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Monthly News Roundup - April 2025

Medically reviewed by Judith Stewart, BPharm. Last updated on May 5, 2025.

FDA Approves FcRn Blocker Imaavy for the Treatment of Generalized Myasthenia Gravis

The United States Food and Drug Administration (FDA) has approved Johnson & Johnson’s Imaavy (nipocalimab-aahu) as the first and only neonatal Fc receptor (FcRn) blocker for use in the treatment of generalized myasthenia gravis (gMG).

• Generalized myasthenia gravis is an autoantibody disease characterized by fluctuating weakness of the skeletal muscles. It is caused by autoantibodies targeting proteins at the neuromuscular junction. Imaavy is approved in anti-AChR and anti-MuSK antibody positive adults and pediatric gMG patients aged 12 years of age and older.
• Imaavy works by blocking FcRn to reduce levels of circulating immunoglobulin G (IgG) antibodies, which have an important role in the pathogenesis of many autoimmune diseases including myasthenia gravis.
• FDA approval of Imaavy was supported by data from the pivotal, ongoing Vivacity-MG3 study which demonstrated 20 months of lasting disease control and symptom relief.
• Imaavy is administered by intravenous infusion over at least 30 minutes for the initial dose, then every two weeks over at least 15 minutes for the maintenance doses.
• Warnings and precautions associated with Imaavy include infections, hypersensitivity reactions, and infusion-related reactions. Common adverse reactions (≥10%) include respiratory tract infections, peripheral edema, and muscle spasms.
• Nipocalimab is also being studied in multiple other conditions including Sjogren's Disease, Hemolytic Disease of the Fetus and Newborn, Fetal and Neonatal Alloimmune Thrombocytopenia, Systemic Lupus Erythematosus, Idiopathic Inflammatory Myopathy, and Chronic Inflammatory Demyelinating Polyneuropathy.

FDA Grants Accelerated Approval for Vanrafia for Proteinuria Reduction in Primary IgA Nephropathy

The FDA has granted accelerated approval for Novartis’ Vanrafia as the first and only selective endothelin A receptor antagonist for proteinuria reduction in primary IgA nephropathy (IgAN).

• IgAN is a rare kidney disease caused by an autoimmune reaction to an abnormal form of immunoglobulin A, resulting in the formation of immune complexes that deposit in the kidneys. Vanrafia works by blocking the endothelin A (ETA) receptor to reduce glomerular inflammation and proteinuria that can lead to progressive kidney damage and loss of kidney function in IgAN.
• Vanrafia was granted accelerated approval based on a pre-specified interim analysis of the Phase III ALIGN study measuring the reduction of proteinuria at 36 weeks compared to placebo. Continued approval of Vanrafia may be contingent on the results of ongoing Phase III ALIGN study evaluating whether Vanrafia slows disease progression as measured by estimated glomerular filtration rate (eGFR) decline at week 136, expected in 2026.
• Vanrafia tablets are administered orally once daily with or without food.
• Vanrafia comes with a Boxed Warning for major birth defects if used during pregnancy. Warnings and precautions include hepatotoxicity, fluid retention, and decreased sperm counts. Common adverse reactions (incidence ≥ 5%) include peripheral edema and anemia.

FDA Approves New Nasal Powder Formulation of Dihydroergotamine for Migraine

The FDA has approved Atzumi, Satsuma Pharmaceuticals’ nasal powder formulation of dihydroergotamine mesylate for the acute treatment of migraine, with or without aura in adults.

• Dihydroergotamine (DHE) is a well established migraine treatment with more than 70 years of therapeutic use. It has previously been FDA approved in injectable (D.H.E. 45) and liquid nasal spray (Migranal, Trudhesa) dosage forms. Atzumi utilizes the SMART (Simple MucoAdhesive Release Technology) platform which combines a proprietary advanced powder and device technology to provide rapid acting relief in a formulation that is easy-to-carry, and quick and easy to self-administer.
• FDA approval of Atzumi was based on two clinical studies (Phase 1 PK trial and ASCEND Phase 3 open-label, long-term safety trial), which demonstrated fast absorption, rapid achievement of high DHE plasma concentrations, and sustained DHE plasma levels over time as well as safety and tolerability in subjects with migraine.
• Atzumi is administered into one nostril by squeezing the device three separate times. The dose may be repeated, if needed, a minimum of 1 hour after the first dose. The maximum dose in a 24-hour period is 10.4 mg (two doses of Atzumi 5.2 mg). The safety of taking more than 4 doses within a 7-day period or 12 doses within a 30-day period has not been established.
• Common adverse reactions (incidence > 1%) include rhinitis, nausea, altered sense of taste, application site reactions, dizziness, vomiting, somnolence, pharyngitis, and diarrhea.

Zevaskyn Approved as the First Cell-Based Gene Therapy for Recessive Dystrophic Epidermolysis Bullosa

The FDA has approved Zevaskyn (prademagene zamikeracel) autologous cell-based gene therapy for the treatment of wounds in adult and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB).

• RDEB is a rare connective tissue disorder caused by a defect in the COL7A1 gene, which results in the inability to produce the functioning type VII collagen that helps anchor the dermal and epidermal layers of the skin. It can cause severe skin wounds that are painful and hard to heal.
• Zevaskyn is supplied as gene-modified cellular sheets that have the functional type VII collagen-producing COL7A1 gene incorporated into the patient’s own skin cells. The sheets are administered surgically by topical application onto wounds enabling normal type VII collagen expression, which facilitates wound healing.
• FDA approval of Zevaskyn was based on the pivotal Phase 3 VIITAL study (NCT04227106) that demonstrated statistically significant healing of large chronic RDEB wounds, and pain reduction. Across 43 wounds treated with a single application of Zevaskyn, 81 percent of wounds showed 50 percent or more healing (P<0.0001) as evaluated at six months, compared to 16 percent in 43 matched control wounds treated with standard of care. The Phase 1/2a study (NCT01263379) of 38 wounds across 7 patients showed that a single surgical application of Zevaskyn was associated with long-term improvement at treated sites over a median follow-up of 6.9 years; range 4 to 8 years.
• Common adverse reactions (incidence ≥5%) include procedural pain and pruritus.
• Zevaskyn is manufactured and distributed by Abeona Therapeutics Inc.

FDA Approves Penpulimab-kcqx for Advanced Nasopharyngeal Carcinoma

The FDA approved Akeso’s programmed death receptor-1 (PD-1)-blocking antibody penpulimab-kcqx for the treatment of adults with metastatic non-keratinizing nasopharyngeal carcinoma (NPC). It is indicated in combination with chemotherapy for first-line treatment, and as a single agent when the disease has progressed on or after platinum-based chemotherapy and at least one other prior line of therapy.

• Nasopharyngeal carcinoma is an aggressive type of squamous cell carcinoma of the epithelial tissue of the nasopharynx. Penpulimab-kcqx is a PD-1 blocking antibody that works to decrease NPC tumor growth by binding to PD-1 and blocking its interaction with PD-L1 and PD-L2.
• FDA approval of penpulimab-kcqx was based on the results of the international Phase III clinical trial AK105-304 and the pivotal AK105-202 study, both of which demonstrated the drug’s clinical benefits and favorable safety profile across two stages of treatment for metastatic NPC.
  - AK105-304 (NCT04974398) was a randomized, double-blind, multi-center trial in 291 patients with recurrent or metastatic NPC who had not received previous systemic chemotherapy. Median progression-free survival was 9.6 months (95% CI: 7.1, 12.5) in the penpulimab-kcqx arm and 7.0 months (95% CI: 6.9, 7.3) in the placebo arm, with 31% and 11% of patients alive and progression-free after 12 months of follow-up in the penpulimab-kcqx and placebo arms, respectively.
  - AK105-202 (NCT03866967) was an open-label, multicenter, single-arm trial of 125 patients with unresectable or metastatic non-keratinizing NPC who had disease progression after platinum-based chemotherapy and at least one other line of therapy. The objective response rate was 28% (95% CI: 20, 37) and median duration of response was not reached (95% CI: 9.2, not estimable).
• Penpulimab-kcqx is the second FDA approved treatment for nasopharyngeal carcinoma after the approval of Loqtorzi (toripalimab-tpzi) in October 2023.

Posted April 2025

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