News for Pediatric Healthcare Providers From the Food and Drug Administration

Marcia L. Buck, PharmD, FCCP, FPPAG, BCPPS


Pediatr Pharm. 2018;24(4) 

In This Article

New Pediatric Indications

A total of 54 new pediatric labeling changes were made by the FDA in 2017, with another six already announced in 2018.[13] These included several new pediatric indications, as well as warnings and other safety data.


Approval for the use of abatacept (Orencia®) in the treatment of severely active polyarticular juvenile idiopathic arthritis (pJIA) was extended to patients 2 years of age and older. Supporting data came from a phase 3 global 24-month open-label study.[14] Patients achieved target steady-state serum trough concentrations and showed improvement in pJIA symptoms.


The FDA extended the approval for aliskiren (Tekturna®) for the treatment of hypertension to children 6 years of age and older. The approval was based on two randomized double-blind trials in children 6–17 years of age.


Several agents previously approved for the management of HIV-1 infection in adults have received approval for use in pediatric patients. The combinations of emtricitabine and tenofovir (Descovy®) and elvitegravir, cobicistat, emtricitabine, and tenofovir (Genvoya®) were approved for use in children weighing at least 25 kg, while raltegravir (Isentress® HD) and the combination of elvitegravir, cobicistat, emtricitabine, and tenofovir (Stribild®) was approved for patients weighing at least 35 kg, while the combination of abacavir, dolutegravir, and lamivudine (Triumeq®) was approved for use in patients weighing at least 40 kg. Raltegravir (Isentress®) was also approved for use in HIV-exposed neonates from 0–4 weeks of age and weighing at least 2 kg.


The approval of cysteamine bitartrate (Procysbi®) to treat nephropathic cystinosis was expanded to include children as young as 1 year of ager. It was previously approved for 2 years and older.


The FDA extended the approval of daptomycin (Cubicin®) for the treatment of Staphylococcus aureus bacteremia and complicated skin and skin structure infections (cSSSI) to include children 1–17 years of age. Supporting data came from several studies, including a randomized trial in 389 children with cSSSI, where the clinical success rate for daptomycin (91%) was similar to that seen with clindamycin or vancomycin.[15]


Approval for the use of eslicarbazepine (Aptiom®) for partial-onset seizures was extended to include children 4 to 17 years of age. The extension was based on extrapolation from studies in adults and older children.


Oral everolimus tablets for the preparation of a suspension (Afinitor Disperz) were approved earlier this month for use in adults and children 2 years of age and older with tuberous sclerosis complex-associated partial-onset seizures. The phase 3 placebo-controlled EXIST-3 trial demonstrated a significant reduction in seizure frequency with everolimus as adjunctive therapy.


The approval for fosphenytoin (Cerebyx®) was extended to include patients from birth to 17 years of age. Fosphenytoin is indicated for the treatment of generalized tonic-clonic status epilepticus, for the prevention and treatment of seizures occurring during neurosurgery, and as a short-term replacement for oral phenytoin.

Inhaled Products for Asthma

Several products received an extension for younger children in 2017. Tiotropium bromide (Spiriva® inhalation spray) and budesonide/formoterol fumarate (Symbicort® inhalation aerosol) were approved for children 6 years of age and older. Fluticasone propionate (ArmonAir™ RespiClick) and fluticasone propionate/salmeterol (AirDuo™ RespiClick) were approved for children 12 years and older.


Use of oral lacosamide (Vimpat®) for the treatment of partial-onset seizures was extended to children 4 years of age and older. Approval was based on safety and efficacy data from over 300 children ranging from 4 to 17 years of age. Safety of the injection has not yet been established in children.


The approval for luliconazole 1% cream (Luzu™) use in the treatment of tinea pedis and tinea cruris has been extended to include use in patients 2 years of age and older. Supporting data came from pharmacokinetic and safety studies showing comparable results to studies in adults, as well as a placebo-controlled trial.


In March, the FDA approved a supplemental new drug application to extend the indications for lurasidone (Latuda®) for the treatment of major depressive episodes associated with bipolar disorder in children and adolescents between 10 and 17 years of age. Lurasidone is an atypical antipsychotic agent with high-affinity binding at serotonin 5-HT7 receptors that is comparable to its affinity for dopamine-2 and 5-HT2A receptors. In addition, it has moderate affinity for 5-HT1A receptors, and minimal to no affinity for H1 and M1 receptors. Approval was based on a randomized, double-blind placebo-controlled phase 3 study in 347 children and adolescents.[16] Lurasidone produced a significant improvement in Children's Depression Rating Scale, Revised (CDRS-R) scores, with a CDRS-R score of -21.0 after treatment, compared to -1.05 in the controls (effect size 0.45, p < 0.0001) and a significant improvement on Clinical Global Impression-Bipolar Version, Severity of Illness scores (-1.49 versus 0.45, effect size 0.44, p < 0.0001).

Ophthalmic Drops

Several ophthalmic products received pediatric indications in the past year, including: brimonidine (Lumify™) for eye irritation, cetirizine (Zerviate™) for allergic conjunctivitis, and the combination of phenylephrine and ketorolac (Omidria®) for prevention of intraoperative miosis and reduction of postoperative pain. The age range for ciprofloxacin (Ciloxan®), gatifloxacin (Zymar®), and moxifloxacin (Vigamox®) for treatment of bacterial conjunctivitis was extended to patients 1 month of age and older.


Use of peramivir (Rapivab®) in the treatment of influenza was extended to include children 2 years of age and older. The drug is given as a single IV dose to patients who have been symptomatic for no more than 2 days.


The approval for perampanel (Fycompa™) as monotherapy for the treatment of partial-onset seizures with or without secondarily generalized seizures was extended to include patients 12 years of age and older. Perampanel had previously been approved as adjunctive therapy in this age range.


The use of ustekinumab (Stelara®) in patients with psoriasis has been extended to adolescents 12 years of age and older. Data from a multicenter, randomized, double-blind, placebo-controlled trial in 110 adolescents showed similar benefit and adverse effects as reported in adult studies.