FDA Drug Approvals: Pediatrics — Year in Review 2018

Mary L Windle, PharmD


January 15, 2019

New Antimicrobials

Seysara (sarecycline)

Sarecycline is a new first-in-class tetracycline-derived antibiotic indicated for adults and children aged 9 years and older with non-nodular moderate-to-severe acne vulgaris. Compared with currently available tetracyclines, it has a narrow spectrum of activity, including less activity against enteric gram-negative bacteria, and it also elicits anti-inflammatory effects.

Clinical trials showed efficacy compared with placebo to be statistically significant. Onset of efficacy, observed by improvement of inflammatory lesions, was evident at the first follow-up visit (ie, 3 weeks). (J Drugs Dermatol. 2018 Sep 1;17(9):987-996)

Xofluza (baloxavir marboxil)

Baloxavir marboxil was approved by the FDA in October 2018 for treatment of influenza in adults and adolescents aged 12 years or older as a single, weight-based oral dose for use within 48 hours of symptom onset. It is a prodrug that inhibits cap-dependent endonuclease, an enzyme specific to influenza, resulting in inhibition of viral replication. In clinical trials, single-dose baloxavir was safe and effective in treating patients with uncomplicated influenza. It is active against influenza A and B, including strains resistant to neuraminidase inhibitors.

Approval of baloxavir marboxil was based on the CAPSTONE-1 trial (n=1436). Patients aged 12-64 years were randomized to receive baloxavir, oseltamivir, or placebo. Of the 1064 patients included in the intention-to-treat infected population, the median time to alleviation of symptoms was 53.7 hours in the baloxavir group compared to 80.2 hours for placebo (P<.001). Similar results were observed with oseltamivir compared to placebo. Additionally, the baloxavir group had a significant decrease in viral load after 1 day of treatment as compared to both the placebo (P<.05) and oseltamivir (P<.05) groups. It is undetermined whether the rapid drop in viral load decreases the risk of virus transmission. (N Engl J Med. 2018 Sep 6;379(10):913-923)

Krintafel (tafenoquine)

In July 2018, the FDA approved tafenoquine, an antiplasmodial 8-aminoquinoline derivative indicated for the radical cure (prevention of relapse) of Plasmodium vivax malaria in patients aged 16 years or older who are receiving appropriate antimalarial therapy for acute P vivax infection. The drug is active against all stages of the P vivax life cycle. Tafenoquine is administered as a single oral dose on the first or second day of appropriate antimalarial therapy (eg, chloroquine) for acute P vivax malaria. Approval was based on an international program of over 4000 participants.

In one of the clinical trials, 329 patients were randomly assigned to a treatment group (chloroquine plus tafenoquine 50 mg [n=55], 100 mg [n=57], 300 mg [n=57], 600 mg [n=56]; or to chloroquine plus primaquine [n=50]; or chloroquine alone [n=54]). Relapse-free efficacy at 6 months was 89.2% with tafenoquine 300 mg and 91.9% with tafenoquine 600 mg compared to chloroquine alone (37.5%). The results showed a significantly improved treatment difference, compared to chloroquine alone, of 51.7% (P<.0001) with tafenoquine 300 mg and 54.5% (P <.0001) with tafenoquine 600 mg. (Lancet. 2014 Mar 22;383(9922):1049-58)


Moxidectin is an antiparasitic drug approved by the FDA in June 2018 to treat onchocerciasis in patients aged 12 years or older. The World Health Organization initiated clinical trials for use in onchocerciasis in 2009. Moxidectin is closely related to ivermectin, but it has a more sustained reduction in microfilarial levels. Its plasma half-life is 20-43 days, and, thereby, it reduces and maintains low skin microfilarial density effectively. FDA approval was based on a double-blind, parallel-group, superiority trial (n=1472) that compared moxidectin (8 mg PO once) with ivermectin (150 mcg/kg PO once). The trial took place in Ghana, Liberia, and the Democratic Republic of the Congo. Results showed skin microfilarial loads (ie, parasite transmission reservoir) were lower from month 1 to month 18 after moxidectin treatment than after ivermectin treatment, with an 86% difference at month 12. Moxidectin would therefore be expected to reduce parasite transmission between treatment rounds more than ivermectin could, thus accelerating progress toward elimination. (Lancet. 2018 Oct 6;392(10154):1207-1216)

TPOXX (tecovirimat)

Tecovirimat is an antiviral protein inhibitor indicated for human smallpox disease caused by variola virus in adults and children who weigh more than 13 kg. It is available through the US government’s Strategic National Stockpile.

Effectiveness of tecovirimat against smallpox was established by studies in animals infected with viruses closely related to variola virus, which demonstrated higher survival rates than placebo. (N Engl J Med. 2018 Jul 5;379(1):44-53)


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