Another month has come and gone, and with it, several FDA approvals and regulatory updates related to the ever-evolving landscape of pediatric health care.

In our May 2026 recap, we list our top FDA-related news items in a quick, easy-to-read format so you can stay informed.

Take a look at our detailed coverage of FDA-related news from May and easily stay in touch with our digital newsletters that bring you practical information for today's pediatrician.

Click the title of each story below for our full coverage of that regulatory update.

FDA updates in pediatric care: May 2026

1. FDA grants rare pediatric disease designation to (Z)-endoxifen for McCune-Albright syndrome

On May 4, 2026, the FDA granted rare pediatric disease designation to investigational oral (Z)-endoxifen for the treatment of females with McCune-Albright syndrome, a rare pediatric endocrine disorder characterized by features including gonadotropin-independent precocious puberty, fibrous dysplasia, and café-au-lait macules. The designation does not indicate approval, safety, or efficacy but may make the sponsor, Atossa Therapeutics, eligible for a priority review voucher if a future marketing application is approved. Because estrogen excess is a key driver of precocious puberty in affected girls, the estrogen receptor–targeting mechanism of (Z)-endoxifen provides a biologically plausible therapeutic approach; however, no phase 2 or phase 3 efficacy data in McCune-Albright syndrome have been reported.

2. FDA approves interchangeable insulin glargine-aldy for type 1 and type 2 diabetes

On May 4, 2026, the FDA approved insulin glargine-aldy (LANGLARA) as an interchangeable biosimilar to Lantus (insulin glargine) for adults and pediatric patients with type 1 diabetes and for adults with type 2 diabetes. The approval was supported by analytical, preclinical, and clinical data demonstrating comparable pharmacokinetics, pharmacodynamics, efficacy, safety, and immunogenicity to the reference product. As an interchangeable biosimilar, LANGLARA may be substituted for Lantus at the pharmacy level where permitted under state law, potentially expanding access to basal insulin therapy.

3. FDA grants Rare Pediatric Disease Designation to SB-101 for urea cycle disorders

On May 4, 2026, the FDA granted rare pediatric disease designation to investigational cell-based liver therapy SB-101 for the treatment of urea cycle disorders, a group of rare inherited metabolic conditions that can cause life-threatening hyperammonemia and severe neurologic injury in infants. Developed by Satellite Bio, SB-101 is designed as an off-the-shelf therapy intended to restore metabolic liver function through the delivery of healthy liver cells. The designation does not indicate approval or establish safety or efficacy but may qualify the company for a priority review voucher if the therapy is ultimately approved.

4. FDA expands ASCENIV label to include pediatric patients aged 2 years and older

On May 4, 2026, the FDA approved a label expansion for ASCENIV, extending its indication to include pediatric patients aged 2 years and older with primary humoral immunodeficiency. Previously approved for patients aged 12 years and older, ASCENIV is a plasma-derived intravenous immune globulin formulated with standard source plasma and respiratory syncytial virus–enriched plasma to provide broad passive immune protection. The expanded indication follows completion of a required pediatric postmarketing assessment and may allow earlier use of immune globulin therapy in children at risk for recurrent infections due to impaired antibody production. The safety profile remains consistent with other intravenous immune globulin products, including boxed warnings for thrombosis and renal dysfunction, and common adverse events such as headache, sinusitis, gastrointestinal symptoms, and upper respiratory tract infections.

5. FDA approves ocrelizumab for pediatric relapsing-remitting multiple sclerosis

On May 8, 2026, the FDA approved intravenous Ocrevus (ocrelizumab; Genentech) for the treatment of relapsing-remitting multiple sclerosis in pediatric patients aged 10 years and older weighing at least 25 kg. The approval was supported by results from the phase 3 OPERETTA II trial, which demonstrated noninferiority to fingolimod in reducing annualized relapse rate and superior reductions in MRI disease activity, including a 48% reduction in new or enlarging T2 lesions and an 87% reduction in gadolinium-enhancing T2 lesions. Safety findings in pediatric patients were consistent with the established adult safety profile, with infrequent serious adverse events and no treatment discontinuations related to ocrelizumab.

6. Levacetylleucine sNDA for ataxia-telangiectasia receives FDA Priority Review

On May 19, 2026, the FDA accepted a supplemental new drug application for AQNEURSA (levacetylleucine) for the treatment of ataxia-telangiectasia in adult and pediatric patients and granted the application Priority Review, with a target action date of September 19, 2026. The submission is supported by results from the phase 3 IB1001-303 trial, which reportedly met its primary and key secondary endpoints, demonstrating improvements in neurologic signs, symptoms, and function in patients with this rare inherited neurodegenerative disorder. Although detailed efficacy and safety data have not yet been released, the company reported that levacetylleucine was generally well tolerated and that no drug-related serious adverse events were observed. If approved, AQNEURSA would become the first FDA-approved therapy specifically indicated for ataxia-telangiectasia, a progressive childhood-onset condition currently managed with supportive multidisciplinary care.

7. FDA approves OTC Differin Epiduo Gel for acne in patients aged 12 years and older

On May 26, 2026, the FDA approved over-the-counter availability of Differin Epiduo Acne Gel (Galderma), a fixed-dose combination of adapalene 0.1% and benzoyl peroxide 2.5%, for patients aged 12 years and older. Previously available by prescription, the topical retinoid–benzoyl peroxide combination has demonstrated efficacy in reducing inflammatory and noninflammatory acne lesions and is supported by clinical guidelines as a core treatment option for mild to moderate acne. The prescription-to-OTC switch is intended to expand access to evidence-based acne therapy for adolescents and adults and may allow earlier treatment initiation before medical evaluation.

8. FDA approves LINZESS for functional constipation in children 2 to 5 years

On May 27, 2026, the FDA approved an expanded indication for LINZESS (linaclotide) to include children aged 2 to 5 years with functional constipation, making it the first and only FDA-approved prescription therapy for this condition in that age group. The approval was supported by a phase 3 randomized, placebo-controlled trial demonstrating improved spontaneous bowel movement frequency with linaclotide 72 mcg compared with placebo, with a safety profile generally consistent with that observed in adults and older pediatric patients. Functional constipation is a common chronic condition in preschool-aged children, and the expanded indication provides an evidence-based treatment option for patients who continue to experience symptoms despite dietary, behavioral, or over-the-counter interventions. The prescribing information continues to include a boxed warning against use in children younger than 2 years because of the risk of serious dehydration.

9. FDA extends adrabetadex NDA review for infantile-onset Niemann-Pick disease type C

On May 28, 2026, the FDA extended the review period for the new drug application for investigational intrathecal adrabetadex in infantile-onset Niemann-Pick disease type C, moving the Prescription Drug User Fee Act target action date to November 17, 2026. The 3-month extension followed the company’s submission of additional information and supporting documentation, which the FDA classified as a major amendment to the application. Adrabetadex has previously received Priority Review, orphan drug designation, and breakthrough therapy designation and is being evaluated for a rare, progressive pediatric neurodegenerative disorder characterized by impaired cholesterol trafficking and early neurologic decline. Although no new efficacy or safety data were reported, the therapy remains available through an expanded access program for eligible patients while regulatory review continues.

10. FDA approves inhaled insulin Afrezza for children and adolescents with diabetes

On May 29, 2026, the FDA approved Afrezza for children and adolescents aged 6 years and older with diabetes, making it the first rapid-acting inhaled insulin available for pediatric patients requiring mealtime insulin. The approval was supported by results from the phase 3 INHALE-1 trial, which demonstrated noninferiority to injectable rapid-acting insulin analogs for glycemic control over 26 weeks and showed no clinically meaningful differences in lung function or hypoglycemia rates between treatment groups. Afrezza provides an alternative to injections and insulin pumps by offering rapid insulin absorption that can be administered at the start of a meal, potentially improving flexibility for children with variable eating patterns and daily schedules. The product carries a boxed warning for acute bronchospasm and is contraindicated in patients with asthma or chronic obstructive pulmonary disease, with spirometry evaluation recommended before treatment initiation.