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Regeneron (REGN) Rare Disease Drug Gets FDA's Priority Review
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Regeneron Pharmaceuticals, Inc. (REGN - Free Report) announced that the FDA has accepted for Priority Review the biologics license application (BLA) for pozelimab. The regulatory body has set a target action date of Aug 20, 2023, for the candidate.
Pozelimab is an investigational fully human monoclonal antibody designed to block the activity of complement factor C5, a protein involved in complement system activation.
The BLA is seeking approval of the candidate as a treatment for adults and children as young as one year old with CHAPLE disease (also known as CD55 deficiency with Hyperactivation of complement, Angiopathic thrombosis and Protein Losing Enteropathy or CD55-deficient protein-losing enteropathy).
CHAPLE is an ultra-rare hereditary immune disease that causes overactivation of the complement system, leading to potentially life-threatening abdominal and cardiovascular symptoms.
The BLA is supported by results from a phase II/III open-label study that evaluated the efficacy and safety of pozelimab in 10 patients aged 1 year or older.
In April 2020, the FDA designated pozelimab for the treatment of CHAPLE as a drug for a “rare pediatric disease.” This gives Regeneron an opportunity to receive a rare pediatric disease priority review voucher should pozelimab be approved for CHAPLE. The candidate was also granted Orphan Drug Designation. Pozelimab was also granted a Fast Track designation in September 2022.
The candidate is also being evaluated in combination with Alnylam’s Pharmaceuticals’ (ALNY - Free Report) cemdisiran (a siRNAi C5 inhibitor) as an investigational combination therapy for the treatment of other complement-mediated disorders, including paroxysmal nocturnal hemoglobinuria (PNH) and myasthenia gravis (MG).
In 2019, Regeneron and Alnylam entered into a global, strategic collaboration to discover, develop and commercialize RNAi therapeutics for a broad range of diseases by addressing therapeutic disease targets expressed in the eye and central nervous system ("CNS"), in addition to a select number of targets expressed in the liver.
Regeneron’s shares have gained 24.2% in the past year against the industry’s decline of 6.7%.
Image Source: Zacks Investment Research
Regeneron delivered better-than-expected fourth-quarter 2022 results even though lead drug Eylea (aflibercept) sales declined.
Regeneron is developing Eylea in collaboration with Bayer (BAYRY - Free Report) . Regeneron records net product sales of Eylea in the United States and Bayer records net product sales of Eylea outside the United States. Regeneron records its share of profits/losses in connection with sales of Eylea outside the United States.
The FDA recently approved the label expansion of Eylea for the treatment of preterm infants with retinopathy of prematurity (ROP).
Image: Bigstock
Regeneron (REGN) Rare Disease Drug Gets FDA's Priority Review
Regeneron Pharmaceuticals, Inc. (REGN - Free Report) announced that the FDA has accepted for Priority Review the biologics license application (BLA) for pozelimab. The regulatory body has set a target action date of Aug 20, 2023, for the candidate.
Pozelimab is an investigational fully human monoclonal antibody designed to block the activity of complement factor C5, a protein involved in complement system activation.
The BLA is seeking approval of the candidate as a treatment for adults and children as young as one year old with CHAPLE disease (also known as CD55 deficiency with Hyperactivation of complement, Angiopathic thrombosis and Protein Losing Enteropathy or CD55-deficient protein-losing enteropathy).
CHAPLE is an ultra-rare hereditary immune disease that causes overactivation of the complement system, leading to potentially life-threatening abdominal and cardiovascular symptoms.
The BLA is supported by results from a phase II/III open-label study that evaluated the efficacy and safety of pozelimab in 10 patients aged 1 year or older.
In April 2020, the FDA designated pozelimab for the treatment of CHAPLE as a drug for a “rare pediatric disease.” This gives Regeneron an opportunity to receive a rare pediatric disease priority review voucher should pozelimab be approved for CHAPLE. The candidate was also granted Orphan Drug Designation. Pozelimab was also granted a Fast Track designation in September 2022.
The candidate is also being evaluated in combination with Alnylam’s Pharmaceuticals’ (ALNY - Free Report) cemdisiran (a siRNAi C5 inhibitor) as an investigational combination therapy for the treatment of other complement-mediated disorders, including paroxysmal nocturnal hemoglobinuria (PNH) and myasthenia gravis (MG).
In 2019, Regeneron and Alnylam entered into a global, strategic collaboration to discover, develop and commercialize RNAi therapeutics for a broad range of diseases by addressing therapeutic disease targets expressed in the eye and central nervous system ("CNS"), in addition to a select number of targets expressed in the liver.
Regeneron’s shares have gained 24.2% in the past year against the industry’s decline of 6.7%.
Image Source: Zacks Investment Research
Regeneron delivered better-than-expected fourth-quarter 2022 results even though lead drug Eylea (aflibercept) sales declined.
Regeneron is developing Eylea in collaboration with Bayer (BAYRY - Free Report) . Regeneron records net product sales of Eylea in the United States and Bayer records net product sales of Eylea outside the United States. Regeneron records its share of profits/losses in connection with sales of Eylea outside the United States.
The FDA recently approved the label expansion of Eylea for the treatment of preterm infants with retinopathy of prematurity (ROP).
Regeneron currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.