Cautionary Warning

FDA restricts bluebird bio’s Skysona to CALD patients without suitable stem cell donors after higher blood cancer risk emerges

1 month ago talkbio0Tagged boxed, CALD, Cautionary Warning, Cerebral Adrenoleukodystrophy, Donor person, Elivaldogene autotemcel, gene therapy, Hematologic Neoplasms, HLA Match, label update, Leukemia, Myelocytic, Acute, MYELODYSPLASTIC SYNDROME, Safety, Skysona, United States Food and Drug Administration

FDA; bluebird bio; Skysona; elivaldogene autotemcel; eli-cel; cerebral adrenoleukodystrophy; CALD; hematologic malignancies; myelodysplastic syndrome (MDS); acute myeloid leukemia (AML); boxed warning; label update; postmarketing safety; HLA-matched donor; gene therapy safety

FDA tightens Skysona label, restricting use to CALD patients without alternative options due to increased blood cancer risk

1 month ago talkbio0Tagged boxed, CALD, Cautionary Warning, Cerebral Adrenoleukodystrophy, Elivaldogene autotemcel, gene therapy, Hematologic Neoplasms, HLA‑matched donor, labeling changes, Leukemia, Myelocytic, Acute, MYELODYSPLASTIC SYNDROME, Safety, Skysona, United States Food and Drug Administration

FDA; bluebird bio; Skysona; elivaldogene autotemcel; labeling changes; boxed warning; hematologic malignancy; myelodysplastic syndrome; acute myeloid leukemia; cerebral adrenoleukodystrophy; CALD; HLA‑matched donor; gene therapy safety; post‑marketing reports

Amid MFN Talks, Lilly Chief Warns US Adoption of International Drug Prices Could Bring ‘Worst of Two Worlds’

1 month ago talkbio0Tagged Cautionary Warning, Chief, Does speak, Drug Industry, drug price, Eli Lilly, International Drug Prices, MFN, Policy, U.S.

Lilly chief; MFN talks; international drug prices; US drug pricing; pharmaceutical industry; policy warning

Sarepta Shifts Focus to siRNA Platform, Lays Off 500 After Elevidys Receives Black Box Warning

2 months ago talkbio0Tagged acute liver injury, black box, Cautionary Warning, ELEVIDYS, gene therapy, layoffs, Muscular Dystrophy, Duchenne, pipeline shift, restructuring, RNA, Small Interfering

Sarepta Therapeutics; siRNA platform; Elevidys; Duchenne muscular dystrophy; layoffs; black box warning; pipeline shift; acute liver injury; gene therapy; restructuring

Applied Therapeutics’ Rare Disease Drug Fails Another Phase 3 Trial After FDA Rejection

4 months ago talkbio0Tagged Aldose Reductase Inhibitor, Apply, Cautionary Warning, Classical galactosemia, clinical trial conduct, Govorestat, Infrequent, Rejection, United States Food and Drug Administration

Applied Therapeutics; govorestat; rare disease; phase 3 failure; FDA rejection; classic galactosemia; aldose reductase inhibitor; clinical trial conduct; FDA warning letter