Functional Concept – Page 4

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

1 month 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

ASCO25 Primer: Navigating Cancer R&D Amid Regulatory Uncertainty

1 month ago talkbio0Tagged ASCO25, Cancer Research, Changing, Diagnostics, expert, Immunotherapy, Neoplasms, perspectives, Policy, Precision Medicine, regulatory uncertainty

ASCO25; cancer research; oncology; regulatory uncertainty; immunotherapy; precision medicine; diagnostics; expert perspectives; policy changes

BioMarin Acquires Inozyme Pharma for $270M, Gaining Phase 3 Rare Disease Therapy

2 months ago talkbio0Tagged Acquisition, BioMarin, Deficiency, ENPP1 gene, Enzyme Replacement Therapy, Infrequent, Inozyme, INZ-701, Pharma, Phase 3 clinical trial

BioMarin; Inozyme Pharma; acquisition; rare disease; INZ-701; ENPP1 Deficiency; enzyme replacement therapy; Phase 3 clinical trial

FDA Delays Decision on Biohaven’s Troriluzole for Rare Disease, Plans Advisory Committee Review

2 months ago talkbio0Tagged Advisory Committees, Ataxia, Spinocerebellar, Biohaven, Deferred, disease drug, Extension, Infrequent, Neurodegenerative Disorders, PDUFA, Troriluzole, United States Food and Drug Administration

Biohaven; FDA delay; troriluzole; spinocerebellar ataxia (SCA); rare disease drug; PDUFA extension; advisory committee; neurodegenerative disease

AbbVie Advances Solid Tumor Program with FDA Approval of Emrelis for Lung Cancer

2 months ago talkbio0Tagged AbbVie, Accelerated, ADC, Approved, Emrelis, Malignant neoplasm of lung, MET protein, human, Non-Small Cell Lung Carcinoma, Protein Overexpression, Solid Neoplasm, Telisotuzumab, therapeutic autologous dendritic cells, United States Food and Drug Administration, vedotin-tllv

AbbVie; Emrelis; FDA Approval; ADC; solid tumor; lung cancer; non-small cell lung cancer; c-Met overexpression; telisotuzumab vedotin-tllv; accelerated approval

Sanofi Announces $20 Billion US Investment Over Five Years

2 months ago talkbio0Tagged Big Pharma, development aspects, domestic partnerships, Drug Industry, Investments, Manufacturing, research, sanofi, Surgical construction, tariffs, U.S.

Sanofi; $20 billion investment; US pharmaceutical industry; manufacturing; research and development; tariffs; job creation; Big Pharma; domestic partnerships