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INSIGHTS+

Disease of the Month – Paroxysmal nocturnal hemoglobinuria (PNH) 

Shots:  Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired blood disorder where the body's immune system attacks and destroys red blood cells and platelets  PharmaShots’ Disease of the Month report aims to educate a broad audience about health conditions that affect communities worldwide.   This report provides a comprehensive overview of disease, including its characteristics,…
October 29, 2025

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NEWS

Novo Nordisk Enters ~$2.1B Deal with Omeros to Advance Zaltenibart for Rare Blood & Kidney Disorders

Shots: Novo Nordisk has entered into a definitive asset purchase & license agreement with Omeros to develop zaltenibart (MASP-3 inhibitor) for rare blood & kidney disorders, while Omeros retains certain rights to other preclinical MASP-3 programs, incl. small-molecule MASP-3 inhibitors for select indications As per the deal, Novo will receive exclusive global rights to develop…
October 16, 2025

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Enhancing Hematologic Care: Lorah Perlee from Regeneron in a Riveting Dialogue Exchange with PharmaShots
VIEWPOINTS

Enhancing Hematologic Care: Lorah Perlee from Regeneron in a Riveting Dialogue Exchange with PharmaShots

Shots:  At ASH 2024, Regeneron shared data from the P-III exploratory cohort investigating the pozelimab and cemdisiran combination (poze-cemdi) in patients with paroxysmal nocturnal hemoglobinuria  The novel combination (poze-cemdi) achieved meaningful control of intravascular hemolysis compared to ravulizumab   PharmaShots welcomes Lorah Perlee, Vice President, Global Program Head, Hematology and Translational Sciences at Regeneron, for an…
June 23, 2025

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Insights+ Key Biosimilars Events of July 2024
INSIGHTS+

Insights+ Key Biosimilars Events of July 2024

Shots:     Biosimilars are developed to be highly similar versions of approved biologics in terms of safety, purity, and potency      Biosimilars are expected to be a cost-effective alternative to the high-priced branded biologics, offering significant and much-needed cost savings to both payers and patients      The major highlights were the US FDA’s approval of Samsung Bioepis’ Epysqli for Treating Paroxysmal…
August 1, 2024

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Novartis
NEWS

Novartis Reports the CHMP’s Positive Opinion for Fabhalta (iptacopan) to Treat Paroxysmal Nocturnal Hemoglobinuria (PNH)

Shots:  The opinion was based on the P-III (APPLY-PNH) & (APPOINT-PNH) trials of Fabhalta (200mg, oral, BID) in PNH patients with residual anemia who switched from Anti-C5 and complement inhibitor naïve (incl. anti-C5 therapies) respectively for 24wks.  In the APPLY-PNH study, 82.3% vs 2.0% had a ≥2 g/dL increase in Hb levels without transfusions while…
March 22, 2024

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