Regeneron Gets FDA Approvals on New Biologic and New High Dose for Eye Disease Therapy

On Aug. 18, 2023, Regeneron Pharmaceuticals announced that FDA has approved Veopoz (pozelimab-bbfg), a monoclonal antibody, for treating adult and pediatric patients one year and older with CHAPLE disease, or CD55-deficient protein-losing enteropathy. The company also announced that FDA has approved Eylea HD (aflibercept) Injection 8 mg, a high-dose version of Eylea, for treating wet age-related macular degeneration (wAMD), diabetic macular edema (DME), and diabetic retinopathy.

According to a company press release, the pozelimab approval marks the first and only treatment indicated specifically for CHAPLE. This approval also signals that pre-approval inspection issues related to the aflibercept 8 mg biologics license application (BLA) have been addressed and that FDA action on the aflibercept 8 mg BLA is expected in the next few weeks.

CHAPLE is a rare, hereditary life-threatening immune disease that is driven by an overactivation of the complement system. Normally, the complement system in healthy individuals is a mechanism for destroying microbes. For those living with CHAPLE, however, individuals are unable to regulate complement activity because of mutations in their CD55 gene, and without proper CD55 regulation, the complement system may attack normal cells. This can cause damage to blood and lymph vessels along the upper digestive tract and lead to loss of circulating proteins. According to the press release, fewer than 10 patients have been identified in the United States who have the disease.

“Most patients with CHAPLE disease are children who face severely debilitating symptoms and often life-threatening complications that begin in infancy,” said Michael Lenardo, chief, Molecular Development of the Immune System Section and co-director, Clinical Genomics Program, National Institute of Allergy and Infectious Disease, National Institutes of Health, in the company press release. “As an investigator in this pivotal trial and one of the discoverers of this disease, I saw first-hand the transformational clinical improvement that pozelimab achieves in those suffering from CHAPLE. The approval of pozelimab is a milestone to celebrate, providing a new medicine that can help these long-suffering patients.”

“Beyond CHAPLE, we believe [pozelimab] has promise in a variety of complement-mediated diseases and are driving forward several clinical programs to explore its broader potential,” said George D. Yancopoulos, board co-chair, president, and chief scientific officer, Regeneron, in the release.

Meanwhile, the approval of Eylea HD Injection 8 mg was based on the 48-week results of two double-masked, active-controlled pivotal trials (PULSAR in wAMD and PHOTON in DME) which evaluated Eylea HD compared to Eylea Injection 2 mg. Both trials met their primary endpoint. Eylea HD demonstrated non-inferior and clinically equivalent vision gains at 48 weeks (with both 12- and 16-week dosing regimens following three initial monthly doses), compared to an Eylea 8-week dosing regimen after initial monthly doses. According to a company press release, the vast majority of patients randomized at baseline to Eylea HD 12- or 16-week dosing regimens (following the three initial monthly doses) were able to maintain these dosing intervals throughout the 48 weeks.

“The FDA approval of Eylea HD is an important advancement in retinal care,” said Peter Kaiser, Chaney Family endowed chair in Ophthalmology Research at the Cole Eye Institute and professor of Ophthalmology at Cleveland Clinic Lerner College of Medicine, in the release. “With Eylea HD, patients with wet age-related macular degeneration or diabetic retinal disease can now receive less frequent injections after their initial monthly doses and still experience the similar visual gains, anatomic improvements, and safety profile of Eylea.”

Source: Regeneron Pharmaceuticals for Veopoz approval and Eylea HD approval