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BeOne Medicines’ BGB-16673 Receives the EMA’s PRIME Designation to Treat Waldenstrom’s Macroglobulinemia

Shots: The EMA has granted PRIority MEdicines (PRIME) designation to BGB-16673 for the treatment of pts with Waldenstrom’s macroglobulinemia (WM), who previously were treated with a BTK inhibitor Designation was based on data showing BGB-16673’s novel ability to degrade both wild-type & mutant BTK forms, incl. resistance-linked variants, which demonstrated anti-tumor activity in B-cell malignancies…

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Purpose Pharma’s Attrogy Receives the CHMP’s Positive Opinion for the Treatment of Hereditary Transthyretin-mediated Amyloidosis  

Shots:  The CHMP has recommended Attrogy (diflunisal) to treat hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adult pts. with stage 1 or stage 2 polyneuropathy  The EC will make decision within 67 days, and if approved, Attrogy will be authorized in all EU & EEA countries Iceland, Lichtenstein and Norway  Diflunisal has received EU ODD for…

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Geron

Geron Receives the EC’s Approval for Rytelo (Imetelstat) to Treat Transfusion-Dependent Anemia due to Lower-Risk MDS

Shots: The EC has approved Rytelo to treat TD anemia in adults with very low, low, or intermediate risk non-del(5q) MDS, unresponsive or ineligible for erythropoietin-based therapy in 30 EEA states; commercialization to begin in select EU states by 2026 pending country-by-country reimbursement Approval was based on P-III (IMerge) study of Rytelo vs PBO…

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Viewpoints_Phil Lambert

Enhancing DMD Care: Phil Lambert from Satellos in a Riveting Conversation with PharmaShots

Shots:  Conventional therapeutic models for Duchenne Muscular Dystrophy (DMD) primarily focus on the role of dystrophin in maintaining muscle cell integrity. However, Satellos’ SAT-3247 takes a novel approach, targeting the lack of dystrophin in muscle stem cells to restore muscle fiber regeneration and repair  Today at PharmaShots, we are joined by Phil Lambert, Chief Scientific…

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New Drug Designations - August 2024

New Drug Designations – August 2024 

Shots:   PharmaShots' designation report provides a concise overview of several drugs and their designations by the FDA, NMPA and EMA. August month’s report includes designations allotted to 10 small molecules, 9 biologics, 7 cell & gene therapies, 1 vaccine, 2 radiopharmaceutical, 1 peptide, 1 antineoplastic and 2 devices   In August 2024, Invenra’s INV724 received the…

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New Drug Designations - July 2024

New Drug Designations – July 2024

Shots:   PharmaShots' designation report provides a concise overview of several drugs and their designations by the FDA, NMPA and EMA. This month’s report includes designations allotted to 16 small molecules, 5 biologics, 11 cell & gene therapies, 1 recombinant protein, 1 vaccine, 1 immunotherapy, 1 radiopharmaceutical, 1 antisense oligonucleotide, 3 RNA therapies 1 herbal medicine…

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New Drug Designations - June 2024

New Drug Designations – June 2024

Shots:   PharmaShots' designation report provides a concise overview of several drugs and their designations by the FDA, NMPA and EMA. This month’s report includes designations allotted to 7 small molecules, 5 biologics, 8 cell & gene therapies, 2 recombinant fusion protein, and 3 devices   This month, ChromaDex’s Nicotinamide Riboside Chloride received US FDA’s ODD &…

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New Drug Designations - May 2024

New Drug Designations – May 2024

Shots:  PharmaShots' designation report provides a concise overview of several drugs and their designations by the FDA, NMPA and EMA. This month’s report includes designations allotted to 12 small molecules, 7 biologics, 9 cell & gene therapies, 1 vaccine, 1 peptide, 1 RNA candidate, 1 recombinant fusion protein, 1 imaging agent and 6 devices  This…

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New Drug Designations - April 2024

New Drug Designations – April 2024

Shots:  PharmaShots' designation report provides a concise overview of several drugs and their designations by the FDA, NMPA and EMA. This month’s report includes designations allotted to 11 small molecules, 7 biologics, 7 cell & gene therapies, 1 antiviral, 1 peptide and 3 devices   Lisata Therapeutics’ LSTA1 received ODD this month and was granted with…

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