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

Drugs in the FDA Pipeline

A wide variety of new drug applications (NDAs) were submitted to the Food and Drug Administration in 2017. Several of these agents are targeted at or have the potential for use in the pediatric population.


In the third quarter of 2017, GW Pharmaceuticals submitted an NDA for its proprietary cannabinoid product (Epildolex®) as an adjunctive therapy for the treatment of seizures in children and adults with Lennox Gastaut syndrome (LGS) or Dravet syndrome. Data supporting approval comes from three phase 3 trials in more than 1,000 patients. The submission has been granted both Rare Pediatric Disease and Orphan Drug designations, as well as a fast track designation.


Sunovion Pharmaceuticals has submitted an NDA for dasotraline, its new treatment for attention deficit hyperactivity disorder (ADHD). Dasotraline is a unique dual-acting dopamine and norepinephrine reuptake inhibitor. The NDA includes the results of placebo-controlled safety and efficacy studies in more than 2,000 children and adults, including two year-long studies.


A second exon-skipping therapy for patients with DMD will likely be undergoing assessment by the FDA this year. Golodirsen binds to exon 53 of dystrophin pre-mRNA, skipping this exon during mRNA processing and allowing for the production of truncated, but functional, dystrophin protein. The ESSENCE study, a global phase 3 randomized, double-blind, placebo-controlled trial, is currently enrolling patients.


At the end of 2017, GSK submitted an NDA for tafenoquine use in the prevention of relapse of Plasmodium vivax malaria in patients 16 years of age and older. Administered as a single dose, tafenoquine provides a clinical cure, preventing future relapse. Tafenoquine was developed through a partnership between GSK and the Medicines for Malaria Venture. Development of the drug began in 2008; it was given Breakthrough Therapy status by the FDA in 2013.