Q1 2026 recap: 6 FDA approvals pediatricians need to know

The first quarter of 2026 marked a period of profound advancement in pediatric health care, driven by a wave of FDA drug approvals that offer renewed hope for some of the most complex and rare pediatric conditions. From historic, first-of-their-kind systemic therapies for ultrarare disorders such as Menkes disease and Hunter syndrome to critical new interventions for achondroplasia and acquired hypothalamic obesity, these regulatory milestones represent monumental strides in pediatric disease management.

Below is a comprehensive recap of 6 major FDA approvals granted in Q1 2026, detailing their clinical backgrounds and indications, along with links to in-depth, related coverage from Contemporary Pediatrics.

Drug approval recap: Q1 2026

1. Tividenofusp alfa-eknm (Avlayah; Denali Therapeutics)¹

Date: March 25, 2026

Indication: To treat the neurological manifestations of Hunter syndrome in pediatric patients.

Background: Based on a dedicated pediatric phase 1/2 open-label trial involving 47 patients aged 3 months to 13 years, the once-weekly intravenous infusion significantly reduced cerebrospinal fluid heparan sulfate, yielding a 91% average decrease from baseline at 24 weeks. This marks the first targeted therapy specifically approved for the neurologic complications of this rare X-linked disorder, providing a critical new intervention that must be initiated before a child develops advanced neurologic impairment.

Related coverage: FDA approves Avlayah for Hunter syndrome

2. Setmelanotide (Imcivree; Rhythm Pharmaceuticals)²
Date: March 19, 2026
Indication: To treat acquired hypothalamic obesity in adults and pediatric patients aged 4 years and older.

Background: Based on the pivotal phase 3 TRANSCEND trial (NCT05774756) of 142 patients, including pediatric subjects, those treated with setmelanotide achieved a –18.4% placebo-adjusted reduction in body mass index at 52 weeks compared with a 2.6% increase in the placebo group. As the first and only approved therapy for this condition, it provides a weight management option for children whose obesity stems from hypothalamic injury.

Related coverage: FDA approves setmelanotide for acquired hypothalamic obesity in patients 4 years and older

3. Navepegritide (Yuviwel; Ascendis Pharma)³
Date: February 27, 2026
Indication: For increasing linear growth in children aged 2 years and older with achondroplasia with open epiphyses.

Background: Based on dedicated pediatric data from 3 randomized, placebo-controlled trials, including the pivotal ApproaCH trial (NCT05598320), the once-weekly administered prodrug demonstrated clinical benefit through an improvement in annualized growth velocity. As the first and only once-weekly treatment indicated for children 2 years and older with open epiphyses, navepegritide offers a new, practical administration option that provides continuous systemic exposure to counteract overactive FGFR3 signaling.

Related coverage: FDA issues historic approval for navepegritide in achondroplasia

4. Dupilumab (Dupixent; Sanofi, Regeneron)⁴
Date: February 24, 2026
Indication: For managing allergic fungal rhinosinusitis (AFRS) in patients as young as 6 years.

Background: Based on adult clinical data extrapolated to children using weight-adjusted dosing rather than a dedicated pediatric AFRS trial, a 52-week study demonstrated that the biologic significantly improved sinus opacification and substantially reduced the need for both systemic corticosteroids and sinus surgery. This marks the first approved therapeutic option for AFRS, providing a crucial intervention for children as young as 6 years who are at great risk for sinus bone erosion.

Related coverage: FDA approves dupilumab for allergic fungal rhinosinusitis

5. Copper histidinate (Zycubo; Sentynl Therapeutics, Inc)⁵
Date: January 12, 2026
Indication: To manage Menkes disease in children.

Background: Based on dedicated pediatric data from 2 open-label, single-arm clinical trials evaluating this subcutaneous formulation against an external control group, patients initiating treatment within 4 weeks of birth demonstrated a 78% reduction in the risk of death. This introduces the first approved treatment for this ultrarare degenerative disorder, offering a critical subcutaneous clinical option that bypasses defective intestinal copper absorption.

Related coverage: FDA approves Zycubo for treatment of Menkes disease in children

6. Icotrokinra (Icotyde; Johnson & Johnson)⁶
Date: March 18, 2026
Indication: For the treatment of moderate to severe plaque psoriasis in adults and pediatric patients 12 years and older weighing at least 40 kg.

Background: Based on the phase 3 ICONIC program, which included the dedicated ICONIC-LEAD trial (NCT06095115) of approximately 2500 patients 12 years and older, 65% of treated patients achieved an Investigator Global Assessment (IGA) score of 0 or 1 at 16 weeks, with the adolescent subgroup notably outperforming the overall population with an 86.4% IGA 0/1 response at week 24. This first-in-class, once-daily oral targeted peptide offers a systemic alternative to injectable biologics for patients 12 years and older.

Relevant coverage: FDA approves icotrokinra, first oral targeted peptide for plaque psoriasis

References

1. FDA approves drug to treat neurologic manifestations of Hunter syndrome. News release. FDA. March 25, 2026. Accessed March 31, 2026. https://www.fda.gov/news-events/press-announcements/fda-approves-drug-treat-neurologic-manifestations-hunter-syndrome?utm_medium=email&utm_source=govdelivery
2. Rhythm Pharmaceuticals announces FDA approval of Imcivree (setmelanotide) for patients with acquired hypothalamic obesity. News release. Rhythm Pharmaceuticals. March 19, 2026. Accessed March 31, 2026. https://ir.rhythmtx.com/news-releases/news-release-details/rhythm-pharmaceuticals-announces-fda-approval-imcivreer-1
3. FDA approves once-weekly Yuviwel (navepegritide) for children with achondroplasia aged 2 years and older. Ascendis Pharma. February 27, 2026. Accessed March 31, 2026. https://investors.ascendispharma.com/news-releases/news-release-details/fda-approves-once-weekly-yuviwelr-navepegritide-children
4. FDA approves drug for adult and pediatric patients aged 6 and older with allergic fungal rhinosinusitis. FDA. February 25, 2026. Accessed March 31, 2026. https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-drug-adult-and-pediatric-patients-aged-6-and-older-allergic-fungal-rhinosinusitis
5. FDA approves first treatment for children with Menkes disease. News release. FDA. January 12, 2026. Accessed March 31, 2026. https://www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-children-menkes-disease
6. FDA approves Icotyde (icotrokinra), the first and only targeted oral peptide IL-23 receptor antagonist, for the treatment of moderate-to-severe plaque psoriasis. News release. Johnson & Johnson. March 18, 2026. Accessed March 31, 2026. https://www.jnj.com/media-center/press-releases