Drug Pipeline News

Each year in the United States, there are approximately 700 to 800 cases of NB, of which 90% are diagnosed before the age of 5 years. More than half of these cases are considered high-risk.

New data presented at the American Society of Hematology (ASH) Annual Meeting and Exposition revealed emicizumab-kxwh (Hemlibra; Genentech) controlled bleeding in babies up to 12 months while demonstrating safety and tolerability.

Lawrence Eichenfield, MD, discusses topical steroid, non-steroidal, and potentially new atopic dermatitis treatments for the pediatric population.

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

Sickle cell disease (SCD) patients aged 12 years and up can now be treated with exa-cel and lovo-cel, following simultaneous approval from the FDA on December 8, 2023.

The US approval makes VLA1553 (Ixchiq; Valneva) the world’s first licensed chikungunya vaccine.

This decision makes Owlet’s Dream Sock the first and only over-the-counter medical-grade pulse oximeter that is cleared for use in infants, according to the company.

Ustekinumab-auub is approved to treat indications that are currently approved for ustekinumab, including moderate to severe plaque psoriasis (candidates for phototherapy or systemic therapy) and for those with active psoriatic arthritis, aged 6 years and older.

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

The FDA has approved abatacept (Orencia) for patients aged ≥2 years with active psoriatic arthritis (PsA), a condition causing joint damage in pediatric patients, offering a new treatment option for juvenile PsA, benefiting young patients, caregivers, and healthcare professionals.

Longer-term data was recently released for dupilumab as a treatment for eosinophilic esophagitis (EoE) in children aged 1 to 11 years, demonstrating a similar safety and efficacy profile for up to 1 year of treatment. A supplemental Biologic License Application was accepted by the FDA in September 2023 with a Priority Review designation.

A usability study demonstrated that individuals older than 18 years could self-administer the intranasal treatment or administer it to eligible patients aged 2 to 49 years, when given instructions and no additional guidance.

A child with profound genetic hearing loss was dosed with an investigational otoferlin gene therapy (DB-OTO), the first patient dosed in a phase 1/2 trial evaluating the treatment. The child experienced improved auditory response at week 6 with no concerning safety signals.

Vamorolone, oral suspension 40 mg / ml, has been approved by the FDA to treat Duchenne muscular dystrophy (DMD) in patients aged 2 years and up, according to Santhera Pharmaceuticals.

The combination vaccine candidates demonstrated a safety profile consistent with Pfizer’s COVID-19 vaccine. A phase 3 trial is anticipated to commence in the coming months, according to a press release from Pfizer.

The Complete Response Letter (CRL) is based on additional efficacy data required by the FDA, according to Sanofi and Regeneron.

The topical gel is the first and only FDA-approved triple-combination, fixed-dose topical treatment for acne, and is set to be available in the first quarter of 2024, according to Bausch Health Companies Inc.

In a phase 2 clinical trial, the safety and efficacy of vosoritide was similar in children aged younger than 5 years to those older than 5 years. 

The FDA approval could simplify meningococcal vaccine schedules while providing broad serogroup coverage.

A significant improvement of stimulated C-peptide levels at week 78 for patients newly diagnosed with type 1 diabetes (T1D) was observed for teplizumab-treated patients compared to placebo. Significant differences between groups for insulin dose, percentage of time in target glucose range, and change in glycated hemoglobin were not observed.

Of the patients that responded to lebrikizumab at week 16 in the phase 3 trials ADvocate 1 and ADvocate 2, 84% achieved a clinically meaningful response in at least 1 domain of the disease (mild signs, symptoms, or quality of life impact) at 52 weeks.

Patients aged 12 years and up with completely resected Stage IIB/C melanoma can now be treated (adjuvant) with FDA-approved nivolumab (Opdivo; Bristol Meyers Squibb Company).

A rapid reduction in pruritis as early as 24 hours after first application was announced as new positive data from a pair of identical, phase 3 studies of tapinarof cream 1% in children as young as 2 years and adults with atopic dermatitis (AD).

Crinecerfont, an investigational, oral, selective corticotropin-releasing factory type 1 receptor antagonist, achieved the primary and key secondary endpoints in a phase 3 study to treat congenital adrenal hyperplasia due to 21-hydroxylase deficiency in children aged 2 to 17 years.

Once-daily, topical roflumilast cream 0.3% has been approved by the FDA for children aged 6 to 11 years for the treatment of plaque psoriasis based on a data from a 4-week Maximal Usage Systemic Exposure study, and safety and efficacy data from a pair of phase 3 trials in adults.

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

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.