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
Biologics
Former CBER deputy director Julie Tierney, who also served at one time as FDA chief of staff, has joined Leavitt Partners as a principal in the Washington, DC, office. The firm says that with over 25 years of experience in FDA law and policy, Tierney “helps life sciences companies, patient
Biologics
FDA has published the FY 2025 Quarter 2 update to the CBER/CDER Data Standards Program Action Plan. The document includes quarterly project updates and a statement that reporting is paused for two projects. The data standards program focuses on four goals: improve data standards for regulatory use, data standards
Biologics
A new report from STAT describes drug review turmoil at FDA that led to FDA Oncology Center of Excellence director Richard Pazdur overriding CBER reviewers and management in the surprise rejection last month of Replimune’s priority-reviewed BLA for its melanoma therapy RP1 (vusolimogene oderparepvec). RP1 is a herpes
Biologics
FDA has issued Regeneron a second complete response letter (CRL) on its BLA for odronextamab, a bispecific antibody targeting CD20 and CD3 in relapsed/refractory follicular lymphoma after two or more lines of systemic therapy. In a financial update, the company says the submission was rejected over inspection concerns at
Biologics
During a 7/30 NewsNation interview, FDA commissioner Martin Makary promoted a popular right-wing conspiracy theory by saying that under its former leadership, the National Institutes of Health (NIH) funded the lab that created the Covid-19 virus. The Hill reports that Makary made the comment in response to
Biologics
Based on conversations with four anonymous knowledgeable sources, Politico is reporting that President Donald Trump overruled FDA commissioner Martin Makary and HHS secretary Robert F. Kennedy, Jr., to insist that CBER director Vinay Prasad be removed from FDA. Prasad had come under attack from conservative presidential whisperer Laura Loomer, former
Biologics
[Analysis by Jim Dickinson and David McFarland] As FDA reels and staff morale plummets under anti-science political interference from the Trump Administration, newly appointed CBER director Vinay Prasad has abruptly departed the agency after being attacked by Trump influencer Laura Loomer for expressing purportedly left-wing political views as
Biologics
FDA has granted Calidi Biotherapeutics fast-track status for CLD-201, an allogeneic stem-cell-loaded oncolytic virus being developed to treat soft tissue sarcoma. The therapy uses adipose-derived mesenchymal stem cells loaded with an oncolytic vaccinia virus to deliver therapy directly to tumor sites, according to the company.
Biologics
Johnson & Johnson has submitted a supplemental BLA seeking to update the prescribing information for Tremfya (guselkumab) to include new data demonstrating its ability to inhibit structural joint damage in adults with active psoriatic arthritis (PsA). The submission is based on 24-week results from the Phase 3b APEX placebo
Biologics
FDA is investigating another death of a young boy who received Sarepta Therapeutics’ gene therapy Elevidys for Duchenne muscular dystrophy. The death of the eight-year-old occurred on 6/7. The company recently complied with an FDA request to suspend the product’s distribution as it investigates ongoing safety
Biologics
FDA is backpedaling its enforcement stance against Sarepta Therapeutics and Duchenne muscular dystrophy (DMD)gene therapy Elevidys after determining the latest death (see story) reported after a patient was administered the product was unrelated. “The U.S. Food and Drug Administration is recommending the removal of the voluntary hold for
Biologics
FDA reportedly will require additional safety data from Sarepta Therapeutics before the agency considers supporting the continued use of the company’s Duchenne muscular dystrophy (DMD) gene therapy Elevidys (delandistrogene moxeparvovec-rokl) following three recent patient deaths — two associated with Elevidys and the other associated with an experimental gene therapy
Biologics
HHS secretary Robert F. Kennedy, Jr. has signed off on a new federal directive removing thimerosal, a mercury-based preservative, from all influenza vaccines distributed in the U.S. The action formally adopts a recommendation made by CDC’s Advisory Committee on Immunization Practices (ACIP) during a 6/25-26