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
Biologics
FDA has approved two new denosumab biosimilars from Shanghai Henlius Biotech and Organon, ostensibly expanding patient access to treatments for osteoporosis and cancer-related bone conditions. The products, Bildyos (denosumab-nxxp) and Bilprevda (denosumab-nxxp), are said to be highly similar to Amgen’s blockbuster drugs Prolia and Xgeva, respectively.
Biologics
FDA has agreed to accept a biosimilar application that does not include clinical efficacy studies (CESs) for a monoclonal antibody, a step that could dramatically change how these drugs are developed and approved. The upcoming submission from University of Illinois adjunct professor and founder of several biopharmaceutical ventures Sarfaraz K.
Biologics
FDA is removing members of its Vaccines and Related Biological Products Advisory Committee (VRBPAC) and Children’s Hospital of Philadelphia’s Vaccine Education Center director Paul Offit reportedly is the first to go. Offit has often been a critic of HHS secretary Robert F. Kennedy Jr. and his health policies.
Biologics
FDA has approved an Eisai/Biogen at-home injection option for Alzheimer’s treatment Leqembi (lecanemab), expanding access to patients in the early stages of the disease. The approval covers Leqembi Iqlik, a once-weekly subcutaneous autoinjector of lecanemab for maintenance dosing. Patients who have completed the 18-month initiation
Biologics
In the wake of CDC director Susan Monarez’s ouster, Senator Bill Cassidy (R-LA), who is chair of the Senate Health, Education, Labor, and Pensions (HELP) Committee, is calling on HHS to postpone a just-announced Advisory Committee on Immunization Practices (ACIP) meeting that is scheduled to review the
Biologics
An FDA review of the Web site maintained by Innate Healthcare Institute in Scottsdale, AZ, found that the company markets “umbilical cord mesenchymal stem cells” for allogenic use in the diagnosis, cure, mitigation, treatment, or prevention of disease or conditions in humans and/or are intended to affect the structure
Biologics
FDA has approved supplemental BLAs from Moderna and Pfizer/BioNTech for their mRNA Covid-19 vaccines with 2025-2026 formulations that target the LP.8.1 variant of SARS-CoV-2, which is the dominant circulating strain of the disease. The agency also approved Novavax’s Nuvaxovid Covid vaccine for
Biologics
FDA has extended by three months its review of an Axogen BLA for Avance Nerve Graft after the agency determined that information submitted by the company in response to an agency request was deemed a major amendment. The information submitted included substantial new manufacturing and facility data not previously reviewed
Biologics
Inovio Pharmaceuticals says it has started a rolling BLA submission for INO-3107, an experimental DNA medicine for recurrent respiratory papillomatosis (RRP), with the goal of securing FDA approval by the end of the year. The company expects to complete the filing in the coming months and plans to request
Biologics
Argenx says it will file a supplemental BLA for Vyvgart (efgartigimod alfa-fcab) to expand its label to include adults with acetylcholine receptor antibody (AChR-Ab) seronegative generalized myasthenia gravis (gMG) after reporting positive topline results from a Phase 3 trial of patients in this patient population. The company says
Biologics
FDA has accepted for priority review a Gamida Cell supplemental BLA for omidubicel and its use in severe aplastic anemia, a rare and often life-threatening condition in which the bone marrow fails to produce sufficient blood cells. Hematopoietic stem cell transplants can be curative, but many patients lack a
Biologics
FDA is announcing a 10/23 public workshop to discuss the regulatory status of patch tests used in diagnosing allergic contact dermatitis (ACD). The CBER-directed one-day virtual event is intended to gain stakeholder input on current approval requirements and potential alternative approaches. Patch testing is the only validated
Biologics
FDA has lifted a clinical hold on Rocket Pharmaceuticals’ pivotal Phase 2 trial of RP-A501, an investigational gene therapy for Danon disease, allowing the study to resume less than three months after being paused. The hold followed the death of a participant who experienced serious complications during the study.