What's next in pediatrics — October 23, 2026

This edition covers five FDA regulatory decisions with assigned target dates (PDUFA) between November 22, 2026 and March 22, 2027 that bear directly on pediatrics: Duchenne muscular dystrophy, SCN2A/SCN8A epileptic encephalopathies, achondroplasia, infant atopic dermatitis and second-season RSV prophylaxis. All dates come from the sponsors' official announcements; the FDA may act early, extend the review or decline.

1. Deramiocel for Duchenne muscular dystrophy

[Pending regulatory decision] [USA] [Confirmed date: November 22, 2026]
What it is: an allogeneic cardiosphere-derived cell therapy (CAP-1002), 150 million cells IV every 3 months; the phase 3 HOPE-3 trial (NCT05126758, n = 106, ambulatory and non-ambulatory) compared it with placebo over 12 months, with upper limb function (PUL v2.0) as the primary endpoint. The FDA extended the date by three months, from August 22, after classifying the submission of 24-month open-label extension data and additional robustness analyses as a "major amendment"; the company is seeking an indication focused on upper limb function.
What problem it solves: progressive loss of upper limb function and cardiomyopathy, the leading cause of death in Duchenne; today we use corticosteroids, vamorolone, givinostat and exon-skipping drugs only for specific mutations, with no heart-specific therapy.
Critical take: a small, sponsor-funded trial, a prior complete response letter (July 2025) and efficacy figures reported by the company; availability outside the US is uncertain.
https://www.capricor.com/investors/sec-filings/all-sec-filings/content/0001104659-26-100071/capr-20260824xex99d1.htm
https://clinicaltrials.gov/study/NCT05126758

2. Relutrigine for SCN2A and SCN8A epileptic encephalopathies

[Pending regulatory decision] [USA] [Confirmed date: December 27, 2026]
What it is: a small-molecule sodium channel modulator for patients with SCN2A- or SCN8A-related developmental and epileptic encephalopathy, under priority review; the application rests on the phase 2 EMBOLD trial, stopped early for efficacy after an interim analysis on the recommendation of the data monitoring committee. The date was extended by three months (from September 27) because the FDA classified the additional sensitivity analyses submitted as a major amendment; no new clinical studies were requested.
What problem it solves: early-onset genetic epilepsies that are often drug-resistant; today several antiseizure drugs are combined (high-dose sodium channel blockers in gain-of-function forms) with inadequate control.
Critical take: phase 2 evidence, reported by the sponsor and stopped early for efficacy, so the magnitude and durability of the effect should be interpreted with caution; according to the company's 8-K, patients receiving relutrigine (n = 51) had a 53% placebo-adjusted reduction in seizures over 16 weeks (p < 0.0002), without stating how many were randomized; the expected benefit depends on variant type (gain or loss of function) and requires access to sequencing.
https://ir.praxismedicines.com/news-releases/news-release-details/praxis-precision-medicines-announces-extension-period
https://ir.praxismedicines.com/news-releases/news-release-details/praxis-precision-medicines-announces-fda-acceptance-and-priority
https://www.sec.gov/Archives/edgar/data/1689548/000168954825000114/prax-20251204.htm

3. Oral infigratinib for achondroplasia

[Pending regulatory decision] [USA] [Confirmed date: February 4, 2027]
What it is: an oral selective FGFR3 inhibitor under priority review; in the 2:1 randomized, double-blind, placebo-controlled phase 3 PROPEL 3 trial (NCT06164951), in children aged 3 to under 18 years with open growth plates, it increased annualized height velocity at 52 weeks (least-squares mean difference +1.74 cm/year; observed mean difference +2.10 cm/year; p < 0.0001) and improved height Z-score (+0.32 SD; p < 0.0001); mild, transient hyperphosphatemia in 3 participants (4%). An EMA submission is planned for Q4 2026.
What problem it solves: achondroplasia has no approved oral treatment; today the approved options are injectable CNP analogues: daily vosoritide and, in the US, once-weekly navepegritide, approved in February 2026 under accelerated approval from age 2.
Critical take: height velocity is a surrogate outcome; the effect on final height, foramen magnum stenosis and sleep apnea is still unknown, and the trial is sponsor-run.
https://www.globenewswire.com/news-release/2026/10/06/3375352/0/en/bridgebio-announces-fda-acceptance-and-priority-review-of-nda-for-oral-infigratinib-for-children-with-achondroplasia.html
https://clinicaltrials.gov/study/NCT06164951
https://s205.q4cdn.com/224494602/files/doc_news/BridgeBio-Reports-Positive-Phase-3-Topline-Results-for-Oral-Infigratinib-with-the-First-Statistically-Significant-Improvements-in-Bod-HSXHC.pdf
https://investors.ascendispharma.com/news-releases/news-release-details/fda-approves-once-weekly-yuviwelr-navepegritide-children

4. Roflumilast cream 0.05% for infants with atopic dermatitis

[Pending regulatory decision] [USA] [Confirmed date: February 23, 2027]
What it is: a supplemental application to extend once-daily roflumilast (PDE4 inhibitor) cream 0.05% to infants aged 3 to 24 months with mild to moderate atopic dermatitis; it relies on the open-label phase 2 INTEGUMENT-INFANT study (101 infants enrolled; at 4 weeks, among the 96 who completed treatment, vIGA-AD success 34.4% and EASI-75 58.3%) and a phase 1 pharmacokinetic study (n = 19). It is currently approved from age 2.
What problem it solves: infants with atopic dermatitis have few approved non-steroidal options; today we use emollients, low-potency topical steroids and crisaborole, and calcineurin inhibitors are used off-label.
Critical take: efficacy is exploratory, with no control group and only 4 weeks of exposure; there are no long-term data at this age.
https://www.globenewswire.com/news-release/2026/07/08/3324020/0/en/fda-accepts-supplemental-new-drug-application-for-arcutis-zoryve-roflumilast-cream-0-05-for-the-treatment-of-mild-to-moderate-atopic-dermatitis-in-infants-down-to-3-months.html
https://www.globenewswire.com/news-release/2026/03/28/3264182/0/en/Arcutis-Presents-New-Phase-2-Results-in-Infants-with-Atopic-Dermatitis-in-Late-Breaking-Session-Today-at-the-2026-American-Academy-of-Dermatology-Annual-Meeting.html
https://www.biospace.com/press-releases/fda-accepts-supplemental-new-drug-application-for-arcutis-zoryve-roflumilast-cream-0-05-for-the-treatment-of-mild-to-moderate-atopic-dermatitis-in-infants-down-to-3-months

5. Clesrovimab for a second RSV season in high-risk children

[Pending regulatory decision] [USA and European Union] [Confirmed date: March 22, 2027 (FDA); EMA under review since July 2026, no public date]
What it is: an expanded indication for the monoclonal antibody clesrovimab to children under 2 at increased risk of severe RSV (chronic lung disease, congenital heart disease, preterm birth with risk factors) through their second season; partially blinded phase 3 SMART trial (NCT04938830): 1:1 versus monthly palivizumab in season 1 (n = 1,003) and an open-label 210 mg dose in season 2 (n = 276).
What problem it solves: a second single-dose option to protect these children in their second season; today the antibody with that indication is nirsevimab.
Critical take: season 2 was open-label with no control group; it provides safety and exposure data, but these are not equivalent to comparative clinical efficacy and do not allow us to predict what the FDA will base its decision on; Merck-sponsored.
https://s2.q4cdn.com/584635680/files/doc_news/U-S--FDA-Accepts-sBLA-for-ENFLONSIA-clesrovimab-cfor-to-Update-its-Respiratory-Syncytial-Virus-RSV-Lower-Respiratory-Tract-Disease-In-S7UWZ.pdf
https://www.merck.com/news/u-s-fda-accepts-sbla-for-enflonsia-clesrovimab-cfor-to-update-its-respiratory-syncytial-virus-rsv-lower-respiratory-tract-disease-indication-to-include-children-under-two-years-at-increa/
https://clinicaltrials.gov/study/NCT04938830

*What's next in pediatrics — October 23, 2026*
Five FDA decisions with dates:
• Nov 22, 2026 — Deramiocel in Duchenne (upper limb function)
• Dec 27, 2026 — Relutrigine in SCN2A/SCN8A epileptic encephalopathies
• Feb 4, 2027 — Oral infigratinib in achondroplasia
• Feb 23, 2027 — Roflumilast cream 0.05% in infants 3–24 months with atopic dermatitis
• Mar 22, 2027 — Clesrovimab for a second RSV season in high-risk under-2s
With a critical take on each: surrogates, open-label trials and sponsorship.
Full text and audio: https://boletinesmedicos.com/en/pediatrics/2026-10-23-viene.html
MedBulletins · boletinesmedicos.com

What's next in pediatrics: five FDA regulatory decisions with assigned dates between November 2026 and March 2027.

• Deramiocel in Duchenne (November 22)
• Relutrigine in SCN2A/SCN8A encephalopathies (December 27)
• Oral infigratinib in achondroplasia (February 4)
• Roflumilast cream in infants with atopic dermatitis (February 23)
• Clesrovimab for a second RSV season (March 22)

For each: the clinical problem it addresses, what we do today and what limits the evidence (surrogate outcomes, early phases, trials without a control group).

Full text and audio: https://boletinesmedicos.com/en/pediatrics/2026-10-23-viene.html

#Pediatrics #ChildNeurology #RSV #RareDiseases #BoletinesMedicos

What's next in pediatrics: 5 FDA decisions Nov 2026–Mar 2027 (Duchenne, SCN2A/SCN8A, oral achondroplasia drug, roflumilast in infants, clesrovimab 2nd RSV season), with a critical take. https://boletinesmedicos.com/en/pediatrics/2026-10-23-viene.html