Functional Concept – Page 4

FDA Launches New Submission Program for Rare Disease Gene Therapies via CDER and CBER

2 months ago talkbio0Tagged Accelerated, Approved, CBER, CDER, gene therapy, Hereditary Diseases, Infrequent, RDEP, regulatory guidance, submission program, United States Food and Drug Administration

CDER; CBER; rare disease; gene therapy; FDA; submission program; accelerated approval; RDEP; regulatory guidance; rare genetic disorders

FDA Announces New Rare Disease Approval Programme

2 months ago talkbio0Tagged Accelerated, Approved, Biological Factors, conditional approval pathway, drug review, Endpoints, Infrequent, real-world data, streamlined process, Surrogate, United States Food and Drug Administration

FDA; rare disease; conditional approval pathway; accelerated approval; biologics; drug review; surrogate endpoints; real-world data; streamlined process

Eisai Secures FDA Approval for Leqembi Autoinjector for Maintenance Dosing in Alzheimer’s Patients

2 months ago talkbio0Tagged Alzheimer 's disease, Approved, at-home, AutoInjector, Biogen, dosing, Eisai, Lecanemab, Leqembi, Maintenance, Subcutaneous Injections, United States Food and Drug Administration

Eisai; Leqembi; lecanemab; FDA approval; autoinjector; subcutaneous injection; maintenance dosing; Alzheimer’s disease; at-home treatment; Biogen

Teva Launches First Generic GLP-1 Weight-Loss Drug in the US

2 months ago talkbio0Tagged Approved, Generic Drugs, Glucagon-Like Peptide 1, liraglutide, Obesity, Saxenda, Teva, United States, United States Food and Drug Administration, Weight Loss

Teva; GLP-1; generic; weight loss; liraglutide; FDA approval; Saxenda; obesity treatment; United States

J&J Halts Phase 2a Rheumatoid Arthritis Program for Imaavy (Nipocalimab) after Lackluster Results

2 months ago talkbio0Tagged Autoimmune Diseases, Clinical Research, Clinical trial failure, daisy, Imaavy, Johnson and Johnson, Nipocalimab, Phase 2a Trial, Rheumatoid Arthritis

Johnson & Johnson; Imaavy; nipocalimab; rheumatoid arthritis; Phase 2a trial; DAISY study; clinical trial failure; anti-TNFα therapy; autoimmune diseases

Personalizing Metabolic Care with Digital Twins: Recent Developments

2 months ago talkbio0Tagged Administration occupational activities, Artificial Intelligence, Biological Markers, Diabetes, digital twins, Glucagon-Like Peptide 1, Health care facility, metabolic health, Obesity, Precision Medicine, real-time guidance, Reversal, Twin Health

digital twins; metabolic health; AI healthcare; personalized medicine; biomarkers; diabetes management; obesity reversal; real-time guidance; GLP-1; Twin Health

Wugen Nabs $115M for Pivotal Study of Allogeneic CAR-T

2 months ago talkbio0Tagged Allogeneic, CAR-T, Clinical Trials, fratricide-resistant, T-cell malignancies, Wugen

Wugen; CAR-T; allogeneic; off-the-shelf therapy; fratricide-resistant; funding; pivotal trial; T-cell malignancies

Amylyx Discontinues Rare Disease Program After Relyvrio Fails to Show Benefit Over Placebo

2 months ago talkbio0Tagged AMX0035, Amylyx, Amyotrophic Lateral Sclerosis, Clinical trial failure, Discontinuation (procedure), disease program, Infrequent, Placebos, Progressive, Relyvrio, Supranuclear paralysis

Amylyx; Relyvrio; AMX0035; progressive supranuclear palsy; ALS; clinical trial failure; rare disease program; placebo; discontinuation

Dynavax Highlights Comparable Efficacy and Improved Tolerability of Z-1018 Shingles Vaccine vs. Shingrix

2 months ago talkbio0Tagged aspects of adverse effects, Dynavax, Effectiveness, Herpes zoster disease, Immunogenicity (package insert), Shingrix, Vaccines, Z-1018

Dynavax; Z-1018; shingles vaccine; Shingrix; vaccine efficacy; side effects; Phase 1/2 trial; immunogenicity

FDA Lifts Hold on Rocket Pharmaceuticals’ Pivotal Gene Therapy Study at Lower Dose

2 months ago talkbio0Tagged C3 complement inhibitor, Capillary Leak Syndrome, Dosage, gene therapy, Glycogen Storage Disease Type IIb, Individual Adjustment, Phase 2 Clinical Trials, Rocket, RP-A501, Study on Hold, United States Food and Drug Administration

FDA; Rocket Pharmaceuticals; gene therapy; Danon disease; clinical hold; RP-A501; dose adjustment; C3 complement inhibitor; capillary leak syndrome; patient death; Phase II trial