FDA

IQVIA’s Doug Long offers a lay of the land, including trends and developing issues that are shaping the current market.

Approval of penpulimab-kcqx marks the company’s US regulatory debut and introduces a new immunotherapy option for advanced nasopharyngeal carcinoma.

The submission is backed by clinical data from over 2,100 patients, including two pivotal Phase III trials in patients treated with trenibotulinumtoxinE for moderate to severe glabellar lines.

The simplified requirements for Camzyos are expected to ease prior contraindications, thereby simplifying treatment and expanding eligibility in patients with symptomatic New York Heart Association Class II-III obstructive hypertrophic cardiomyopathy.

Dupixent is the first new targeted therapy to receive FDA-approval for chronic spontaneous urticaria in adults and adolescents over 12 years of age in over a decade.

Regulatory action was based on data from the Phase III QUASAR trial, which demonstrated that Eylea HD dosed every eight weeks achieved non-inferior visual acuity outcomes compared to Eylea in patients with macular edema following retinal vein occlusion.

Approval of Opdivo plus Yervoy combination was based on results from the Phase III CheckMate-9DW trial, which demonstrated significantly improved overall survival and overall response rate in patients with unresectable or metastatic hepatocellular carcinoma.

Full approval of Vitrakvi was based on results from three clinical trials in patients with unresectable or metastatic NTRK fusion-positive solid tumors.

Approval of the Opdivo plus Yervoy combination regimen was based on results from the Phase III CheckMate-8HW trial, which was the largest immunotherapy study in patients with previously untreated, unresectable, or metastatic microsatellite instability-high or mismatch repair deficient colorectal cancer.

Brimochol PF, a combination of brimonidine and carbachol, was found to enhance depth of focus and visual acuity in clinical trials among patients with presbyopia.

Results from Phase II studies demonstrated that rilzabrutinib showed clinically meaningful outcomes in patients with warm autoimmune hemolytic anemia and IgG4-related disease.

Results from the Phase III SPACE trial show that Ajovy significantly reduced monthly migraines in pediatric patients between six and 17 years of age.

BIIB080 marks the first antisense oligonucleotide targeting tau to enter clinical development for the treatment of Alzheimer disease.

Qfitlia is the first FDA-approved antithrombin-lowering therapy indicated for routine prophylaxis to prevent or reduce bleeding episodes in patients over 12 years of age with hemophilia A or B.

Approval of Imfinzi marks the first and only perioperative immunotherapy indicated for muscle-invasive bladder cancer.

As part of the Fast Track designation, Sanofi is set to launch a Phase I/II trial to evaluate the immunogenicity and safety of its novel mRNA vaccine in preventing chlamydia.

Approval of Vykat XR marks the first treatment indicated for hyperphagia in patients with Prader-Willi syndrome.

Blujepa marks the first new oral antibiotic to gain FDA approval for uncomplicated urinary tract infections in nearly 30 years.

Fanhalta marks the first and only treatment for C3 glomerulopathy to gain FDA approval.

Dr. Dina Radenkovic, CEO of Gameto, discusses the key considerations for bringing iPSC-based fertility treatments to market from a regulatory and manufacturing perspective.

Dr. Dina Radenkovic, CEO of Gameto, discusses the use of iPSCs in fertility treatments, the most significant scientific hurdles they've encountered in developing and scaling these therapies (particularly in relation to ovarian aging), and how they're addressing them.

Fred Aslan, MD, CEO, Artiva Biotherapeutics, explains what the FDA Fast Track designation for AlloNK in autoimmune diseases means for the acceleration of its development and potential approval of the therapy.

What biopharma companies need to know about this evolving regulatory pathway.

Omlyclo, a biosimilar to Xolair, is indicated for the treatment of moderate to severe persistent asthma, chronic rhinosinusitis with nasal polyps, immunoglobulin E-mediated food allergies, and chronic spontaneous urticaria.

Jonathon Whitton, AuD, PhD, VP, auditory global program head, Regeneron, discusses how key regulatory designations, such as the FDA's Regenerative Medicine Advanced Therapy designation, facilitate accelerated development and commercialization of DB-OTO through increased collaboration with regulators.