Lined up for the FDA in the coming weeks are a cell-based gene therapy for a rare skin disease and two product expansions for Regeneron, one with partner Sanofi.
The FDA has three big decisions slated for the back half of April, two of which are for Regeneron’s blockbuster franchises.
Read below for more.
Regeneron, Sanofi Eye Another Expansion for Dupixent
Following an initial rejection in October 2023, Regeneron and Sanofi are again trying to expand their anti-inflammatory antibody Dupixent into chronic spontaneous urticaria (CSU). The FDA’s decision is due on April 18.
To support their bid for label expansion, the pharma partners submitted data from the Phase III LIBERTY-CUPID clinical program. Data from Study A, released in July 2021, showed that Dupixent could nearly double itch reduction and urticaria activity scores versus antihistamines. Study B, meanwhile, found a numeric improvement in itching and hives versus antihistamines in patients refractory to omalizumab, as per a February 2022 readout, though the study did not reach statistical significance. It was terminated due to futility.
These two studies formed the backbone of Dupixent’s initial failed attempt at an approval for CSU, which the FDA rebuffed due to the lack of efficacy data. In their resubmission, Regeneron and Sanofi filed additional data from a third trial, dubbed Study C, which also showed a nearly 50% drop in itch and urticaria activity.
If approved, Dupixent would be the first targeted option for CSU in a decade, according to a November 2024 news release.
Regeneron Proposes Longer Dosing Interval for Eylea HD
In a bid to maintain the market dominance of its blockbuster biologic Eylea, Regeneron is working to improve the product profile of the high-dose (HD) formulation of the drug. In line with this, the company is proposing a longer dosing duration for Eylea HD, for which the FDA is expected to release its verdict by April 20.
In its presentation at the J.P. Morgan Healthcare Conference in January, Regeneron announced that the FDA was already reviewing its application for 24-week dosing for Eylea HD in wet age-related macular degeneration and diabetic macular edema. The biotech is also building toward an application for prolonged Eylea HD dosing in retinal vein occlusion.
Eylea HD is a longer-acting version of the original drug and, with less frequent injection, it is designed to be more convenient to the patient. In August 2023, data from Regeneron’s Phase III PULSAR trial confirmed that even at dosing intervals at least 12 weeks long, high-dose Eylea did not sacrifice vision gains while also helping patients maintain their treatment schedules.
Despite a differentiated product profile, Eylea HD has had trouble getting off the ground. In the fourth quarter of 2024, the drug brought in $305 million in sales, far below the analyst consensus of $336 million.
Abeona Awaits Approval for Autologous Gene Therapy in Rare Skin Disease
Abeona Therapeutics is developing prademagene zamikeracel, also called pz-cel, for recessive dystrophic epidermolysis bullosa. The FDA is reviewing the biotech’s application and is expected to release its verdict on April 29.
Recessive dystrophic epidermolysis bullosa (RDEB) is a rare genetic disorder characterized by fragile skin that is highly prone to wounds and blisters. The disease is caused by a mutation in the COL7A1 gene, which encodes for a key component of collagen that helps to stabilize and strengthen the skin.
Pz-cel addresses this underlying cause of RDEB by restoring a patient’s ability to produce functional collagen. The therapy is derived from patients’ own skin cells with genetically corrected copies of COL7A1.
In the Phase III VITAL trial, patients treated with pz-cel saw wound healing success greater than 50%. At 6 months, wounds dropped by 81% in the pz-cel arm versus controls. Abeona also filed Phase I/IIa data with up to 8 years of follow-up to support pz-cel’s application.
If approved, pz-cel would be the first autologous, cell-based gene therapy for RDEB, the biotech said in November 2024.
