The second quarter of 2025 delivered a wave of impactful FDA approvals across a spectrum of rare and chronic diseases, expanding treatment options and advancing precision medicine. Highlights included the first-ever therapies approved for immunoglobulin G4-related disease with inebilizumab and alkaptonuria with nitisinone, marking long-awaited milestones for underserved patient communities. New approvals for atrasentan in IgA nephropathy and beremagene geperpavec gene therapy for recessive dystrophic epidermolysis bullosa also broke new ground in nephrology and dermatology, respectively, providing innovative options where few existed before.
Meanwhile, regulatory progress in respiratory and infectious diseases brought approvals like once-daily roflumilast foam for scalp and body psoriasis, mepolizumab for eosinophilic COPD, and clesrovimab for RSV prevention in infants, reflecting a trend toward targeted therapies that simplify treatment and improve adherence. Additionally, the approval of twice-yearly lenacapavir for HIV prevention offered a transformative new approach to PrEP, while new treatments for giant cell arteritis and generalized myasthenia gravis underscored the quarter’s broad reach in expanding care.
Check out this Q2 2025 FDA news month in review for a recap of HCPLive’s coverage of the top FDA approvals and research from the past few months:
Top FDA Approvals in Q2 2025:
1. Atrasentan (Vanrafia)
Date: April 2, 2025
Indication: Reducing proteinuria in patients with IgA nephropathy (IgAN)
Background: Approval based on interim results from the phase 3 ALIGN trial, where atrasentan demonstrated significant reductions in proteinuria. These findings support its use as a disease-modifying therapy in IgAN, addressing a critical unmet need in this patient population.
Related Content: Understanding Atrasentan (Vanrafia) for IgA Nephropathy, with Richard Lafayette, MD
2. Inebilizumab (Uplizna)
Date: April 3, 2025
Indication: Treatment of adults with immunoglobulin G4-related disease (IgG4-RD)
Background: Approval supported by data from the phase 3 NATRON trial, in which inebilizumab significantly reduced the risk of disease flare compared to placebo. This marks the first FDA-approved therapy specifically for IgG4-RD, a chronic fibroinflammatory condition previously managed with less targeted treatments.
3. Beremagene Geperpavec (Vyjuvek)
Date: April 29, 2025
Indication: Treatment of recessive dystrophic epidermolysis bullosa (RDEB)
Background: Approval based on data from the GEM-3 trial, showing Vyjuvek, a topical gene therapy, significantly improved wound healing and skin integrity in patients with RDEB. Vyjuvek is the first gene therapy approved for this devastating genetic skin disorder.
4. Upadacitinib (Rinvoq)
Date: April 29, 2025
Indication: Treatment of giant cell arteritis (GCA) in adults
Background: Approval based on results from the SELECT-GCA trial, where upadacitinib achieved sustained remission and reduced glucocorticoid use. This provides a novel oral treatment option for patients with GCA, expanding therapeutic choices for this inflammatory vascular disease.
5. Nipocalimab (Zilbrysq)
Date: April 30, 2025
Indication: Treatment of generalized myasthenia gravis (gMG) in adults and children aged ≥12 years
Background: Approval based on data from the Vivacity-MG study, which demonstrated that nipocalimab significantly improved muscle strength and reduced disease severity in patients with gMG, offering a new targeted therapy for this neuromuscular disorder.
6. Mepolizumab (Nucala)
Date: May 22, 2025
Indication: Treatment of eosinophilic chronic obstructive pulmonary disease (COPD)
Background: Approval based on phase 3 trials demonstrating mepolizumab significantly reduced exacerbation rates in patients with elevated eosinophil counts. Mepolizumab becomes the first biologic approved for this COPD phenotype, marking an advance in personalized respiratory care.
7. Roflumilast Foam 0.3% (Zoryve)
Date: May 22, 2025
Indication: Treatment of plaque psoriasis involving the scalp and body in patients aged ≥12 years
Background: Approval supported by positive results from the phase 3 ARRECTOR trial, where once-daily roflumilast foam provided significant improvements in psoriasis affecting hard-to-treat areas like the scalp. This non-steroidal topical option expands treatment choices for patients.
Related content: Understanding Roflumilast Foam’s FDA Approval for Scalp and Body Psoriasis, with Jennifer Soung, MD
8. Clesrovimab (Enflonsia)
Date: June 9, 2025
Indication: Prevention of RSV lower respiratory tract disease in neonates and infants entering their first RSV season
Background: Approval based on pivotal phase 2b/3 CLEVER trial data, which showed clesrovimab reduced RSV-associated medically attended lower respiratory infections by 60.5% and hospitalizations by 84.3% through 5 months. Enflonsia is the first RSV preventive given at a fixed 105 mg dose regardless of weight, offering durable protection for infants.
Related Content: RX Review: RSV Prevention Strategies and the Changing Epidemiologic Landscape
9. Garadacimab-gxii (ANDEMBRY)
Date: June 16, 2025
Indication: Prophylactic prevention of hereditary angioedema (HAE) attacks in patients aged ≥12 years
Background: Approval based on the phase 3 VANGUARD trial, where garadacimab-gxii achieved ≥99% median reduction in HAE attacks compared to placebo, with 62% of treated patients remaining attack-free. ANDEMBRY is the first HAE therapy targeting factor XIIa, offering once-monthly subcutaneous administration.
https://www.hcplive.com/view/fda-approves-first-twice-yearly-hiv-prevention-option
FDA Approves Lenacapavir, First Twice-Yearly HIV Prevention Option
On June 18, 2025, Gilead announced FDA approval of lenacapavir (Yeztugo) as a twice-yearly injectable pre-exposure prophylaxis (PrEP) for preventing sexually acquired HIV in adults and adolescents weighing at least 35 kg. Based on phase 3 PURPOSE 1 and PURPOSE 2 trials, lenacapavir demonstrated superior efficacy with zero HIV infections in cisgender women during the study and significantly fewer infections in men and gender-diverse individuals compared to daily oral PrEP. This novel dosing regimen offers a transformative option to improve adherence and reduce stigma associated with more frequent PrEP dosing.
FDA Approves Nitisinone (HARLIKU) for Treatment of Alkaptonuria
On June 19, 2025, Cycle Pharmaceuticals announced FDA approval of nitisinone (HARLIKU) for reducing urine homogentisic acid in adults with alkaptonuria (AKU), making it the first and only treatment approved for this rare genetic disorder. Based on a post-hoc analysis of a 2009 trial, nitisinone demonstrated improvements in patient-reported outcomes and physical function over three years, supporting its efficacy despite the original trial missing its primary endpoint. HARLIKU’s approval offers a long-awaited option to address the significant disease burden, including joint damage and decreased mobility, in AKU patients.