Biologics
Pfizer and Valneva say they will submit a BLA for their investigational Lyme disease vaccine candidate based on data demonstrating more than 70% efficacy in a late-stage clinical trial, marking a significant step toward what could become the first approved human vaccine for the disease. The Phase 3 VALOR
Biologics
CBER director Vinay Prasad’s departure at the end of next month is raising questions across the biotechnology sector about how the agency’s approach to vaccines, gene therapies and rare-disease treatments could change in the months ahead. Prasad has had a tumultuous tenure that lasted less than a
Biologics
FDA has expanded the approved use of GSK’s Arexvy, a respiratory syncytial virus (RSV) vaccine that now includes the in adults aged 18 to 49 who are at increased risk of severe disease. The vaccine was previously authorized for preventing RSV-related lower respiratory tract disease (LRTD) in adults
Biologics
FDA’s Vaccines and Related Biological Products Advisory Committee has voted to recommend that seasonal influenza vaccines for the 2026–2027 U.S. season use a trivalent formulation, targeting two influenza A strains and one influenza B strain. Panel members voted 7–0 in favor of recommended strains for both
Biologics
FDA has cited a U.K.-based clinical research center for failing to promptly report adverse events and for potential falsification of study records during a late-stage clinical trial of a respiratory syncytial virus (RSV) vaccine. In a just-posted untitled letter dated 8/5/2025, FDA said an
Biologics
FDA has released an updated draft guidance outlining new and revised recommendations for companies developing biosimilar products, including clarifications on the use of foreign comparator products in clinical studies and requirements for retaining study samples. The document revises and replaces a 2021 draft guidance titled New and Revised Draft Q&
Biologics
FDA has issued Incyte a complete response letter (CRL) for its supplemental BLA for Zynyz (retifanlimab-dlwr) injection (375mg) for an additional indication for treating adult patients with metastatic non-small cell lung cancer in combination with platinum-based chemotherapy. The sBLA was based on data from the Phase 3
Biologics
CBER director Vinay Prasad will step down at the end of April, according to HHS. His departure comes on the heels of several recent controversial decisions at the Center and news of an internal investigation into complaints from staff. HHS did not provide additional details about the reason for his
Biologics
A senior FDA official sharply criticized UniQure’s investigational gene therapy for Huntington's disease, calling it a “failed product” during a 3/5 conference call with reporters and intensifying doubts about the treatment’s regulatory prospects, according to Reuters. The remarks came days after the company said FDA
Biologics
A just-released complete-response letter is shedding light on FDA’s rejection last month of Regenxbio’s BLA for its gene therapy clemidsogene lanparvovec, citing fundamental concerns about the study population, choice of control, and use of a novel cerebrospinal fluid biomarker as the basis for accelerated approval. The
Biologics
uniQure says FDA does not agree that data from the company’s Phase 1-2 studies — compared against an external control cohort — are sufficient to constitute the primary evidence of effectiveness required for a BLA for its Huntington’s disease gene therapy AMT-130. The agency strongly recommended that a
Biologics
Pfizer CEO Albert Bourla on Monday publicly criticized the leadership of FDA’s biologics center, saying the company “has a problem” with the agency’s top vaccines regulator. Speaking at the TD Cowen healthcare conference, Bourla said CBER director Vinay Prasad was dismissing the recommendations of career agency scientists. “I
Biologics
FDA has accepted for review a Vanda Pharmaceuticals BLA for imsidolimab, a potential new treatment for generalized pustular psoriasis (GPP), a rare and life-threatening autoinflammatory skin disorder. The agency has set a review action target date of 12/12. GPP is characterized by sudden flares of widespread pustules, red
Biologics
After failing to find a buyer, BioMarin has decided to voluntarily withdraw from the market its Roctavian (valoctocogene roxaparvovec-rvox) gene therapy for treating adults with severe hemophilia A without antibodies to adeno-associated virus serotype 5. Since its 2023 approval, Roctavian failed to achieve commercial success due to low
Biologics
Johnson & Johnson has submitted a supplemental BLA seeking approval of Imaavy as a treatment for warm autoimmune hemolytic anemia (wAIHA), a rare and potentially life-threatening blood disorder that currently has no FDA-approved therapies. The drug was approved by FDA 4/2025 for generalized myasthenia gravis in patients
Biologics
FDA has agreed to review Moderna’s seasonal influenza mRNA vaccine after previously refusing to file the BLA (see earlier story), a reversal that follows mounting scrutiny of the agency’s handling of mRNA products. The agency flip-flop followed what media reports describe as an unusual, high-level meeting
Biologics
FDA’s recent refusal to review Moderna’s application for an mRNA-based seasonal influenza vaccine has reignited concerns that vaccine policy — particularly involving mRNA technology — is becoming increasingly entangled with politics, with potentially far-reaching consequences for public health and drug development, according to a Competitive Enterprise Institute blog
Biologics
The Senate HELP Committee has released a report with 15 recommendations for reforming FDA. The report says innovation in medical care and treatment is meaningless unless it creates products that actually help patients. “Discoveries that never leave the lab help no one,” it declares. “FDA has enormous leverage over how
Biologics
CBER is facing mounting scrutiny after its top official, director Vinay Prasad, overruled career staff and refused the review of Moderna’s application for a new mRNA flu vaccine — a move that insiders, industry executives and analysts say reflects broader disruption inside the Center. Now, we are learning that earlier
Biologics
FDA has granted Krystal Biotech a Regenerative Medicine Advanced Therapy (RMAT) designation for KB707, an inhalable gene therapy being investigated for treating advanced or metastatic non-small cell lung cancer (NSCLC). KB707 is described as a redosable herpes simplex type 1 virus-based immunotherapy that is designed to deliver genes
Biologics
FDA has cited AGC Biologics for deficiencies in laboratory data controls and inventory management at its Bothell, WA, drug substance manufacturing site, according to a just-released Form FDA-483. FDA investigators inspected the facility 4/2025 and cited inadequate review of laboratory data associated with in-process and final
Biologics
CBER director Vinay Prasad reportedly overruled the Center’s career reviewers in deciding to issue Moderna a refusal-to-file letter (see earlier story) for its BLA for an mRNA influenza vaccine, a move that is highly unusual because these procedural decisions are typically handled by review staff, not Center