FDA Drug Approvals — 2019 Midyear Review

Mary L Windle, PharmD


August 09, 2019

Pediatric Agents

Dengvaxia (dengue vaccine)

Dengue vaccine was approved by the FDA for individuals aged 9-16 years with laboratory-confirmed previous dengue infection and living in endemic areas. It elicits dengue-specific immune responses against the 4 dengue virus serotypes (ie, serotypes 1, 2, 3, and 4). It is only approved for individuals previously infected by any dengue virus serotype or for whom this information is unknown. Those not previously infected are at increased risk for severe dengue disease when vaccinated and subsequently infected with dengue virus.

The approval was based on data from 2 placebo-controlled studies in patients (N>35,000) living in dengue-endemic areas. Patients were randomized 2:1 to receive either the vaccine or saline placebo and monitored for symptomatic virologically confirmed dengue (VCD) starting at day 0. Vaccine efficacy was assessed beginning 28 days after the third vaccination for 12 months. The vaccine was approximately 76% effective in preventing symptomatic VCD disease among patients aged 9-16 years who were seropositive for dengue at baseline.[11]

Zolgensma (onasemnogene abeparvovec)

Onasemnogene abeparvovec is a recombinant AAV9-based gene therapy designed to deliver a copy of the gene encoding the human survival motor neuron (SMN) protein. It is indicated for gene replacement therapy in children aged 2 years or younger with spinal muscular atrophy (SMA) type 1 (also called Werdnig-Hoffman disease) who have biallelic mutation in the survival motor neuron 1 (SNM1) gene.

Approval was based on the ongoing phase 3 STR1VE trial and the completed phase 1 START trial. Fifteen patients with SMA type 1 received a single dose of intravenous adeno-associated virus serotype 9 carrying SMN complementary DNA encoding the missing SMN protein. As of the data cutoff, all 15 patients were alive and event-free at age 20 months, as compared with a rate of survival of 8% in a historical cohort. In the high-dose cohort, a rapid increase from baseline in the score on the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) scale followed gene delivery, with an increase of 9.8 points at 1 month and 15.4 points at 3 months, as compared with a decline in this score in a historical cohort. Of the 12 patients who had received the high dose, 11 sat unassisted, 9 rolled over, 11 fed orally and could speak, and 2 walked independently. Elevated serum aminotransferase levels occurred in 4 patients and were attenuated by prednisolone.[14]

Interim data analysis from the ongoing phase 3 STR1VE trial described 21 (95%) of 22 patients were alive and event-free. The median age was 9.5 months, with 6 (86%) of 7 patients aged 0.5 months or older surviving event-free. Interim results also showed ongoing improvement of motor milestones (eg, holding head erect, rolling over, sitting without support).[15,16]

Other notable pediatrics approvals

Victoza (liraglutide) was approved as an adjunct to diet and exercise to improve glycemic control in children aged 10 years or older with type 2 diabetes mellitus.

Benlysta (belimumab) is the first drug approved for children aged 5 years or older with systemic lupus erythematosus.

Jakafi (ruxolitinib) gained approval for steroid-refractory acute graft versus host disease in patients aged 12 years or older.

Livalo (pitavastatin) was approved for heterozygous familial hypercholesterolemia in children aged 8 years or older.

Corlanor (ivabradine) was approved for the treatment of children aged 6 months or older with stable symptomatic heart failure due to dilated cardiomyopathy who are in sinus rhythm with an elevated heart rate.

Gattex (teduglutide) is now approved for children aged 1 year or older with short bowel syndrome.

Mavyret (glecaprevir/pibrentasvir) was approved for adolescents to treat hepatitis C viral infection.

Symdeko (tezacaftor/ivacaftor) has a new lower dosage form and an expanded indication for children with cystic fibrosis aged 6 years or older.

Kalydeco (ivacaftor) was approved for cystic fibrosis in children as young as 6 months.

Fragmin (dalteparin) was approved for the treatment of symptomatic venous thromboembolism to decrease recurrence in children aged 1 month or older.

Avycaz (ceftazidime/avibactam) was approved for complicated urinary tract infections, including pyelonephritis, in patients aged 3 months or older.

Acyclovir dosing information from the National Institutes of Health lends clarity for newborns and premature infants with herpes simplex virus infection caused by adult genital herpes following vaginal delivery.

Dupixent (dupilumab) was approved for patients aged 12 years or older with moderate-to-severe atopic dermatitis.

Ruzurgi (amifampridine) is a new brand with an approved indication for children as young as 6 years with Lambert-Eaton myasthenic syndrome.

Lyrica (pregabalin) indication for partial-onset seizures was expanded to include children as young as 1 month.

Nayzilam (midazolam intranasal) was approved in children aged 12 years or older for acute treatment of intermittent, stereotypic episodes of frequent seizure activity.

Ravicti (glycerol phenylbutyrate) is a nitrogen-binding agent for long-term management of adults and children (including newborns) with urea cycle disorders.

Emflaza (deflazacort) is indicated for the treatment of Duchenne muscular dystrophy in patients aged 2 years or older.


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