Drug Pipeline News

Lawrence Eichenfield, MD, highlights the sNDA submission of tapinarof cream, 1% for the topical treatment of atopic dermatitis in children aged 2 years and older.

The sNDA submission follows additional positive topline data that was presented in Janurary 2024, highlighting an open-label, long-term extension study evaluating tapinarof cream, 1%.

The twice-daily oral corticosteroid is indicated for 12 weeks of treatment.

Vivian Hernandez-Truillo, MD, FAAP, FAAAAI, FACAAI; and Theresa Bingemann, MD, provide reaction and commentary regarding recently FDA-approved dupilumab to treat EoE in pediatric patients aged 1 to 11 years.

In this Contemporary Pediatrics Q+A interview, Weily Soong, MD, breaks down FDA-approved tralokinumab-ldrm for patients aged 12 to 17 years with moderate-to-severe atopic dermatitis.

The approval makes dupilumab the first and only treatment specifically indicated for eosinophilic esophagitis (EoE) patients aged 1 to 11 years that weigh at least 33 lbs (15 kg).

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

Compared to placebo, linaclotide demonstrated a statistically significant and clinically meaningful improvement in spontaneous bowel movement(s) (SBM) frequency rate, and was subsequently approved by the FDA in June 2023.

MediWound Ltd is seeking to expand the label for NexoBrid, already approved for eschar removal in adult patients, to the pediatric indication after the FDA accepted a supplemental Biologics Application for this patient population.

Marstacimab reduced the annualized bleeding rate (ABR) by 35% and nearly 92%, respectively, compared to routine prophylaxis and on-demand treatment in hemophilia A and B patients without inhibitors, according to Pfizer.

Berdazimer gel, 10.3% demonstrated positive, topline data in the phase 3 randomized clinical B-SIMPLE4 trial (NCT04535531) that featured a total of 891 patients aged 6 months or older who had 3 to 709 raised molluscum contagiosum lesions.

Biopharmaceutical company, Invivyd, made the request to the FDA for its investigational antibody, VYD222.

Birch triterpenes (FILSUVEZ; Chiesi Global Rare Diseases) is the first approved treatment for wounds associated with junctional epidermolysis bullosa (JEB), according to the developer.

Seborrheic dermatitis patients aged 9 years and older can now use roflumilast foam 0.3% following its approval by the FDA. 

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.