Pediatric Updates From the Food and Drug Administration

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


Pediatr Pharm. 2019;25(4) 

In This Article

Drugs in the FDA Pipeline

A wide variety of new drug applications (NDAs) were submitted to the FDA in 2018 and the first months of 2019. Several are expected to be approved for use in children at the time of marketing, while others have the potential for use in the pediatric population but will require subsequent study.


The review of a third new antiseizure medication for the treatment of patients with Dravet syndrome, fenfluramine (Fintepla®), was recently refused by the FDA. The decision to postpone review was based on a failure to include the necessary data to assess the effects of chronic administration of fenfluramine and the inclusion of an incorrect version of a clinical dataset which prevented completion of the review process. The manufacturer is currently working with the FDA to comply with the request for the additional documents and anticipates review of their NDA later this year.[8]


An NDA for a second exon-skipping therapy for patients with Duchenne muscular dystrophy (DMD), golodirsen (SRP-4053), was submitted to the FDA in February. Golodirsen binds to exon 53 of dystrophin pre-mRNA. Skipping this exon during mRNA processing allows production of truncated, but functional, dystrophin protein. The efficacy and safety of the drug were established in 25 boys with DMD known to have a deletion of the dystrophin gene amenable to exon 53 skipping. The drug produced both an increase in the quantity of dystrophin expression compared to baseline and increased dystrophin intensity as measured by immunohistochemistry. Golodirsen is currently being studied in the ESSENCE study, a global, randomized double-blind, placebo-controlled trial assessing the safety and efficacy of golodirsen with casimersen, an exon 45 skipping agent also be developed by the manufacturer.


The erythroid maturation agent luspatercept is a first-in-class treatment for anemia associated with myelodysplastic syndromes and beta-thalassemia. While not yet studied in children, a recently published phase 2 trial in adults with beta-thalassemia included patients as young as 20 years of age. The phase 3 BELIEVE trial also included young adults.[9] This randomized, double-blind, placebo-controlled study was conducted at 65 sites in 15 countries and enrolled 336 patients, with a median age of 30 years. Patients received luspatercept 1 mg/kg or placebo by subcutaneous injection every 21 days for up to 48 weeks in addition to standard supportive care. The results of these trials, as well as those of a phase 3 trial in MSD-related anemia were included in the NDA.

Minocycline Foam

A new topical minocycline foam is currently being evaluated by the FDA for the treatment of moderate-to-severe acne vulgaris in adults and children 9 years of age and older. The NDA includes data from two placebo-controlled phase 3 trials.[10] In both studies, the drug met the primary endpoints with statistically significant difference in improvement in inflammatory lesion count and in Investigator Global Assessment (IGA) treatment scores.

Oxycodone Extended-release Tablets

The number of abuse-deterrent opioid products continued to grow over the past year as prescribers look for new ways to mitigate the risks for opioid misuse. Intellipharmaceutics International recently submitted an NDA for their new oxycodone extended-release tablet (Rexista®) formulated with their proprietary abuse-deterrent design, referred to as the novel point of divergence drug delivery system (nPODDDS™). This system alters the product to discourage chewing or licking of the tablet and makes crushing of the tablet difficult, reducing the likelihood of insufflation, inhalation, or injection. Apart from this change, the product will be equivalent to traditional oxycodone tablets.