United States Food and Drug Administration – Page 5

FDA Rejects Scholar Rock’s SMA Drug Due to Catalent Indiana Manufacturing Issues

4 weeks ago talkbio0Tagged apitegromab, Catalent Indiana, Complete, Drug Approval, manufacturing issues, Response Letter, Scholar Rock, SMA, Spinal Muscular Atrophy, United States Food and Drug Administration

FDA; Scholar Rock; Apitegromab; SMA; Spinal Muscular Atrophy; Catalent Indiana; Complete Response Letter; Manufacturing issues; Drug approval

Ionis Heads to FDA as Antisense Therapy Aces Pivotal Study in Rare Neurological Disease

4 weeks ago talkbio0Tagged access program, Alexander Disease, Antisense Oligonucleotides, Fast Track, Identifier, Improvement, Infrequent, Ionis Pharmaceuticals, nervous system disorder, orphan drug designation, Phase I – III study, Speed, United States Food and Drug Administration, Zilganersen

Ionis Pharmaceuticals; zilganersen; antisense oligonucleotide; Alexander disease; pivotal Phase I–III study; FDA Fast Track designation; rare neurological disease; expanded access program; walk speed improvement; Orphan Drug designation

FDA Delays Decision on Sanofi’s Oral Multiple Sclerosis Drug to December 2025

4 weeks ago talkbio0Tagged BTK inhibitor, Clinical action, December 2025, drug review, Multiple Sclerosis, nrSPMS, PDUFA, sanofi, Tolebrutinib, United States Food and Drug Administration

FDA action date; Sanofi; tolebrutinib; multiple sclerosis; BTK inhibitor; nrSPMS; drug review delay; December 2025; PDUFA

Parkinson’s Patients Prefer Medtronic’s Adaptive Brain Stimulation Over Traditional Settings, Major Study Shows

4 weeks ago talkbio0Tagged ADAPT-PD study, adaptive deep brain stimulation, aDBS, Approved, Deep Brain Stimulation, Medtronic, Parkinson 's disease, personalized neuromodulation, real-time brain monitoring, United States Food and Drug Administration

Parkinson’s disease; adaptive deep brain stimulation; aDBS; Medtronic; ADAPT-PD study; FDA approval; DBS; personalized neuromodulation; real-time brain monitoring

FDA Approves Stealth BioTherapeutics’ Forzinity as First Treatment for Barth Syndrome After Rejection and Delay

4 weeks ago talkbio0Tagged 3-Methylglutaconic aciduria type 2, Abnormality of mitochondrial metabolism, Accelerated, Approved, Elamipretide, Forzinity, Infrequent, Stealth BioTherapeutics, United States Food and Drug Administration

FDA approval; Stealth BioTherapeutics; Forzinity; elamipretide; Barth syndrome; rare disease; mitochondrial dysfunction; accelerated approval

FDA Grants Accelerated Approval to Stealth BioTherapeutics’ Forzinity, the First Treatment for Barth Syndrome

4 weeks ago talkbio0Tagged 3-Methylglutaconic aciduria type 2, Accelerated, Approved, Clinical Trials, Elamipretide, Forzinity, Infrequent, Mitochondrial Diseases, Pediatric Disorder, Stealth BioTherapeutics, United States Food and Drug Administration

FDA approval; Stealth BioTherapeutics; Forzinity; elamipretide; Barth syndrome; mitochondrial disease; accelerated approval; rare disease; pediatric disease; clinical trial

Sanofi’s MS Drug Tolebrutinib Faces FDA Delay Amid Extended Review

4 weeks ago talkbio0Tagged Breakthrough Therapy, Bruton 's tyrosine kinase inhibitor, Deferred, GEMINI trial, HERCULES trial, Multiple Sclerosis, NDA, nrSPMS, PERSEUS trial, regulatory review, sanofi, Tolebrutinib, United States Food and Drug Administration

Sanofi; tolebrutinib; FDA delay; multiple sclerosis; nrSPMS; Bruton’s tyrosine kinase inhibitor; breakthrough therapy; HERCULES trial; GEMINI trial; PERSEUS trial; NDA; regulatory review

Merck Wins FDA Approval for Subcutaneous Keytruda as Exclusivity Challenges Approach

4 weeks ago talkbio0Tagged Approved, Biosimilars, checkpoint inhibitors, Immunotherapy for cancer, Keytruda, Keytruda Qlex, Merck, patent expiration, Solid Neoplasm, Subcutaneous Injections, United States Food and Drug Administration

Merck; Keytruda; subcutaneous injection; FDA approval; Keytruda Qlex; patent expiration; biosimilars; checkpoint inhibitors; solid tumors; cancer immunotherapy