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
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[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
Biologics
FDA has extended by three months its review of a GSK BLA for Blenrep (belantamab mafodotin) combinations for treating patients with relapsed or refractory multiple myeloma who have received at least one prior line of therapy. The new user fee review action target date is 10/23, which is intended
Biologics
An 8/23-8/31/2023 FDA inspection at the O3UV facility in Grand Ledge, MI, documented multiple violations in the firm’s illegal manufacturing of autohemotherapy devices. A 7/7 Warning Letter says the firm’s products are adulterated because the company does not have an approved PMA or
Biologics
FDA has rejected a Replimune priority-reviewed BLA for its melanoma therapy RP1 (vusolimogene oderparepvec). In a just-issued complete response letter, the agency said it could not approve RP1 in combination with nivolumab due to concerns about the clinical trial data. According to Replimune, FDA found the pivotal IGNYTE
Biologics
FDA has won its regulatory battle (see earlier story) against gene therapy maker Sarepta Therapeutics. Late last evening (7/21) the company announced that it will agree to FDA’s request to pause all shipments of Elevidys (delandistrogene moxeparvovec) for Duchenne muscular dystrophy in the U.S. “This proactive step
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Laura Loomer, a far-right influencer and conspiracy theorist, has it out for CBER director Vinay Prasad. Loomer, who often has president Trump’s ear and influenced the ouster of a number of “disloyal” Trump officials and aides, now has her sights on Prasad, who she says should be removed
Biologics
An HHS official tells FDA Webview the agency is taking a "hard look" at pulling from the market Sarepta Therapeutics’ Elevidys (delandistrogene moxeparvovec-rokl), an adeno-associated virus vector-based gene therapy for treating Duchenne muscular dystrophy patients. The move is in response to two reports of fatal
Biologics
CBER director Vinay Prasad has again overridden medical reviewers to limit a Covid vaccine’s use, according to a just-posted “decisional memo.” The latest incident involves the agency’s 7/9 approval of Moderna’s sBLA for Spikevax, the company’s Covid-19 vaccine, in children aged six months
Biologics
FDA is currently conducting benefit-risk modeling studies based on the evolving risk of Covid-19 severe outcomes and the long-term cardiac damage from vaccine-associated myocarditis, agency leaders say in a new JAMA article that outlines their rationale in ordering recent safety labeling change to mRNA Covid vaccines.