Contemporary Pediatrics week in review: September 28-October 2, 2026

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

Thank you for visiting the Contemporary Pediatrics® website. Take a look at some of our top stories from last week (Monday, September 28 to Friday, October 2, 2026) and click each link to read and watch anything you may have missed.

1.) FDA approves tiratricol as first treatment for peripheral thyrotoxicosis in MCT8 deficiency

The FDA approved tiratricol (Emcitate), an oral liquid T3 analogue, as the first therapy for peripheral thyrotoxicosis in patients with MCT8 deficiency, also known as Allan-Herndon-Dudley syndrome. The approval drew on the ReTRIACt randomized withdrawal trial, in which 15 males age 4 and older showed a statistically significant reduction in T3 levels on continued tiratricol versus placebo over 30 days (P = .034), along with open-label data from Triac Trial I showing mean serum T3 dropping from 4.97 to 1.82 nmol/L over 12 months and resting heart rate falling by roughly 9 beats per minute.

The approved indication addresses only the peripheral thyrotoxicosis component of the disease, not the severe neurodevelopmental impairment that drives most of its burden; neurodevelopmental endpoints in a companion trial did not reach statistical significance, and the pivotal evidence itself is limited in size and duration. Whether earlier treatment can change children's developmental trajectories remains an open question.

2.) FDA approves Camzyos for pediatric patients with symptomatic obstructive hypertrophic cardiomyopathy

Mavacamten (Camzyos), a selective cardiac myosin inhibitor, became the first disease-targeted pharmacologic option approved for adolescents with symptomatic obstructive hypertrophic cardiomyopathy, for patients weighing at least 30 kg. In the phase 3 SCOUT-HCM trial of 44 adolescents (ages 12-17, mean age 14.6), the drug reduced left ventricular outflow tract gradient by 48.5 mm Hg versus 0.5 mm Hg with placebo, a between-group difference of 48.0 mm Hg (P < .001).

Adverse event rates were similar between groups, with two serious adverse events in each arm, no ejection fraction declines below 50%, and no deaths. The approval is limited to symptomatic patients, so not every adolescent with hypertrophic cardiomyopathy will be a candidate.

3.) FDA approves expandable Autus valve for congenital pulmonary valve disease

The FDA approved the Autus Size-Adjustable Valve, the first US-approved surgically implanted pulmonary valve with polymeric leaflets and the first heart valve designed to be expanded after implantation as a child grows. Edwards Lifesciences holds the approval for the device, developed by Autus Valve Technologies. In a 62-patient study across 12 US sites, all enrollees had successful implantation, and at 6-month follow-up among the first 60 patients there were no deaths, strokes, or clot-related complications, though 3 patients had asymptomatic valve frame fractures and 2 had reduced mobility in one leaflet. The valve starts at about 13 mm and can be expanded via balloon catheter to 22 mm over time.

The FDA noted that clinical experience with the expansion procedure itself remains limited, and longer-term data are still needed to confirm durability and whether expansion truly reduces the number of future surgeries.

4.) FDA approves zilurgisertib for fibrodysplasia ossificans progressiva in patients aged 12 years and older

Zilurgisertib (Atebrioz), an oral ALK2 inhibitor taken once daily at 100 mg, was approved for fibrodysplasia ossificans progressiva in patients 12 and older, becoming the third FDA-approved therapy for the ultra-rare bone disorder and the first fixed-dose oral option. In the 63-patient PROGRESS phase 2 study, total new heterotopic ossification volume fell by 3.2 cm³ with zilurgisertib versus a 24.6 cm³ increase with placebo through week 24.

The prespecified primary endpoint, the proportion of patients developing new lesions, favored treatment (3.1% versus 16.7%, an 81% relative reduction) but did not reach statistical significance (P = .0986). The drug carries an embryo-fetal toxicity warning requiring effective contraception, and common side effects included headache, joint pain, and respiratory infection.

5.) BLA submitted to FDA for Viaskin peanut patch in children aged 4 to 7 years with peanut allergy

DBV Technologies submitted a Biologics License Application, with a request for priority review, for the Viaskin peanut patch, an epicutaneous immunotherapy for children ages 4 to 7 with peanut allergy. The filing is supported by the phase 3 VITESSE trial of 654 children across 86 sites, in which 46.6% of treated children met 12-month responder criteria versus 14.8% on placebo, and about 83% of treated children raised their tolerance threshold compared with 48% of placebo recipients. Application-site reactions were the most common side effect; anaphylaxis occurred in 0.5% of treated children, and adherence was 96.2%.

Full trial results have not yet appeared in a peer-reviewed journal, and the responder endpoint reflects desensitization under controlled conditions rather than sustained unresponsiveness or free peanut consumption — fewer than half of treated children met that threshold. The therapy is intended to be used alongside continued allergen avoidance and epinephrine readiness, not as a replacement for either.