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Rocket Pharmaceuticals has voluntarily withdrawn its BLA for RP-L102 (mozafancogene autotemcel), an investigational gene therapy for treating Fanconi Anemia.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
Biologics
An 11/11-11/19/2024 FDA inspection at the Janssen Vaccines drug manufacturing facility in Incheon, South Korea, found significant violations of current good manufacturing practice (CGMP) regulations for finished drugs. A just-released 7/18 Warning Letter says the specific violations were: * failure to thoroughly investigate any unexplained
Biologics
FDA has approved a Krystal Biotech supplemental BLA for a label expansion on its gene therapy Vyjuvek (beremagene geperpavec-svdt), adding patients with dystrophic epidermolysis bullosa (DEB) from birth. The labeling now allows DEB patients and their caregivers to apply the topical gene therapy at home, rather than requiring administration
Biologics
A CBER untitled letter to AstraZeneca cites the firm for running a misleading television direct-to-consumer advertisement for its nasal spray flu vaccine FluMist, warning the company that the commercial misbrands the product and violates federal law. In the 9/9 letter from CBER director Vinay Prasad, the agency
Biologics
CBER is hosting a 10/22 virtual town hall focused on gene therapy manufacturing requirements for BLAs and post-licensure changes. The Office of Therapeutic Products event brings together experts from the Office of Gene Therapy and the Division of Manufacturing and Product Quality. FDA officials will address questions related
Biologics
CBER director Vinay Prasad has been reassigned his previous additional duties as FDA’s chief medical and scientific officer, a combination role for commissioner Marty Makary’s closest advisor. In this capacity, “he will serve as a trusted advisor to the FDA commissioner and other senior officials on cross-cutting
Biologics
The latest Make America Healthy Again (MAHA) Commission report, “Make Our Children Healthy Again: Assessment,” says FDA has a role to play in working with the National Institutes of Health to “build systems for real-world safety monitoring of pediatric drugs and create programs to independently replicate findings from industry-
Biologics
FDA has issued two final guidances related to biosimilar development. The final guidance Development of Therapeutic Protein Biosimilars: Comparative Analytical Assessment and Other Quality-Related Considerations provides recommendations on the design and evaluation of comparative analytical studies intended to support a demonstration that a proposed therapeutic protein product is biosimilar
Biologics
CBER director Vinay Prasad demanded that YouTube videos in which he criticized the Covid-19 vaccines be removed. The videos were part of some 350 clips of doctors and commentators that were posted on the YouTube channel maintained by Jonathan Howard, a New York City neurologist and psychiatrist, according to
Biologics
Memos posted on FDA’s Web site from CBER director Vinay Prasad suggest the agency is raising the bar on future Covid-19 vaccination approvals. For example, as part of the agency’s approval last week of the upcoming Covid-19 seasonal vaccines, Prasad wrote in one memo that CBER