FDA Decision Deadlines Loom for Regeneron, Sanofi, and Abeona

In the coming weeks, the U.S. Food and Drug Administration (FDA) is set to make critical decisions on three notable pharmaceutical products, including two from Regeneron Pharmaceuticals and one from Abeona Therapeutics. These decisions could potentially reshape treatment options for several conditions, ranging from chronic skin disorders to rare genetic diseases.

Regeneron and Sanofi Seek Dupixent Expansion for Chronic Spontaneous Urticaria

Regeneron and Sanofi are making a second attempt to expand the label of their blockbuster anti-inflammatory antibody, Dupixent, to include chronic spontaneous urticaria (CSU). The FDA's decision is expected by April 18, following an initial rejection in October 2023.

The companies have bolstered their application with additional data from the Phase III LIBERTY-CUPID clinical program. Study A demonstrated that Dupixent could nearly double itch reduction and urticaria activity scores compared to antihistamines. While Study B showed numeric improvements in patients refractory to omalizumab, it did not reach statistical significance. A third trial, Study C, revealed a nearly 50% reduction in itch and urticaria activity.

If approved, Dupixent would become the first targeted therapy for CSU in a decade, potentially offering new hope for patients with this challenging condition.

Regeneron Aims to Extend Eylea HD's Dosing Interval

Regeneron is seeking to enhance the competitiveness of its high-dose (HD) formulation of Eylea by proposing a longer dosing interval. The FDA's verdict on this application is anticipated by April 20.

The company is pursuing approval for a 24-week dosing regimen for Eylea HD in wet age-related macular degeneration and diabetic macular edema. This extended interval could significantly improve patient convenience without compromising efficacy. Data from the Phase III PULSAR trial demonstrated that Eylea HD maintained vision gains even with dosing intervals of at least 12 weeks.

Despite its improved product profile, Eylea HD has faced challenges in market penetration. In Q4 2024, the drug generated $305 million in sales, falling short of analyst expectations of $336 million.

Abeona's Gene Therapy for Rare Skin Disease Awaits FDA Decision

Abeona Therapeutics is on the cusp of a potential breakthrough for patients with recessive dystrophic epidermolysis bullosa (RDEB), a rare genetic skin disorder. The FDA is set to decide on the approval of prademagene zamikeracel (pz-cel), an autologous cell-based gene therapy, by April 29.

Pz-cel aims to address the underlying cause of RDEB by restoring patients' ability to produce functional collagen. The therapy utilizes patients' own skin cells, which are genetically modified to correct the mutated COL7A1 gene responsible for the disease.

In the Phase III VITAL trial, pz-cel demonstrated significant efficacy, with treated patients experiencing wound healing success greater than 50%. At the 6-month mark, wounds in the pz-cel arm decreased by 81% compared to controls.

If approved, pz-cel would represent a groundbreaking advancement as the first autologous, cell-based gene therapy for RDEB, offering new hope for patients with this debilitating condition.