Drug Pipeline News

The approval of the prescription injection for children aged 9 years and up is based on data from a phase 2 and an ongoing phase 3 trial extension study, that demonstrated the lowering of urinary oxalate levels for individuals with primary hyperoxaluria type 1 (PH1).

Individuals 12 years and older can now receive the Novavax COVID-19 Vaccine, Adjuvanted (2023-2024 Formula) (NVX-CoV2601) to protect against variants that are currently circulating, after receiving an Emergency Use Authorization from the FDA.

Tocilizumab-bavi (Tofidence; Biogen) demonstrated a biosimilarity to tocilizumab (Actemra; Genentech) based on multifaceted clinical and non-clinical data for polyarticular juvenile idiopathic arthritis (PJIA) and systemic juvenile idiopathic arthritis (sJIA) in children aged 2 years and up. This makes it the first tocilizumab biosimilar to be FDA-approved in the United States, according to Biogen.

Review some of the top stories from the Contemporary Pediatrics website over the last week, and catch up on anything you may have missed.

Results from the phase 3 MIRA-4 pediatric trial demonstrated a favorable safety and tolerability profile with no reported adverse events. Rupa Wong, MD, offers thoughts on the approval, and why it may not change day-to-day practice when it comes to the pediatric population.

Positive topline results from a pair of identical phase 3 trials support the submission of a supplemental New Drug Application to the FDA for Arcutis Biotherapeutics’ roflumilast cream 0.15%, a once-daily topical to treat mild to moderate atopic dermatitis (AD) in children 6 years or older.

Based on recent positive phase 3 results, Arcutis Biotherapeutics,. intends to submit a supplemental New Drug Application with the FDA for roflumilast cream 0.05% to treat mild to moderate atopic dermatitis in children aged 2 to 5 years.

The FDA issued the Complete Response Letter (CRL) based on the request for a completed pharmacokinetic/pharmacodynamic study to assess how repeat doses of the epinephrine nasal spray compare to repeat doses of an epinephrine injection product. ARS Pharma is set to file a Formal Dispute Resolution Request to appeal the CRL.

A supplemental biologics license application for ALTUVIIIO (Antihemophilic factor; Sanofi) has been accepted by the FDA for use in patients aged 12 years and younger with severe hemophilia A.

Review some of the top stories from the Contemporary Pediatrics website over the last week, and catch up on anything you may have missed.

In this Contemporary Pediatrics interview, Bernard A. Cohen, MD, discusses VP-102, approved by the FDA on July 22, 2023, to treat molluscum contagiosum in pediatric patients 2 years and up.

The effects of GARDASIL 9 (Human Papillomavirus 9-valent Vaccine, Recombinant) in patients aged 9 to 15 years has indicated efficacy, according to a recent long-term follow-up study.

Patients 6 years and up with ADHD now have alternate options for prescription lisdexamfetamine dimesylate (Vyvanse; Takeda Pharmaceuticals USA Inc), after the FDA-approval of multiple generic options.

This new data is consistent with previous results on efficacy and on safety shown in global phase 3 studies that had enrolled both adults and adolescents.

Approved for use at 32 weeks through 36 weeks gestation, Pfizer’s maternal respiratory syncytial vaccine (Abrysvo), is delivered through a single dose injection to the muscle, and is the first vaccine for use in pregnant individuals to prevent lower respiratory tract disease (LRTD) and severe LRTD because of RSV in infants (birth to 6 months).

Regeneron states all 10 patients achieved normalization of serum albumin and serum IgG concentrations by week 12 in a phase 2/3 trial that investigated the safety and efficacy of pozelimab-bbfg. These concentrations were maintained through 72 weeks.

Palovarotene is now the first and only treatment for the ultra-rare bone disease fibrodysplasia ossificans progressive (FOP), which impacts approximately 400 people in the United States and 900 globally.

Basis for a Biologics License Application submission to the FDA is being built for PXVX0317, following topline results demonstrated in a pair of phase 3 trials, including 1 trial featuring adolescents and adults aged 12 to 64 years.

The resubmission, which included new long-term follow-up data and a post-hoc propensity match study, was issued a complete response letter by the federal agency, which requires “more data to support marketing approval,” according to a press release from Mesoblast Limited.

The ACIP voted in unanimous fashion, 10 to 0, to recommend routine use of nirsevimab-alip for newborns and infants younger than 8 months, born during or entering the first RSV season according to a press release from Sanofi.

Ervebo has been approved by the FDA to prevent the Ebola virus for individuals aged 12 months and older, after being initially approved for those 18 years and older in 2019.

If nintedanib is approved by the FDA, it would be the first and only treatment for patients aged 6 to 17 with fibrosing interstitial lung disease (ILD).

Results from a pivotal phase 2 clinical trial revealed axatilimab met the primary outcome in all cohorts for graft versus host disease (GVHD) in pediatric and adult patients. Based on its safety and efficacy profile demonstrated in clinical trials, Syndax and Incyte intend to file a biologics license application (BLA) for axatilimab with the FDA by the end of 2023.

Following the resubmission of a new drug application, the FDA has approved VP-102 to treat molluscum contagiosum, a contagious skin disease transmitted through direct skin-to-skin contact that primarily affects children.

According to Pfizer, the investigational vaccine to protect against Group B Streptococcus (GBS) generated maternal antibody responses against 6 capsular polysaccharide serotypes and efficiently transferred antibodies to the infants. The announcement of this phase 2 data comes in July, which is International Group B Streptococcus Month.

The approval follows a unanimous vote of support from the FDA Antimicrobial Drugs Advisory Committee of nirsevimab-alip’s favorable benefit-risk profile for the prevention of Respiratory Syncytial Virus (RSV) lower respiratory tract disease (LRTD) in newborns and infants amid their first RSV season.

GLP-1 receptor agonists, used to treat diabetes, are now also being used for obesity in children and adolescents.

Norgestrel (Opill; HRA Pharma) is the first nonprescription oral contraceptive approved for use in the United States.

Improvement in the left ventricular ejection fraction (LVEF) and statistically significant benefit in the Performance of the Upper Limb (PUL v2.0) scale were observed following 2 years of CAP-1002 treatment in Duchenne muscular dystrophy (DMD) patients.

According to Italfarmaco Group, a New Drug Application (NDA) for givinostat has been accepted and granted priority review by the FDA to treat Duchenne muscular dystrophy (DMD) patients, following positive topline phase 3 trial results.