FDA New Drug Approvals Overview - April 2025

This article provides a brief overview of the FDA's new drug approvals in April 2025, highlighting new drug stars in the pharmaceutical world that bring new hope for life and health:

VANRAFIA

Developing Company: NOVARTIS

Approval Type: New Drug Application (NDA)

Approval Date: April 2, 2025

Indication: Reducing proteinuria levels in adults with primary Immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression.

On April 2, 2024, Novartis announced that the FDA had granted accelerated approval for its selective endothelin A receptor antagonist, Vanrafia (atrasentan). This is the first and only selective endothelin A receptor antagonist approved for reducing proteinuria in primary IgAN. Vanrafia works by inhibiting the activity of the endothelin A receptor, reducing proteinuria and helping to manage the disease. It is a once-daily, non-steroidal oral treatment that can be seamlessly added to supportive therapies for IgAN, including renin-angiotensin system (RAS) inhibitors, and can be used with or without sodium-glucose cotransporter-2 (SGLT2) inhibitors. It is a foundational therapy and does not require a Risk Evaluation and Mitigation Strategy (REMS).

PENPULIMAB-KCQX

Developing Company: Akeso Biopharma

Approval Type: New Drug Application (NDA)

Approval Date: April 23, 2025

Indication:

• First-line treatment: In combination with cisplatin or carboplatin and gemcitabine, for the treatment of adult patients with recurrent or metastatic non-keratinizing nasopharyngeal carcinoma (NPC).
• Post-line treatment: Monotherapy for adult patients with NPC who have progressed after at least one line of platinum-based chemotherapy.

On April 24, 2025, the FDA approved Penpulimab-KCQX (brand name: Paenpulimab), a differentiated anti-PD-1 monoclonal antibody engineered with an IgG1 subtype and Fc modifications to enhance therapeutic efficacy while reducing adverse effects. Developed by Akeso Biopharma, this therapy is further developed and commercialized through a joint venture with Cstone Pharmaceuticals. Previously, Penpulimab-KCQX had been approved for four indications in China, including the combination therapy for advanced or metastatic squamous non-small cell lung cancer and the treatment of recurrent or metastatic nasopharyngeal carcinoma (NPC) after chemotherapy.

IMAAVY

Developing Company: JANSSEN BIOTECH

Approval Type: New Drug Application (NDA)

Approval Date: April 29, 2025

Indication: Treatment of adult and pediatric patients aged 12 years and older with acetylcholine receptor (AChR) or muscle-specific kinase (MuSK) antibody-positive generalized myasthenia gravis (gMG).

On April 30, 2025, Johnson & Johnson announced that the FDA had approved its innovative antibody therapy, Imaavy (nipocalimab-aahu). Imaavy is the first Fc receptor neonatal (FcRn) blocker approved for treating antibody-positive generalized myasthenia gravis (gMG). Its mechanism, which significantly reduces IgG levels and provides clinical functional improvement, makes it an important treatment option for this rare disease.

OPDIVO (nivolumab) + YERVOY (ipilimumab)

Developing Company: Bristol Myers Squibb

Approval Type: Supplemental Biologics License Application (sBLA)

Approval Date: April 8, 2025

Indication:

• Opdivo + Yervoy combination therapy: For the treatment of patients aged 12 and older with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) colorectal cancer (CRC).
• Opdivo monotherapy: For patients aged 12 and older with MSI-H/dMMR metastatic colorectal cancer (CRC) who have progressed after fluorouracil, oxaliplatin, or irinotecan-based chemotherapy.

Opdivo + Yervoy is the first "dual immune" therapy combination approved for use in colorectal cancer.

VITRAKVI (larotrectinib)

Product Category: NTRK Inhibitor; Broad-spectrum Anticancer Drug

Developing Company: Bayer HealthCare Pharmaceuticals

Approval Type: Supplemental New Drug Application (sNDA)

Approval Date: April 9, 2025

Indication:

• For adult and pediatric patients with solid tumors who meet the following criteria:
• Diagnosed with NTRK gene fusion based on validated testing methods, excluding known acquired resistance mutations
• Patients with metastatic disease or those for whom surgical resection could lead to severe complications
• Patients with no satisfactory alternative treatment or who have failed previous treatments

VYVGART HYTRULO

Product Category: FcRn Inhibitor and Hyaluronidase Combination Formulation

Developing Company: Argenx

Approval Type: Supplemental Biologics License Application (sBLA)

Approval Date: April 10, 2025

Indication:

• AChR+ Generalized Myasthenia Gravis (gMG)
• Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)

VALTOCO (diazepam nasal spray)

Product Category: Benzodiazepine Nasal Spray

Developing Company: Neurelis

Approval Type: Supplemental New Drug Application (sNDA)

Approval Date: April 15, 2025

Indication: For patients aged 2 years and older with cluster seizures or acute repetitive seizures

DUPIXENT (dupilumab)

Product Category: Fully Human IL-4Rα Monoclonal Antibody/Immunomodulator

Developing Companies: Regeneron & Sanofi

Approval Type: Supplemental Biologics License Application (sBLA)

Approval Date: April 17, 2025

Indication: For chronic spontaneous urticaria (CSU) in patients aged 12 years and older who have inadequate control with H1 antihistamines. The first new targeted therapy for CSU in years.