Drug Pipeline News

The FDA Advisory Committee recently voted in support of approval for Pfizer's maternal immunization vaccine to help prevent RSV in infants. In this Contemporary Pediatrics® interview, Tina Tan, MD, FAAP, FIDSA, FPIDS, pediatric infectious diseases attending, Ann & Robert H. Lurie Children's Hospital, Chicago, explains what this means for this patient population ahead of an expected FDA decision in August, 2023.

This formulation of ibuprofen is the only non-opioid product approved to treat pain in infants that is delivered through injection, according to Cumberland Pharmaceuticals.

The once-daily topical cream for the treatment of atopic dermatitis (AD) has demonstrated positive topline results in 2 phase 3 studies in adults and children aged 2 years and up. According to Dermavant, a supplemental new drug application (sNDA) filed with the FDA is anticipated in Q1 of 2024.

The REFLECT Scoliosis Correction System from Globus Medical is intended to correct progressive scoliosis in pediatric patients while simultaneously maintaining stability and motion as well as allowing for future modulated growth.

Nirsevimab’s efficacy against infant hospitalizations due to respiratory syncytial virus was reinforced after a phase 3b clinical trial, demonstrating an 83% reduction in infant hospitalizations.

With the recent FDA-approved indication for children aged 2.5 years and older, somapacitan-beco is the first and only once-weekly growth hormone (GH) for children and adults.

According to Pfizer, the company’s pneumococcal conjugate vaccine Prevnar 20 has been approved by the FDA to treat infants and children aged 6 weeks to 17 years for the prevention of invasive pneumococcal disease (IPD).

According to a phase 2 study, remission of macrophage activation syndrome (MAS) secondary to systemic juvenile idiopathic arthritis (sJIA) or adult-onset Still’s disease (AOSD) was achieved in patients taking emapalumab who failed high-dose glucocorticoids.

Ritlecitinib could be a suitable treatment option for alopecia areata patients aged 12 years and older following positive results from a recent trial.

Roflumilast is a once-daily, topical phosphodiesterase type 4 (PDE4) inhibitor under development for treatment of inflammatory dermatoses, focusing on the hair-bearing areas including the face, scalp, and trunk.

Following voluntary market withdrawal from Covis Pharma, the FDA has decided to withdraw the approval of Makena and its generics for reducing the risk of preterm birth.

Data suggest confirmatory trials conducted for drugs granted accelerated approval by the FDA have taken a median 3.5 years to complete since 2012.

Leniolisib is the first and only treatment approved in the United States for APDS, a rare primary immunodeficiency.

Pediatric patients with BRAF V600E low-grade glioma as young as 1 year can now be treated with dabrafenib plus trametinib following its FDA approval.

Evkeeza is the first angiopoietin-like 3 (ANGPTL3) inhibitor indicated for patients as young at 5 years to control high levels of low-density lipoprotein cholesterol (LDL-C) as a result of HoFH.

A recent study indicated that lebrikizumab treatment for 16 weeks was effective for adolescents and adults with moderate-to-severe atopic dermatitis.

Results from a recent phase 3 clinical trial demonstrated topline safety and efficacy results for tapinarof cream, 1% (VTAMA; Dermavant) as a potential treatment for atopic dermatitis in patients 2 years and older.

If approved by the FDA, this 5-in-1 investigational candidate could provide the broadest meningococcal serogroup coverage.

The FDA has approved trofinetide (Daybue; Acadia Pharmaceuticals) for treating Rett syndrome in patients aged 2 years and older.

Covis, the manufacturer of the controversial preterm birth prevention drug Makena, has announced it will voluntarily withdraw the drug from the market after recommendations from the Center for Drug Evaluation and Research.

A supplemental New Drug Application for empagliflozin (Jardiance; Boehringer Ingelheim and Eli Lilly and Company) in pediatric patients with type 2 diabetes has been accepted by the FDA.

Following positive results from a phase 3 clinical trial, the FDA has accepted a New Drug Application for berdazimer gel, 10.3% (Novan) for treating patients with molluscum contagiosum.

A supplemental Biologics License Application has been accepted by the FDA for treating adolescents and adults with chronic spontaneous urticaria.

Velmanase alfa-tycv (Lamzede, Chiesi Global Rare Diseases) has received approval by the FDA for treating alpha-mannosidosis in adult and pediatric patients.

Omaveloxolone (SKYCLARYS, Reata Pharmaceuticals) has received FDA approval for treating Friedreich’s ataxia in patients aged 16 years and older.

The wearable device received its original FDA go-ahead in 2019, and has since demonstrated effectiveness in preventive and acute care for migraine treatment in adolescents and adults.

A new drug application for VP-102 for treating molluscum contagiosum has been accepted by the FDA.

Previously known as efanesoctocog alfa, once-weekly ALTUVIIIO is indicated for routine prophylaxis and on-demand treatment to control bleeding episodes for adults and children with hemophilia A.

A New Drug Application has been submitted to the FDA for potential approval of roflumilast foam 0.3% for the treatment of seborrheic dermatitis in adults and adolescents.

The federal agency set a Prescription Drug User Fee Act (PDUFA) action date of August 2023.