Temporal Concept – Page 7

New Confirmatory Study Suggests Progress in Applied Therapeutics–FDA Alignment for Rare Disease Drug

3 months ago talkbio0Tagged Apply, Clinical Trials, CMT-SORD, confirmatory study, Govorestat, Infrequent, regulatory alignment, United States Food and Drug Administration

Applied Therapeutics; govorestat; CMT-SORD; rare disease; FDA; confirmatory study; clinical trial; regulatory alignment

Sanofi Injects $625M Into Biotech Investment Arm to Accelerate Innovation

3 months ago talkbio0Tagged Cash, cell/gene therapy, digital health, expansion, Immunology, Infrequent, Infusion procedures, Investments, Neurology speciality, portfolio, sanofi, Sanofi Ventures, venture capital

Sanofi; Sanofi Ventures; biotech investment; $625M cash infusion; digital health; immunology; rare diseases; neurology; venture capital; cell/gene therapy; portfolio expansion

BMS Breaks Clinical Losing Streak with Positive Phase 3 Myeloma Readout for Iberdomide

3 months ago talkbio0Tagged CELMoD, Clinical Trials, EXCALIBER-RRMM, iberdomide, Minimal Residual Disease Negativity, Multiple Myeloma, Myers, Phase 3, Progression-Free Survival, Protein Degradation, Metabolic, Relapsing course, squibb, Unresponsive to Treatment

Bristol Myers Squibb; iberdomide; multiple myeloma; clinical trial; EXCALIBER-RRMM; minimal residual disease negativity; CELMoD; protein degradation; progression-free survival; phase 3; relapsed or refractory

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

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

3 months 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

3 months 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

3 months 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

ACIP Vote Restricts Combined MMRV Vaccine for Children Under 4, Raising Access Concerns

3 months ago talkbio0Tagged ACIP, CDC, CDC vaccine recommendation, Chickenpox, Child, Childhood, Immunization, measles, MMRV vaccine, Mumps, Rubella, vaccine access, Vaccines

ACIP; MMRV vaccine; measles; mumps; rubella; varicella; childhood immunization; Vaccines for Children (VFC) program; CDC vaccine recommendation; vaccine access

Regeneron Prepares U.S. Submission for Garetosmab After Breakthrough Phase III Result in Ultra-Rare FOP

3 months ago talkbio0Tagged bone lesion prevention, Drugs, Orphan, fibrodysplasia, Fibrodysplasia Ossificans Progressiva, Garetosmab, Infrequent, OPTIMA trial, ossificans, Phase III, progressiva, Regeneron, Submission, United States Food and Drug Administration

Regeneron; garetosmab; FOP; fibrodysplasia ossificans progressiva; Phase III; OPTIMA trial; bone lesion prevention; rare disease; FDA submission; orphan drug

Areteia Therapeutics Posts Phase 3 Win for Oral Asthma Drug Dexpramipexole

3 months ago talkbio0Tagged Areteia, biologic blockbusters, Dexpramipexole, EXHALE-4, oral asthma therapy, Phase 3, Pulmonary Eosinophilia, topline

Areteia Therapeutics; dexpramipexole; Phase 3; oral asthma therapy; biologic blockbusters; eosinophilic asthma; EXHALE-4; topline results