concerns – Talk Bio

Intercept Withdraws Ocaliva from US Market After FDA Safety Concerns

1 month ago talkbio0Tagged Clinical Trials, concerns, Drug withdrawal syndrome, Injury of liver, Intercept Pharmaceuticals, Liver diseases, Ocaliva, Primary Biliary Cholangitis, regulatory setbacks, Safety, United States Food and Drug Administration

Intercept Pharmaceuticals; Ocaliva; liver disease; primary biliary cholangitis; FDA; drug withdrawal; safety concerns; liver injury; clinical trial hold; regulatory setbacks

FDA Rejection Derails PTC Therapeutics’ Rare Disease Hopeful

2 months ago talkbio0Tagged Clinical Trials, concerns, Effectiveness, factor IX, Failure (biologic function), Friedreich 's ataxia, Rare Diseases, Rejection, Response process, United States Food and Drug Administration, vatiquinone

PTC Therapeutics; FDA rejection; vatiquinone; Friedreich’s ataxia; rare disease; efficacy concerns; complete response letter; clinical trial failure

FDA Official Says Sarepta Faces Major Hurdles to Reintroduce DMD Therapy Elevidys

3 months ago talkbio0Tagged approval status, CBER, concerns, ELEVIDYS, gene therapy, Muscular Dystrophy, Duchenne, Pauses, Safety, Scientific Study, shipment, United States Food and Drug Administration

Sarepta; Elevidys; FDA; Duchenne muscular dystrophy; gene therapy; CBER; safety concerns; shipment pause; new studies; approval status

Roche Halts Elevidys Distribution in Several Countries Following Sarepta and FDA Safety Concerns

3 months ago talkbio0Tagged concerns, ELEVIDYS, gene therapy, halt, Muscular Dystrophy, Duchenne, patient deaths, Roche (company), Safety, shipment, United States Food and Drug Administration

Roche; Sarepta Therapeutics; Elevidys; Duchenne muscular dystrophy; gene therapy; FDA; shipment halt; safety concerns; patient deaths

Sarepta Halts US Shipments of Duchenne Therapy Elevidys After FDA Pressure

3 months ago talkbio0Tagged concerns, DMD, ELEVIDYS, gene therapy, Muscular Dystrophy, Duchenne, patient deaths, Safety, shipment, Suspensions, United States Food and Drug Administration

Sarepta Therapeutics; Elevidys; Duchenne muscular dystrophy (DMD); FDA; gene therapy; safety concerns; shipment suspension; patient deaths

Sarepta Stands Behind Elevidys After FDA Requests Withdrawal Amid Safety Concerns

3 months ago talkbio0Tagged Cessation of life, Clinical Trials, concerns, ELEVIDYS, gene therapy, Market, Muscular Dystrophy, Duchenne, Safety, United States Food and Drug Administration, Withdraw (activity)

Sarepta; Elevidys; FDA; gene therapy; market withdrawal; safety concerns; Duchenne muscular dystrophy; deaths; clinical trial hold

FDA Advisory Panel Rejects GSK’s Blenrep in New Combinations Over Safety, Efficacy Concerns

3 months ago talkbio0Tagged Advisory Panel, Blenrep, concerns, DREAMM, Drug Approval, Multiple Myeloma, Ocular Toxicity, Refractory Plasma Cell Myeloma, Relapsing course, Safety, SmithKline Beecham, trials, United States Food and Drug Administration

Blenrep; GSK; FDA Advisory Panel; multiple myeloma; ocular toxicity; safety concerns; DREAMM trials; drug approval; relapsed or refractory multiple myeloma