Biologics
enGene says its investigational gene therapy detalimogene voraplasmid produced a 62% complete response rate at six months in patients with high-risk, BCG-unresponsive non–muscle invasive bladder cancer (NMIBC), according to new preliminary data from the company’s Phase 2 LEGEND trial. The company’s analysis included patients treated
Biologics
FDA has approved Janssen’s Darzalex Faspro (daratumumab and hyaluronidase-fihj, Janssen Biotech) for adults with high-risk smoldering multiple myeloma (SMM), marking the first approved therapy for this early stage of the blood cancer. The decision is based on data from the Phase 3 AQUILA trial (NCT03301220), which compared
Biologics
The 11/5 Medpage Investigative Roundup highlights key findings in a STAT report (subscription required) about chaos at CBER under the leadership of Center director Vinay Prasad. The report indicates that several CBER employees have filed human resources complaints about the director’s management style and practices. It says employees
Biologics
FDA has issued Alvotech a complete response letter on its BLA for AVT05, a biosimilar to Janssen Pharmaceuticals’ Simponi (golimumab), in both prefilled syringe and autoinjector formats. The letter cited deficiencies identified during a July pre-license inspection of Alvotech’s Reykjavik manufacturing facility, according to the company. Alvotech says
Biologics
Gene therapy company uniQure says that FDA indicated during a recent pre-BLA meeting that the agency may no longer view the company’s Phase 1-2 data for its Huntington’s disease (HD) therapy AMT-130, analyzed against an external control, as sufficient to serve as primary evidence to
Biologics
FDA has granted interchangeability designations to Celltrion’s denosumab biosimilars, Stoboclo (denosumab-bmwo) and Osenvelt (denosumab-bmwo), making them fully substitutable at the pharmacy level for Amgen’s reference biologics Prolia and Xgeva, respectively. The action marks the first time interchangeable status has been awarded to biosimilar versions of denosumab,
Biologics
Johnson & Johnson has submitted a supplemental BLA seeking to expand the use of its biologic therapy Stelara (ustekinumab) to treat children aged two years and older with moderately to severely active ulcerative colitis (UC). The filing is based on data from the Phase 3 UNIFI Jr study, which evaluated
Biologics
FDA has issued a clinical hold on Intellia Therapeutics’ MAGNITUDE and MAGNITUDE-2 Phase 3 trials for nexiguran ziclumeran (nex-z) after the company disclosed Grade 4 liver transaminases and increased total bilirubin in a patient who was dosed with nex-z in the MAGNITUDE trial. The company earlier this
Biologics
FDA Office of Therapeutic Biologics and Biosimilars (OTBB) director Sarah Yim said the agency is focused on making biosimilar development faster and more predictable — without undermining scientific rigor. Referencing recent analyses on rising development costs, Yim warned: “If biosimilar development becomes too costly, that’s a problem,” and stressed a
Biologics
FDA has accepted for priority review a BioMarin Pharmaceutical supplemental BLA for Palynziq (pegvaliase-pqpz) to expand the therapy’s use for treating adolescents aged 12–17 with phenylketonuria (PKU). The agency has set a user fee review action target date of 2/28/2026. The submission is supported by
Biologics
FDA is proposing a major shift in how biosimilars are evaluated, signaling that many products may no longer need traditional comparative clinical efficacy studies (CES) to demonstrate biosimilarity. A new draft guidance, Scientific Considerations in Demonstrating Biosimilarity to a Reference Product: Updated Recommendations for Assessing the Need for Comparative Efficacy
Biologics
FDA has issued Regeneron a complete response letter for its pre-filled Eylea HD syringe supplemental BLA due to unresolved inspection findings at third-party manufacturer Catalent’s troubled Indiana facility. Regeneron now says it plans to submit by 1/2026 an application to include a new pre-filled syringe
Biologics
FDA has accepted for priority review an Astellas supplemental BLA for Padcev (enfortumab vedotin-ejfv) in combination with Merck’s Keytruda (pembrolizumab) for treating cisplatin-ineligible patients with muscle-invasive bladder cancer (MIBC). The submission seeks approval for the regimen as both a neoadjuvant treatment before surgery and an adjuvant
Biologics
Moderna’s bid to bring the first vaccine for congenital cytomegalovirus (CMV) to market has ended in disappointment. The company says its investigational CMV vaccine, mRNA-1647, failed to meet the primary endpoint in a large Phase 3 trial, prompting the company to discontinue development for use in preventing congenital
Biologics
FDA is hosting a one-day public workshop 10/23 to explore new approaches for approving patch test allergens used in diagnosing allergic contact dermatitis (ACD) — a common inflammatory skin condition affecting millions of Americans. The virtual workshop is intended to bring together regulators, clinicians, academic researchers, and industry stakeholders
Biologics
Attorneys at Hyman, Phelps & McNamara say they are encouraged by an FDA draft guidance outlining a roadmap for postapproval monitoring of cell and gene therapies (CGT). Writing in the law firm’s online FDALawBlog, the attorneys acknowledge that FDA and industry are in the early stage of commercial CGT
Biologics
FDA has accepted for review a Rocket Pharmaceuticals BLA resubmission for Kresladi (marnetegragene autotemcel; marne-cel), a lentiviral vector–based gene therapy for severe Leukocyte Adhesion Deficiency-I (LAD-I). The agency has set a 3/28/2026 user fee review action target date. The resubmission addresses issues raised in
Biologics
Regeneron Pharmaceuticals says new clinical data show its investigational gene therapy DB-OTO has restored meaningful hearing in nearly all children treated for genetic hearing loss caused by OTOF gene variants. The company plans to submit a BLA later this year after discussing its options with the agency. The data
Biologics
FDA has approved Celltrion’s Eydenzelt (aflibercept-boav), a biosimilar referencing Regeneron and Bayer’s blockbuster Eylea (aflibercept) for treating several major retinal diseases, including neovascular (wet) age-related macular degeneration, macular edema following retinal vein occlusion, diabetic macular edema, and diabetic retinopathy. FDA’s decision was based on analytical,
Biologics
CBER director Vinay Prasad has made recent leadership changes at the Center, appointing several new acting deputy directors and reassigning key staff within the Center. Katherine Szarama has moved from HHS’ Advanced Research Projects Agency for Health to be acting CBER deputy director, according to a MedPage Today report. She
Biologics
A 5/14-5/20/2024 FDA inspection at New Life Medical Services in Lutz, FL, found that the company sells human products derived from umbilical cord, amniotic fluid, and exosomes for allogeneic use. A 9/24 Warning Letter says the firm’s products are legally considered to be unapproved
Biologics
Complement Therapeutics has received the green light from FDA to begin human testing of its lead gene therapy, CTx001, in patients with geographic atrophy secondary to age-related macular degeneration. The IND approval paves the way for the company to launch Opti-GAIN, a Phase 1-3 trial evaluating the