5 Notable FDA Approvals From March Highlight Advancements Across Therapeutic Areas

March was a busy month for the FDA, with several drug approvals across diverse therapeutic areas, including Crohn disease, uncomplicated urinary tract infections (uUTIs), and muscle-invasive bladder cancer (MIBC).

Here is a closer look at 5 notable FDA approvals from March, highlighting advancements across these and other disease states.

From Crohn disease to uncomplicated urinary tract infections and beyond, the FDA issued several high-impact drug approvals last month. | Image Credit: Tada Images - stock.adobe.com

1. FDA Approves Denosumab Biosimilars Stoboclo and Osenvelt

On March 4, the FDA approved Stoboclo and Osenvelt (both denosumab-bmwo; Celltrion), biosimilars to Prolia and Xgeva (denosumab), respectively, for the treatment of osteoporosis and various forms of bone loss.¹ This comes about a year after Wyost and Jubbonti (denosumab-bddz; Sandoz) became the first FDA-approved biosimilars to reference denosumab.²

The approval was supported by data from a phase 3 trial (NCT04757376) on postmenopausal women with osteoporosis.¹ It evaluated the efficacy, pharmacodynamics (PD), pharmacokinetics (PK), safety, and immunogenicity of denosumab-bmwo vs the reference drug.

The trial met its primary end point based on PD equivalence. It also demonstrated comparable efficacy, PK, and safety outcomes between groups through week 78, including in patients who transitioned from the reference drug to denosumab-bmwo.

Stoboclo, a receptor activator of the NF-κb ligand inhibitor referencing Prolia, is approved as a 60-mg/mL injection for postmenopausal women with osteoporosis at high risk for fracture. It is also indicated to increase bone mass in men receiving androgen deprivation therapy for nonmetastatic prostate cancer who are at high risk for fracture.

Osenvelt, a receptor activator of NF-κb ligand inhibitor referencing Xgeva, is approved as a 120-mg/1.7-L for the prevention of skeletal-related events in patients with multiple myeloma and in those with metastases from solid tumors. It is also indicated for skeletally mature adolescents with giant cell tumors of bone that are unresectable or where surgical resection is likely to result in severe morbidity.

Learn about additional indications here.

2. FDA Approves Guselkumab for Adult Patients With Crohn Disease

The FDA approved guselkumab (Tremfya; Johnson & Johnson) for adult patients with moderate to severe active Crohn disease on March 21, making it the first and only interleukin-23 inhibitor to offer subcutaneous and intravenous induction options.³

This approval was based on the phase 3 GRAVITI study (NCT05197049) that evaluated the efficacy and safety of guselkumab in adult patients through an analysis of guselkumab subcutaneous induction and maintenance therapy vs placebo. The study demonstrated clinical remission and endoscopic response with guselkumab at 1 year.

For subcutaneous induction, guselkumab is given as 400 mg at weeks 0, 4, and 8, delivered as 2 consecutive 200-mg injections from a single induction pack; it also comes in a 200-mg prefilled syringe. Similarly, 200 mg is infused at the same intervals for intravenous induction. Meanwhile, maintenance options include a 100-mg subcutaneous injection starting at week 16 and then every 8 weeks after, or a 200-mg injection starting at week 12 and then every 4 weeks after.

“With the approval of Tremfya, it is now possible to achieve meaningful improvements in clinical and endoscopic outcomes with the flexibility of self-administration from the start,” Chris Gasink, MD, vice president of medical affairs at Johnson & Johnson Innovative Medicine, said in a press release.⁴

3. FDA Approves Oral Iptacopan for C3 Glomerulopathy

The FDA approved oral iptacopan (Fabhalta; Novartis) on March 21 to reduce proteinuria in adults with C3 glomerulopathy (C3G), an ultrarare, progressive kidney disease.⁵ This makes it the first and only FDA-approved treatment for this disease.

The approval was based on data from the phase 3 APPEAR-C3G study (NCT04817618), which assessed the safety and efficacy of twice-daily oral iptacopan among 74 adult patients with C3G. It involved a 6-month randomized, double-blinded treatment period with patients taking iptacopan (n = 38) or placebo (n = 36), followed by a 6-month open-label period with all patients receiving iptacopan.

The study met its primary end point, showing a statistically significant reduction in proteinuria from baseline to 6 months with iptacopan vs placebo. Consequently, patients in the iptacopan cohort showed a 35.1% reduction in 24-hour urine protein-creatinine ratio (UPCR) vs the placebo cohort (1-sided P = .0014; 95% CI, 13.8-51.1).

Additionally, there was sustained improvement with iptacopan, as measured by a composite renal end point of at least a 50% reduction in UPCR and a maximum 15% reduction in estimated glomerular filtration rate (eGFR) at 12 months. Also, compared with patients’ historical decline, the researchers observed an improvement in eGFR trajectory with iptacopan.

“This approval of [iptacopan] is historic for the entire C3G community as now, for the first time, we have a therapy that is believed to treat the underlying cause of the disease, providing the potential for a new standard of care for patients,” Carla Nester, MD, MSA, FASN, APPEAR-C3G study coinvestigator, said in a press release.⁶

4. FDA Approves First New Class of Oral Antibiotics for Uncomplicated UTIs in Nearly 30 Years

On March 25, the FDA approved gepotidacin (Blujepa; GSK) to treat uUTIs in female patients aged 12 or older, introducing the first new class of oral antibiotics for this condition in nearly 30 years.⁷

The approval was supported by positive results from the phase 3 EAGLE-2 (NCT04020341) and EAGLE-3 (NCT04187144) trials, which compared the efficacy and safety of oral gepotidacin with that of the antibiotic nitrofurantoin in eligible adolescent and adult female patients.

In EAGLE-2, therapeutic success was observed in 50.6% (n = 162) of patients treated with gepotidacin and 47.0% (n = 135) of those treated with nitrofurantoin (adjusted difference, 4.3%; 95% CI, –3.6% to 12.1%). Similarly, in EAGLE-3, therapeutic success rates were 58.5% (n = 162) for patients treated with gepotidacin and 43.6% (n = 115) for those treated with nitrofurantoin (adjusted difference, 14.6%; 95% CI, 6.4%-22.8%).

The researchers determined that gepotidacin was noninferior to nitrofurantoin in both studies, but it demonstrated superiority in EAGLE-3.

"We are proud to have developed Blujepa, the first in a new class of oral antibiotics for uUTIs in nearly 3 decades, and to bring another option to patients given recurrent infections and rising rates of resistance to existing treatments," Tony Wood, chief scientific officer of GSK, said in a press release.⁸

5. FDA Approves Durvalumab Combo for Muscle-Invasive Bladder Cancer

Neoadjuvant durvalumab (Imfinzi; AstraZeneca) with gemcitabine and cisplatin plus adjuvant durvalumab received FDA approval on March 31 as perioperative immunotherapy against MIBC.⁹ This marks the first and only approved perioperative immunotherapy for this disease.

The approval was based on data from an interim analysis of NIAGARA (NCT03732677), an ongoing phase 3 trial with a primary completion date of June 30. The study includes 2 arms: an experimental and a control arm. The experimental arm receives durvalumab plus chemotherapy (cisplatin and gemcitabine), while the control arm receives the chemotherapy combination alone.

An interim analysis of data from the ongoing trial demonstrates overall positive outcomes when comparing the 2 arms. For example, patients receiving the investigational treatment experienced a 32% reduction in the risk of disease progression, recurrence, not undergoing surgery, or death (HR, 0.68; 95% CI, 0.56-0.82; P < .001). Additionally, the 2-year overall survival rate was 82.2% in the experimental group vs 75.2% in the control arm.

“This approval for the durvalumab-based perioperative regimen is a major breakthrough for people with MIBC, nearly half of whom see their cancer return despite chemotherapy and surgery with curative intent,” Matthew Galsky, MD, NIAGARA investigator, The Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, said in a press release.¹⁰

Honorable Mentions

The agency’s approvals in March extended beyond those highlighted above.

Additional FDA approvals from last month include:

1. FDA Approves Revakinagene Taroretcel for Macular Telangiectasia Type 2
2. FDA Approves Expanded Indication of Eculizumab for Pediatric Generalized Myasthenia Gravis
3. FDA Approves Vutrisiran to Mitigate Cardiomyopathy-Related Risks and Conditions
4. FDA Approves Cabozantinib for Advanced Pancreatic Neuroendocrine Tumors
5. FDA Approves Fitusiran for Reducing Bleeds in Patients With Hemophilia A/B

References

1. Santoro C. FDA approves denosumab biosimilars stoboclo and osenvelt. AJMC^®. March 4, 2025. Accessed April 17, 2025. https://www.ajmc.com/view/fda-approves-denosumab-biosimilars-stoboclo-and-osenvelt
2. Jeremias S. FDA approves first denosumab biosimilars. The Center for Biosimilars^®. March 5, 2024. Accessed April 17, 2025. https://www.centerforbiosimilars.com/view/fda-approves-first-denosumab-biosimilar
3. Santoro C. FDA approves guselkumab for adult patients with Crohn disease. AJMC. March 21, 2025. Accessed April 17, 2025. https://www.ajmc.com/view/fda-approves-guselkumab-for-adult-patients-with-crohn-disease
4. US FDA approves Tremfya (guselkumab), the first and only IL-23 inhibitor offering both subcutaneous and intravenous induction options, for adult patients with moderately to severely active Crohn’s disease. News release. Johnson & Johnson; March 20, 2025. Accessed March 21, 2025.  https://www.jnj.com/media-center/press-releases/u-s-fda-approves-tremfya-guselkumab-the-first-and-only-il-23-inhibitor-offering-both-subcutaneous-and-intravenous-induction-options-for-adult-patients-with-moderately-to-severely-active-crohns-disease
5. McNulty R. FDA approves oral Iptacopan for C3 glomerulopathy. AJMC. March 21, 2025. Accessed April 17, 2025. https://www.ajmc.com/view/fda-approves-oral-iptacopan-for-c3-glomerulopathy
6. Novartis receives third FDA approval for oral Fabhalta (iptacopan) – the first and only treatment approved in C3 glomerulopathy (C3G). News release. Novartis. March 20, 2025. Accessed March 21, 2025. https://www.novartis.com/news/media-releases/novartis-receives-third-fda-approval-oral-fabhalta-iptacopan-first-and-only-treatment-approved-c3-glomerulopathy-c3g
7. McCormick B. FDA approves first new class of oral antibiotics for uncomplicated UTIs in nearly 30 years. AJMC. March 25, 2025. https://www.ajmc.com/view/fda-approves-first-new-class-of-oral-antibiotics-for-uncomplicated-utis-in-nearly-30-years
8. Blujepa (gepotidacin) approved by US FDA for treatment of uncomplicated urinary tract infections (uUTIs) in female adults and paediatric patients 12 years of age and older. News release. GSK; March 25, 2025. Accessed March 25, 2025. https://www.gsk.com/en-gb/media/press-releases/blujepa-gepotidacin-approved-by-us-fda-for-treatment-of-uncomplicated-urinary-tract-infections/
9. Shaw ML. FDA approves durvalumab combo for muscle-invasive bladder cancer. AJMC. March 31, 2025. Accessed April 17, 2025. https://www.ajmc.com/view/fda-approves-durvalumab-combo-for-mibc
10. Imfinzi approved in the US as the first and only perioperative immunotherapy for patients with muscle-invasive bladder cancer. News release. AstraZeneca; March 31, 2025. Accessed March 31, 2025.  https://www.astrazeneca.com/media-centre/press-releases/2025/imfinzi-approved-in-the-us-for-bladder-cancer.html