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…

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…

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…

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…

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 &…

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…

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…

Shots:
PharmaShots' designation report provides a concise overview of several drugs and their designations by the FDA, MHRA and EC. This month’s report includes designations allotted to 10 small molecules, 6 biologics, 5 cell & gene therapy, 1 recombinant fusion protein, 2 actineoplastics, 1 antidepressant, 1 drug conjugate, 1 analgesic and 6 devices
Trellis Bioscience's…

Shots:
PharmaShots' designation report provides a concise overview of several drugs and their designations by the US FDA, EMA and China. This month’s report includes 3 biological drugs, 12 small molecules, 13 cell and gene therapies, 1 peptide, 3 exosome-based therapy and 4 devices
Atsena Therapeutics’ capsid AAV.SPR gene therapy, focused on the treatment of Leber congenital…

Shots:
PharmaShots' designation report provides a concise overview of several drugs and their designations by the US FDA and the EMA. This month’s report includes 2 biological drugs, 12 small molecules, 10 cell and gene therapies, 2 vaccines, 1 peptide, 1 exosome-based therapy and 3 devices
HuidaGene Therapeutics’ HG204 CRISPR RNA-editing therapy, focused on the treatment of MECP2 duplication…