Q1 2026 Recap: Top FDA Approvals

The first quarter of 2026 brought a series of notable US Food and Drug Administration (FDA) approvals, highlighting continued momentum in innovation across diverse therapeutic areas, including rare diseases, endocrinology, dermatology, psychiatry, and hepatology. A recurring theme was the expansion of treatment options for underserved populations, particularly pediatric and rare disease communities, alongside a growing emphasis on more convenient, patient-centered care.

Several decisions marked important firsts, including the first approved therapies for conditions like pediatric acquired thrombotic thrombocytopenic purpura (aTTP), cerebral folate deficiency, and acquired hypothalamic obesity. The FDA also advanced novel therapeutic approaches, with approvals introducing new mechanisms and classes, as well as first-in-indication treatments poised to reshape care in areas like psoriasis and cholestatic pruritus in primary biliary cholangitis (PBC).

At the same time, the quarter reflected ongoing progress in optimizing existing therapies, with higher-dose options, new pediatric indications, and innovations in drug delivery aimed at improving adherence and quality of life.

Check out this Q1 2026 FDA news month in review for a recap of HCPLive’s coverage of the top FDA approvals and research from the past few months:

FDA Approves Caplacizumab-yhdp (Cablivi) as First Pediatric aTTP Therapy

Date: January 5, 2026

Indication: Pediatric patients ≥ 12 years of age with aTTP in combination with plasma exchange and immunosuppressive therapy

Background: Approval supported by efficacy data from a retrospective chart review study of 30 patients 2-18 years of age showing 80% of patients achieved clinical remission. Builds upon caplacizumab’s initial 2019 approval for adults with aTTP and makes caplacizumab the first treatment for pediatric aTTP.

FDA Approves ProlivRx, First At-Home Neuromodulation Device for MDD

Date: January 12, 2026

Indication: Adults with major depressive disorder (MDD) who did not respond to ≥ 1 antidepressant

Background: Supported by clinical evidence from the MOOD Study, a randomized, controlled, multicenter clinical trial, evaluating Proliv Rx in MDD with inadequate response to antidepressant medications.

FDA Approves First Dual-Agent Eye Drop for Near Vision Loss

Date: January 28, 2026

Indication: Presbyopia in adults

Background: Supported by results from a pair of pivotal phase 3 trials, BRIO I and BRIO II, conducted in adults with presbyopia. Represents the first regulatory authorization of a fixed-dose, dual-agent topical therapy for age-related near vision degradation.

FDA Approves ET-600 Desmopressin Acetate (Desmoda) for Central Diabetes Insipidus

Date: February 25, 2026

Indication: Central diabetes insipidus in pediatric patients

Background: Marks the first and only FDA-approved oral liquid formulation of desmopressin

FDA Approves 3 New Pediatric Indications for Once-Weekly Somapacitan-beco (Sogroya)

Date: February 27, 2026

Indication: Children ≥ 2.5 years of age with Idiopathic Short Stature (ISS), short stature born Small for Gestational Age (SGA) and with no catch-up growth by 2 years of age, or growth failure associated with Noonan Syndrome (NS)

Background: Supported by data from the REAL8 study as part of the ongoing REAL clinical trial program.

FDA Grants Accelerated Approval to Navepegritide for Achondroplasia in Children Aged 2 Years and Older

Date: February 27, 2026

Indication: Children ≥ 2 years of age with achondroplasia and open epiphyses

Background: Based on improvements in annualized growth velocity (AGV) from 3 phase 3 trials, with continued approval contingent on confirmatory evidence of clinical benefit

FDA Approves Leucovorin Calcium (Wellcovorin) Tablets as First Cerebral Folate Deficiency Treatment

Date: March 10, 2026

Indication: Adult and pediatric patients who have a confirmed variant in the folate receptor 1 gene (CFD-FOLR1)

Background: Based on a systematic review of the published literature on the topic, including published case reports with patient-level information, as well as mechanistic data

FDA Approves Secukinumab for Pediatric Patients 12 Years and Older with HS

Date: March 13, 2026

Indication: Moderate to severe hidradenitis suppurativa (HS) in pediatric patients ≥ 12 years of age

Background: Approval makes it the only IL-17A inhibitor for this population, adding a differentiated biologic option to an area where treatment choices for younger patients have historically been limited

Icotrokinra, an Oral Il-23 Inhibitor, Receives FDA Approval for Psoriasis

Date: March 18, 2026

Indication: Moderate to severe plaque psoriasis in adults and pediatric patients ≥ 12 years of age weighing ≥ 40 kg who are candidates for systemic therapy or phototherapy

Background: Marks the first for an oral targeted peptide selectively blocking the IL-23 receptor and introduces a new mechanistic class to the psoriasis treatment landscape backed by data from multiple trials in the phase 3 ICONIC program

FDA Approves Higher Dose Semaglutide (Wegovy HD) Injection 7.2 mg for Obesity

Date: March 19, 2026

Indication: To be used along with a reduced calorie diet and increased physical activity to help adults with obesity lose weight and keep it off, provided they have tolerated the 2.4 mg dosage for ≥ 4 weeks and additional weight reduction is clinically indicated

Background: Based on results from the STEP UP trial program, a phase 3b 72-week randomized, double-blinded, placebo-controlled and active-controlled superiority trial designed to evaluate the efficacy and safety of semaglutide injection 7.2 mg compared to semaglutide injection 2.4 mg and placebo as an adjunct to lifestyle intervention.

FDA Approves Linerixibat (Lynavoy) As First Treatment for Cholestatic Pruritus in PBC

Date: March 19, 2026

Indication: Cholestatic pruritus in adult patients with PBC

Background: Approval makes it the first medicine approved in the US for this indication, supported by data from the global phase 3 GLISTEN trial, which met both primary and key secondary endpoints, demonstrating significant, rapid and sustained improvements in cholestatic pruritus and itch-related sleep interference versus placebo

FDA Approves Setmelanotide (Imcivree) for Acquired Hypothalamic Obesity

Date: March 19, 2026

Indication: Acquired hypothalamic obesity in adults and pediatric patients ≥ 4 years of age

Background: Approval makes it the first and only FDA-approved therapy for this condition, based on data from the global, randomized, double-blind, placebo-controlled phase 3 TRANSCEND trial

FDA Approves Insulin Icodec (Awiqli) as First Once-Weekly Basal Insulin for Type 2 Diabetes

Date: March 26, 2026

Indication: Adjunct to diet and exercise to improve glycemic control in adults with type 2 diabetes

Background: Makes it the first and only once-weekly basal insulin available in the US, based on results from the ONWARDS type 2 diabetes phase 3a program, which comprised 5 randomized, active-controlled, treat-to-target clinical trials in approximately 4000 adults with type 2 diabetes.