FDA Decisions Loom for Amgen, Aldeyra, and Argenx in Coming Weeks

The pharmaceutical industry is poised for significant developments as the U.S. Food and Drug Administration (FDA) prepares to announce crucial decisions on three therapies in early April. These decisions could potentially expand treatment options for patients with dry eye disease, IgG4-related disease, and generalized myasthenia gravis.

Aldeyra's Reproxalap Seeks Approval for Dry Eye Disease

Aldeyra Therapeutics is anticipating an FDA decision by April 2 on its RASP modulator reproxalap for the treatment of dry eye disease. The Massachusetts-based biotech's application is supported by Phase III data from August 2024, which demonstrated that a 0.25% dose of reproxalap ophthalmic solution significantly reduced eye discomfort compared to a vehicle control.

Reproxalap, a small molecule designed as a topical eye therapy, targets and inhibits reactive aldehyde species, which are typically present in high concentrations during eye inflammation. This decision comes after a previous setback in October 2023, when the FDA cited a lack of efficacy evidence and requested additional studies.

Amgen Seeks Expansion for Uplizna in IgG4-Related Disease

By April 3, the FDA is expected to announce its verdict on Amgen's supplemental application for Uplizna (inebilizumab) in IgG4-related disease. Uplizna, an anti-CD19 antibody initially approved in 2020 for neuromyelitis optica spectrum disorder, works by depleting both mature and immature B cells.

Amgen's application is based on data from the Phase III MITIGATE trial, which showed that Uplizna reduced the risk of disease-related flares by 84% compared to placebo. The study also reported that nearly 60% of Uplizna-treated patients achieved flare-free, corticosteroid-free complete remission at 52 weeks, versus just over 20% in the placebo group.

Argenx Awaits Approval for Vyvgart Prefilled Syringe

Argenx is expecting an FDA decision by April 10 on its prefilled syringe product for Vyvgart (efgartigimod) in patients with generalized myasthenia gravis and chronic inflammatory demyelinating polyneuropathy (CIDP). Vyvgart, a human IgG1 antibody fragment, works by binding to the neonatal Fc receptor, thereby lowering circulating IgG levels.

Initially approved in 2021 for intravenous infusion, Vyvgart received FDA approval for a subcutaneous formulation (Vyvgart Hytrulo) in June 2023. According to Karen Massey, Chief Operating Officer at argenx, the prefilled syringe represents the next stage in Vyvgart's development and could potentially boost product sales by attracting new prescribers and supporting its rollout in CIDP.