysfunction, normal regulation of plasma kallikrein activity is not present, which leads to uncontrolled increases in plasma kallikrein activity and results in angioedema attacks.

Approval was based on the HELP clinical trial, which investigated the efficacy and safety of lanadelumab for long-term prophylaxis against angioedema attacks in HAE. Of 125 patients, 113 completed the trial. All lanadelumab dosing regimens significantly reduced the mean monthly attack rates of HAE compared with placebo (P<.001) over the 26-week duration of the study. The regimen of 300 mg SC every 2 weeks reduced attacks by 92.8% from baseline in patients with fewer than two attacks per month and by 88.2% in patients whose baseline was two to fewer than three attacks per month. (JAMA. 2018 Nov 27;320(20):2108-2121)


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