Regeneron Announces FDA Approval of Two New Therapies: Aflibercept 8 mg for Retinal Diseases and Pozelimab for CHAPLE Syndrome

On August 18, Regeneron announced that the FDA had approved the listing of two new drugs, Aflibercept 8mg and Pozelimab.

NO.1 Aflibercept 8mg

Eylea 8mg (Aflibercept) Approved for the Treatment of Wet Age-Related Macular Degeneration (wAMD), Diabetic Macular Edema (DME), and Diabetic Retinopathy. This is the second ophthalmology drug developed by Regeneron.

This approval is based on the positive data from the Phase III PULSAR study of aflibercept 8mg for the treatment of wAMD and the Phase II/III PHOTON study for the treatment of DME.

At week 48, both studies met the primary endpoint of non-inferiority, showing that aflibercept 8mg administered every 12 or 16 weeks was non-inferior to aflibercept 2mg administered every 8 weeks in improving Best Corrected Visual Acuity (BCVA) scores. The vast majority of patients were able to maintain the 12- or 16-week injection interval. The success of these two pivotal studies means that the aflibercept 8mg formulation can extend the treatment interval from every 2 months to every 4 months.

In both studies, the safety of 8mg aflibercept was similar to that of 2mg aflibercept, and no patients experienced adverse reactions such as retinal vasculitis, occlusive retinitis, or endophthalmitis.

Aflibercept is a VEGFR-Fc fusion protein jointly developed by Bayer and Regeneron. It was first approved for marketing in the United States in November 2011 under the trade name Eylea (Aflibercept 2mg). Regeneron retains exclusive rights to Aflibercept in the United States, while Bayer has obtained exclusive marketing rights outside the United States.

According to Regeneron's financial report, the sales of Eylea (Aflibercept 2mg) reached $9.647 billion in 2022, increasing by 4% year-on-year. Among this, the sales in the United States were $6.265 billion (exclusively occupied by Regeneron), and the sales in markets outside the United States were $3.383 billion (handled by Bayer, of which Regeneron received $1.3 billion). In the first half of this year, the global sales of Eylea were $4.667 billion, including $2.934 billion in the United States and $1.733 billion in regions outside the United States.

In addition, the marketing application for aflibercept 8mg formulation is also under review in the EU and Japan.

NO.2 Pozelimab

Pozelimab Approved for the Treatment of CHAPLE Syndrome. This is the world's first drug for treating this disease.

CHAPLE Syndrome, also known as CD55 Deficiency with Complement Hyperactivation, Angiopathic Thrombosis, and Protein-Losing Enteropathy or CD55 Deficiency-Mediated Protein-Losing Enteropathy, is an ultra-rare hereditary immune disorder caused by biallelic loss-of-function mutations in CD55 that lead to excessive complement activation. The lack of CD55 in the human body causes the complement system to attack normal cells, damaging lymphatic vessels and blood vessels in the upper digestive tract, resulting in the loss of blood cells and proteins. Most patients experience symptoms such as abdominal pain, bloody diarrhea, vomiting, malnutrition, slow growth, leg swelling (edema), recurrent infections, and thrombosis, which can be life-threatening in severe cases. According to statistics, there are fewer than 100 CHAPLE syndrome patients worldwide.

Pozelimab is a fully human IgG4 monoclonal antibody targeting complement factor C5 developed by Regeneron using its proprietary VelocImmune technology platform. It aims to block the activity of complement factor C5 and prevent complement pathway-mediated diseases.

The approval for marketing in this case is mainly based on the positive results of a Phase II/III single-arm clinical trial (NCT04209634). This study included a total of 10 patients aged 1 year and above with CHAPLE syndrome, aiming to evaluate the efficacy and safety of Pozelimab. Patients were required to receive an initial intravenous injection of Pozelimab at a dose of 30mg/kg on day one, followed by weekly subcutaneous injections of a specific amount of Pozelimab based on body weight. The primary endpoint of the study was the proportion of patients achieving normalization of serum albumin levels and showing improvement in clinical symptoms (including abdominal pain, number of daily bowel movements, and facial and peripheral edema).

The results showed that at week 24 of treatment, 100% of patients had rapid and sustained normalization of serum albumin, with clinical symptoms improved or no deterioration. In addition, the number of hospitalization days and albumin infusion times were significantly reduced, and the age-weight score and age-height score also increased in clinical significance. In terms of safety, 7 patients experienced mild or moderate adverse events (AEs), the most common AEs being iron deficiency, fever, rhinitis, urticaria, and vomiting.

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