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Innovation Organization (BIO) has submitted detailed comments to FDA recommending broader application of its draft guidance on “Monoclonal Antibodies (mAbs): Streamlined Nonclinical Safety Studies.”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
Biologics
Sarepta Therapeutics is announcing that its gene therapy Elevidys significantly slowed disease progression over three years in ambulatory patients with Duchenne muscular dystrophy, according to topline results from the Phase 3 EMBARK trial. The company says patients treated with Elevidys maintained higher levels of motor function than untreated controls across
Biologics
A coordinated advocacy campaign by the Huntington’s disease community helped set the stage for a high-priority meeting between FDA and uniQure to discuss the regulatory path forward for the company’s experimental gene therapy AMT-130. UniQure said earlier this month that it had secured a Type A
Biologics
Moderna and Merck say that updated five-year follow-up data show that their personalized mRNA cancer vaccine candidate, intismeran autogene, significantly reduced the risk of melanoma recurrence or death when used in combination with Merck’s immunotherapy Keytruda (pembrolizumab), compared with Keytruda alone, according to the companies. In a
Biologics
FDA has asked Capricor Therapeutics for a full clinical study report from the company’s Phase 3 HOPE-3 trial as part of its ongoing BLA review for Deramiocel, a proposed cell therapy for Duchenne muscular dystrophy. The request follows the agency’s review of topline HOPE-3 data that
Biologics
Valneva says it has voluntarily withdrawn its BLA for Ixchiq, the company’s chikungunya vaccine, based on FDA safety-related restrictions on the product. In 8/2025, CBER suspended Valneva’s BLA due to serious safety concerns that the vaccine could be causing chikungunya-like illness in vaccine recipients. The
Biologics
Scholar Rock says it is planning to resubmit this year its BLA seeking approval of apitegromab, a first-in-class muscle-targeted therapy for spinal muscular atrophy (SMA). In 9/2025, FDA issued a complete response letter that cited observations made during an FDA inspection of Catalent Indiana, a third-
Biologics
FDA has accepted for priority review an Argenx supplemental BLA seeking to expand the use of its myasthenia gravis drug Vyvgart (efgartigimod alfa-fcab) to adults with acetylcholine receptor antibody–negative generalized myasthenia gravis. The agency set a user fee review action target date of 5/10. If approved, the
Biologics
FDA has accepted for review a PharmaEssentia supplemental BLA seeking to expand the label of its interferon therapy Besremi (ropeginterferon alfa-2b-njft) to include adults with essential thrombocythemia. The agency has set a user fee review action target date of 8/30. If approved, the expanded indication would allow
Biologics
CBER has released its calendar year 2026 guidance agenda showing it plans to produce 35 draft and final guidances in four categories — Blood and Blood Components (five guidances), Therapeutic Products (19 guidances), Other (two guidances), and Vaccines and Allergenics (nine guidances). In the Vaccines and Allergenics category, CBER says it
Biologics
FDA has issued Pierre Fabre Pharmaceuticals a second Complete Response Letter rejecting its BLA for Atara Biotherapeutics’ investigational cell therapy for a rare and often fatal post-transplant cancer, citing the need for additional clinical evidence. The rejection appears to be one of the first rejections under a new CBER
Biologics
FDA says it is formalizing and more broadly communicating a flexible regulatory approach to chemistry, manufacturing and controls for cell and gene therapies, a move the agency says is intended to speed development of innovative treatments while maintaining product quality standards. An FDA notice says details are being shared on
Biologics
With all the staffing turmoil FDA went through in 2025, drug and biologic approvals dropped below totals in the previous two years. A Fierce Pharma analysis finds that there were 46 novel drug approvals in 2025, compared to 55 in 2023 and 50 in 2024. In addition, there were 18
Biologics
Mainstream media stories at the end of 2025 focused on the ongoing controversy over vaccines brought about by recommendations from CBER director Vinay Prasad and newly-appointed CDER director (the fifth director in 2025) Tracy Beth Hoeg. The Guardian reports that Hoeg’s appointment “could signify a closer partnership between