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.
Biologics
Experts interviewed for an online Clinical Trials Arena post have differing opinions on the impact of a recent FDA rule barring new clinical trials that involve sending U.S. citizens’ living cells to China and other “hostile” countries for genetic engineering before they are returned to be infused into the
Biologics
FDA has extended until the fourth quarter the review of two Regeneron Pharmaceuticals regulatory submissions for Eylea HD (aflibercept) injection 8 mg regulatory submissions because information submitted to address a recent inspection was deemed a major amendment. One submission was a chemistry, manufacturing and controls prior-approval supplement for the
Biologics
Viking Therapeutics is reporting mixed data from a Phase 2 study for its oral obesity drug VK2735, with patients losing up to 12.2% of body weight over 13 weeks. Despite the encouraging efficacy, shares of the biotech tumbled more than 35% at one point as investors focused on high
Biologics
A resubmitted Omeros BLA for narsoplimab in hematopoietic stem cell transplant-associated thrombotic microangiopathy (TA-TMA) has been hit with a three-month extension after the company provided additional information pursuant to an FDA request. The new user fee review action target date is 12/26. In communicating the extension,
Biologics
FDA has granted Beam Therapeutics a Regenerative Medicine Advanced Therapy designation for BEAM-101, an investigational gene-edited cell treatment for sickle cell disease. The designation follows the agency’s orphan drug status awarded in June for the therapy. Updated clinical data from the BEACON Phase 1/2 clinical trial
Biologics
HHS has revived a task force on safer childhood vaccines, a federal panel created by Congress to improve child vaccine safety, quality, and oversight. The original task force was disbanded in 1998. The new task force is led by NIH director Jay Bhattacharya, and includes representation by senior FDA and
Biologics
The emergency use authorization for Pfizer’s Covid-19 vaccine for children under five may not be renewed by FDA this fall, according to The Guardian. An internal email obtained by The Guardian indicates that Moderna may be asked to step in to fill the gap. If the agency action
Biologics
IO Biotech is reporting that its investigational cancer vaccine Cylembio (imsapepimut and etimupepimut, adjuvanted), combined with Merck’s Keytruda (pembrolizumab), improved progression-free survival (PFS) compared with Keytruda alone in a pivotal Phase 3 trial for first-line treatment of advanced melanoma. While the results were clinically meaningful, according to
Biologics
FDA has mandated significant labeling changes for Bluebird Bio’s Skysona (elivaldogene autotemcel), a gene therapy approved for treating early, active cerebral adrenoleukodystrophy (CALD), after seeing an increase in reports of life-threatening hematologic malignancies among patients who received the treatment. Skysona, a lentiviral autologous hematopoietic stem cell (HSC)-based