Peptide News Digest

#Fda-Approval

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Regulatory · View digest

Regeneron Pharmaceuticals (NASDAQ: REGN) Received FDA Approval Thursday August 20, 2026 for Pasatru (Garetosmab-Grts, an Anti-Activin A Monoclonal Antibody Administered as Subcutaneous Injection) for the Reduction of the Formation of New Heterotopic Ossification (HO) Lesions and Clinician-Assessed Flare-Ups in Adults With Fibrodysplasia Ossificans Progressiva (FOP), a Rare Genetic Disease in Which Skeletal Muscle and Connective Tissue Progressively Turn Into Bone Through Extra-Skeletal Ossification Triggered by Activin A Signaling; The Pasatru Approval Marks the Second FDA-Approved Therapy for FOP After Ipsen's Sohonos (Palovarotene) Approved in 2023, Extends Regeneron's Growing Rare-Disease Commercial Portfolio Alongside Eylea (Aflibercept), Dupixent (Dupilumab), and the Antibody-Drug Conjugate Programs in Development

Regeneron Pharmaceuticals (NASDAQ: REGN) received FDA approval Thursday August 20, 2026 for Pasatru (garetosmab-grts, an anti-activin A monoclonal antibody administered as subcutaneous injection) for the reduction of the formation of new heterotopic ossification (HO) lesions and clinician-assessed flare-ups in adults with fibrodysplasia ossificans progressiva (FOP). FOP is a rare autosomal-dominant genetic disease affecting roughly 1 in 2 million people globally (approximately 800 patients in the US) in which skeletal muscle and connective tissue progressively turn into bone through extra-skeletal ossification triggered by activin A signaling through mutated ACVR1 (activin receptor A type 1) receptors. Patients typically develop the first flare-ups in early childhood, with progressive immobilization by adulthood as ossification advances across major joints. Pasatru's mechanism: garetosmab binds activin A and blocks its signaling through the mutated ACVR1 receptors, reducing the flare-up frequency and slowing new HO lesion formation. The approval marks the second FDA-approved therapy for FOP after Ipsen's Sohonos (palovarotene, a retinoic acid receptor gamma agonist small molecule) approved in 2023. Pasatru offers a mechanistically distinct alternative for patients who cannot tolerate palovarotene or who need combination or sequential therapy. The Pasatru approval extends Regeneron's growing rare-disease commercial portfolio alongside Eylea (aflibercept for wet AMD), Dupixent (dupilumab for atopic dermatitis and asthma), and multiple antibody-drug conjugate programs in development. Pricing and launch details have not been publicly disclosed but rare-disease pricing typically runs $300,000-$500,000 per patient per year.

Regulatory · View digest

FDA Approves Merck's LIPFENDRA (Enlicitide) 20 mg Tablets Thursday July 16 as the First and Only Once-Daily Oral PCSK9 Inhibitor to Reduce LDL-C in Adults With Hypercholesterolemia (Including Heterozygous Familial Hypercholesterolemia): The Novel Macrocyclic Peptide Delivered 56% Placebo-Adjusted LDL Reduction in the CORALreef Lipids Phase 3 Trial and 59% Reduction in CORALreef HeFH, Matching the Efficacy of Injectable PCSK9 Monoclonal Antibodies at a $315 Per Month List Price Roughly One-Third the Cost of Injectable Repatha and Praluent

The US Food and Drug Administration approved Merck's LIPFENDRA (enlicitide) 20 mg tablets Thursday July 16, 2026 as an adjunct to diet and exercise to reduce low-density lipoprotein cholesterol (LDL-C) in adults with hypercholesterolemia, including heterozygous familial hypercholesterolemia (HeFH). LIPFENDRA is a novel macrocyclic peptide and becomes the first FDA-approved oral PCSK9 inhibitor. In the registrational Phase 3 CORALreef Lipids trial, enlicitide achieved a 56% placebo-adjusted LDL-C reduction; in CORALreef HeFH the reduction was 59%. Every other FDA-approved PCSK9 inhibitor (Amgen's Repatha/evolocumab, Regeneron/Sanofi's Praluent/alirocumab) is delivered by subcutaneous injection every two to four weeks; enlicitide is the first approved as a once-daily oral tablet. Merck priced LIPFENDRA at $315 per month list price, roughly one-third the approximately $700-900/month list prices of the injectable PCSK9 antibodies. The approval extends the macrocyclic peptide platform's clinical validation and opens a new oral chapter for a drug class that had been injectable-only since the first FDA approvals in 2015.