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
Biologics
FDA has issued Moderna a refusal-to-file letter on its BLA for an investigational seasonal influenza vaccine mRNA-1010, despite the company’s use of a Priority Review Voucher intended to speed the process. The letter, signed by CBER director Vinay Prasad, cited the company’s choice of comparator
Biologics
FDA has cited Lonza Portsmouth for significant manufacturing and quality control deficiencies at its New Hampshire cell therapy facility, raising concerns about aseptic processing, environmental controls, documentation practices, and deviation management related to the production of Vertex Pharmaceuticals and CRISPR Therapeutics’ gene-edited therapy Casgevy. FDA investigators conducted the inspection
Biologics
The Biotechnology Innovation Organization (BIO) has submitted detailed comments to FDA recommending broader application of its draft guidance on “Monoclonal Antibodies (mAbs): Streamlined Nonclinical Safety Studies.” The comments highlight ways the agency could reduce animal testing while maintaining rigorous safety standards. BIO praised FDA for emphasizing the 3R principles — replace,
Biologics
AstraZeneca and Daiichi Sankyo’s antibody–drug conjugate Datroway (datopotamab deruxtecan) has been granted an FDA priority review as a potential first-line treatment for patients with unresectable or metastatic triple-negative breast cancer (TNBC) who are not candidates for immunotherapy. Triple-negative breast cancer accounts for about 15% of
Biologics
FDA is signaling a supportive but cautious regulatory posture toward the development of chimeric antigen receptor (CAR) T-cell therapies for autoimmune and rheumatic diseases, according to a perspective article published in the Annals of Internal Medicine by agency officials. CAR-T cell therapies — genetically engineered T cells programmed to
Biologics
Robert Malone, the vice chair of the CDC Advisory Committee on Immunization Practices (ACIP) and a close ally of HHS secretary Robert F. Kennedy Jr., has publicly accused FDA commissioner Marty Makary of blocking the effort to remove mRNA Covid vaccines from the market. Axios reports that when Malone responded
Biologics
FDA has accepted for review a Summit Therapeutics BLA seeking approval for ivonescimab in combination with chemotherapy in patients with epidermal growth factor receptor (EGFR)-mutated locally advanced or metastatic non-squamous non-small cell lung cancer (NSCLC) post-tyrosine kinase inhibitor (TKI) therapy. The agency has set a user
Biologics
FDA has placed clinical holds on two Regenxbio investigational gene therapy programs for ultra-rare pediatric diseases following the identification of a central nervous system tumor in a single patient treated with one of its candidates. A hold was placed on RGX-111, an experimental gene therapy for mucopolysaccharidosis Type
Biologics
FDA has approved a Scancell Holdings IND to begin a registrational Phase 3 trial of iSCIB1+, the company’s lead ImmunoBody immunotherapy, in patients with advanced melanoma. The agency agreed that progression-free survival could act as the surrogate primary endpoint, the company says. The late-stage trial is intended