FDA Drug Approvals: Pediatrics — Year in Review 2018

Mary L Windle, PharmD


January 15, 2019

More New Drug Approvals

Symdeko (tezacaftor/ivacaftor)

Tezacaftor/ivacaftor is indicated for patients aged 12 years or older with cystic fibrosis who are homozygous for the F508del mutation or who have at least one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to tezacaftor/ivacaftor based on in vitro data and/or clinical evidence. It is available as a copackaged fixed-dose combination tablet of tezacaftor 100 mg and ivacaftor 150 mg, plus a separate ivacaftor 150-mg tablet.

Approval was based on the EVOLVE and EXPAND phase 3 clinical trials. Results showed measured improvements across multiple disease measures, including lung function and pulmonary exacerbations, compared with ivacaftor monotherapy. (N Engl J Med. 2017 Nov 23;377(21):2013-2023; N Engl J Med. 2017 Nov 23;377(21):2024-2035)

Oxervate (cenegermin)

Cenegermin is a recombinant nerve growth factor approved for neurotrophic keratitis.

Approval was based on the REPARO study (n=156). Patients were randomized 1:1:1 to cenegermin 10 mcg/mL, 20 mcg/mL, or vehicle. Safety and effectiveness in children aged 2 years or older was supported by evidence from adequate and well-controlled trials in adults with additional safety data in children.

At week 4, 19.6% of vehicle-treated patients achieved corneal healing (<0.5-mm lesion staining) compared with 54.9% receiving cenegermin 10 mcg/mL (+35.3%; 97.06% CI, 15.88-54.71; P<.001) and 58% receiving cenegermin 20 mcg/mL (P<.001).

At week 8, 43.1% of vehicle-treated patients achieved corneal healing, compared with 74.5% receiving cenegermin 10 mcg/mL (P=.001) and 74% receiving 20 mcg/mL (P=.002). Post hoc analysis of corneal healing by the more conservative measure (0-mm lesion staining and no other persistent staining) maintained statistically significant differences between cenegermin and vehicle at weeks 4 and 8. More than 96% of patients who healed after controlled cenegermin treatment remained recurrence free during a 48-week follow-up. (Ophthalmology 2018 Sep;125(9):1332-1343)

Crysvita (burosumab)

Burosumab is indicated for adults and children with X-linked hypophosphatemia. Burosumab is a recombinant fully human monoclonal IgG1 antibody against the phosphaturic hormone, fibroblast growth factor 23 (FGF23). X-linked hypophosphatemia is a bone disease characterized by phosphate-wasting due to excess activity of FGF23, which results in suppression of renal tubular phosphate reabsorption and renal production of 1,25 dihydroxy vitamin D.

Approval for children was based on a 64-week, randomized, open-label study in 52 patients aged 5-12 years, which showed that treatment with burosumab improved rickets, increased serum phosphorus levels, decreased serum alkaline phosphatase activity, and increased growth. The indication is also supported by 40-week data from an open-label study in 13 patients aged 1-4 years. In these patients, burosumab improved rickets and lower-limb deformity, increased serum phosphorus levels, and decreased serum alkaline phosphatase activity. (N Engl J Med. 2018 May 24;378(21):1987-1998)

Approval in adults with X-linked hypophosphatemia (n=134) was supported by a randomized, double-blind, placebo-controlled study. Burosumab treatment resulted in a higher proportion of patients achieving serum phosphorus levels above the lower limit of normal compared with placebo (P<.0001). A higher rate of complete healing of active fractures and pseudofractures was also observed compared with placebo. (J Bone Miner Res. 2018 Aug;33(8):1383-1393)

Omegaven (fish oil triglycerides)

Fish oil triglycerides is a parenteral emulsion indicated as a source of calories and fatty acids in pediatric patients with parenteral nutrition–associated cholestasis (PNAC).

Effectiveness was established in two open-label clinical trials of 82 pediatric patients (aged 3-42 weeks), including preterm neonates with estimated gestational age of older than 24 weeks at birth. (Omegaven prescribing information)


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