The US FDA Grants Accelerated Approval to Stealth BioTherapeutics’ Forzinity for Barth Syndrome

Shots: The US FDA has received accelerated approval from the US FDA for Forzinity (elamipretide HCl)] to improve muscle strength in pts with Barth syndrome (Wt.≥30 kg or 66 lbs) Approval was based on efficacy and safety data from the TAZPOWER trial, which showed improved knee muscle strength Forzinity is a mitochondrial cardiolipin binder. It…

ByDipanshu DixitSeptember 19, 2025

NEWS

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 inhibitorDesignation 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…

ByRidhi RastogiAugust 1, 2025

NEWS

Omeros Submits MAA to the EMA for Narsoplimab (OMS721) to Treat TA-TMA

SShots:Omeros submitted an MAA to the EMA for narsoplimab to treat TA-TMA, with data demonstrating a 61% response rate and a 3x improvement in OS vs matched external controls; over 130 pts treated under expanded access data were also includedEMA granted Narsoplimab ODD status, and CHMP review will start mid-July under centralized…

ByPrince GiriJune 30, 2025

NEWS

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…

ByDipanshu DixitApril 25, 2025

NEWS

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…

ByRidhi RastogiMarch 12, 2025

VIEWPOINTS

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…

BySaurabh ChaubeyNovember 20, 2024

INSIGHTS+

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…

BySenior EditorSeptember 25, 2024

INSIGHTS+

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…

BySenior EditorAugust 27, 2024

INSIGHTS+

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 &…

BySenior EditorJuly 30, 2024

INSIGHTS+

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…

BySenior EditorJune 25, 2024

The US FDA Grants Accelerated Approval to Stealth BioTherapeutics’ Forzinity for Barth Syndrome

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

Omeros Submits MAA to the EMA for Narsoplimab (OMS721) to Treat TA-TMA

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

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

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

New Drug Designations – August 2024

New Drug Designations – July 2024

New Drug Designations – June 2024

New Drug Designations – May 2024

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