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Attorneys at Hyman, Phelps & McNamara say they are encouraged by an FDA draft guidance outlining a roadmap for postapproval monitoring of cell and gene therapies (CGT).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
Biologics
Lexeo Therapeutics says it has received FDA guidance on a potential accelerated approval pathway for its investigational gene therapy LX2006. The agency says FDA indicated it is open to allowing pooled data from ongoing Phase 1-2 trials and a future pivotal study to support a BLA for accelerated approval,
Biologics
FDA has posted a five-item FDA Form-483 after inspecting Covid-19 vaccine maker Serum Institute of India’s Maharashtra manufacturing facility 7/2024. The agency cited the firm over Covid-19 syringe-filling line deficiencies. It also said the company had insufficient data for a validation study related
Biologics
Rocket Pharmaceuticals has voluntarily withdrawn its BLA for RP-L102 (mozafancogene autotemcel), an investigational gene therapy for treating Fanconi Anemia. The move follows the company’s previously announced corporate prioritization to focus its resources on programs with the clearest regulatory and commercial pathways. The company notes its decision “does not
Biologics
FDA has accepted for review a Daiichi Sankyo and AstraZeneca supplemental BLA for Enhertu (fam-trastuzumab deruxtecan-nxki) use followed by paclitaxel, trastuzumab, and pertuzumab (THP) as a neoadjuvant treatment for patients with high-risk, early-stage HER2-positive breast cancer. The agency has set a user fee review action
Biologics
AbbVie 9/30 submitted a BLA seeking approval for its investigational antibody-drug conjugate pivekimab sunirine (PVEK) to treat blastic plasmacytoid dendritic cell neoplasm (BPDCN), a rare and aggressive blood cancer. The submission is based on data from AbbVie’s global Phase 1/2 CADENZA trial, which evaluated the safety
Biologics
Novo Nordisk has resubmitted a BLA for Awiqli (insulin icodec), a once-weekly basal insulin injection for adults with Type 2 diabetes. If approved, the company says Awiqli would become the first once-weekly basal insulin available in the U.S., offering an alternative to daily injections. The resubmission follows
Biologics
IO Biotech says it has recently been advised by FDA not to submit a BLA for its experimental cancer vaccine Cylembio based on results from a recently completed Phase 3 trial in advanced melanoma. The FDA input came during a pre-BLA meeting at which the agency concluded that data
Biologics
Capricor Therapeutics says it has reached agreement with FDA on the design and role of its HOPE-3 trial as it works to resubmit its BLA for Deramiocel and its use in treating Duchenne muscular dystrophy (DMD). The update follows a Type A meeting with the agency, convened after it
Biologics
CBER has begun to post, more proactively, inspection-related Form FDA-483s under the Freedom of Information Act’s provision on “frequently requested records.” Under the regulation, FDA can release electronic copies of any records that have been previously released if it determines that the records are likely to be
Biologics
FDA has released a draft guidance outlining methods drugmakers should use to collect long-term safety and efficacy data once cell and gene therapy products are approved. The recommendations reflect growing concern over how to monitor treatments that may have lifelong effects but are tested in relatively small patient populations
Biologics
FDA says that despite repeated requests, Cellebration LLC, headquartered in San Juan, Puerto Rico, has failed to provide records sought by the agency since 10/18/2024. A 9/12 Warning Letter says the Cellebration Web site was reviewed by FDA, which found that the company offers cellular products derived
Biologics
FDA has published a draft guidance, Expedited Programs for Regenerative Medicine Therapies for Serious Conditions, with the agency’s recommendations on the expedited development and review of such therapies. The document describes the expedited programs available to sponsors of regenerative medicine therapies for serious conditions, including those products designated as
Biologics
FDA has published a draft guidance, Innovative Designs for Clinical Trials of Cellular and Gene Therapy Products in Small Populations, with recommendations for sponsors who are planning clinical trials of cell and gene therapy (CGT) products intended for use in a disease or condition that affects a small population. The