Biologics
Three Ropes & Gray attorneys say questions remain about the so-called “plausible mechanism” pathway for approving certain bespoke, personalized therapies described by FDA commissioner Marty Makary and CBER director Vinay Prasad in a recent New England Journal of Medicine article. Writing in an online post, the attorneys outline the
Biologics
FDA has approved Novartis’ Itvisma (onasemnogene abeparvovec-brve), a new formulation of a gene therapy for spinal muscular atrophy (SMA), allowing the treatment for adults and children two years of age and older with confirmed mutations in the SMN1 gene. Itvisma contains the same active ingredient as Zolgensma, the intravenous
FDA Policy/General
FDA has issued a draft guidance, Cross-Center Master Files: Where to Submit, with recommendations for master file holders about to which Center a master file is to be submitted (1) that is referenced in and intended to support more than one regulatory submission for which the lead Center for
Biologics
FDA is investigating the death of a pediatric patient with congenital thrombotic thrombocytopenic purpura (cTTP) who developed neutralizing antibodies to the enzyme ADAMTS13 after treatment with Takeda’s Adzynma, a recombinant ADAMTS13 therapy approved in 2023 for the rare, life-threatening blood disorder. In a new safety communication, the agency
Biologics
Based on information from two anonymous senior administration officials, Politico is reporting that FDA commissioner Marty Makary and HHS secretary Robert F. Kennedy, Jr., are embroiled in a feud over vaccine policy and agency personnel. The article provides additional insight into earlier reporting on Kennedy having discussed plans to scale
Biologics
Zymeworks says its HER2-targeted bispecific antibody Ziihera (zanidatamab-hrii) significantly improved outcomes for patients with HER2-positive gastroesophageal adenocarcinoma (GEA) in a large Phase 3 study. Topline results from the HERIZON-GEA-01 trial showed that Ziihera combined with chemotherapy, with or without the PD-1 inhibitor Tevimbra (tislelizumab)
Biologics
FDA has accepted for review an Outlook Therapeutics BLA resubmission for ONS-5010 (bevacizumab-vikg), an investigational ophthalmic formulation of bevacizumab for treating wet age-related macular degeneration (wet AMD). The agency reportedly told the company that the submission is a complete, Class 1 response to an 8/27 complete
Biologics
FDA has approved Shanghai Henlius Biologics’ Poherdy (pertuzumab-dpzb), the first interchangeable biosimilar to Perjeta (pertuzumab), Genentech’s HER2-targeted therapy for breast cancer. Poherdy is described as a HER2/neu receptor antagonist indicated for use in combination with trastuzumab and chemotherapy in several settings, including: * Metastatic breast cancer (MBC)
Biologics
A biotechnology company specializing in adult-derived stem cells is urging FDA to loosen federal regulation of some regenerative therapies, arguing that products meeting strict manufacturing and facility standards should be governed under the agency’s lower-risk framework for human cell and tissue products. A just-filed citizen petition
Biologics
enGene says its investigational gene therapy detalimogene voraplasmid produced a 62% complete response rate at six months in patients with high-risk, BCG-unresponsive non–muscle invasive bladder cancer (NMIBC), according to new preliminary data from the company’s Phase 2 LEGEND trial. The company’s analysis included patients treated
Biologics
FDA has approved Janssen’s Darzalex Faspro (daratumumab and hyaluronidase-fihj, Janssen Biotech) for adults with high-risk smoldering multiple myeloma (SMM), marking the first approved therapy for this early stage of the blood cancer. The decision is based on data from the Phase 3 AQUILA trial (NCT03301220), which compared
Biologics
The 11/5 Medpage Investigative Roundup highlights key findings in a STAT report (subscription required) about chaos at CBER under the leadership of Center director Vinay Prasad. The report indicates that several CBER employees have filed human resources complaints about the director’s management style and practices. It says employees
Biologics
FDA has issued Alvotech a complete response letter on its BLA for AVT05, a biosimilar to Janssen Pharmaceuticals’ Simponi (golimumab), in both prefilled syringe and autoinjector formats. The letter cited deficiencies identified during a July pre-license inspection of Alvotech’s Reykjavik manufacturing facility, according to the company. Alvotech says
Biologics
Gene therapy company uniQure says that FDA indicated during a recent pre-BLA meeting that the agency may no longer view the company’s Phase 1-2 data for its Huntington’s disease (HD) therapy AMT-130, analyzed against an external control, as sufficient to serve as primary evidence to
Biologics
FDA has granted interchangeability designations to Celltrion’s denosumab biosimilars, Stoboclo (denosumab-bmwo) and Osenvelt (denosumab-bmwo), making them fully substitutable at the pharmacy level for Amgen’s reference biologics Prolia and Xgeva, respectively. The action marks the first time interchangeable status has been awarded to biosimilar versions of denosumab,
Biologics
Johnson & Johnson has submitted a supplemental BLA seeking to expand the use of its biologic therapy Stelara (ustekinumab) to treat children aged two years and older with moderately to severely active ulcerative colitis (UC). The filing is based on data from the Phase 3 UNIFI Jr study, which evaluated
Biologics
FDA has issued a clinical hold on Intellia Therapeutics’ MAGNITUDE and MAGNITUDE-2 Phase 3 trials for nexiguran ziclumeran (nex-z) after the company disclosed Grade 4 liver transaminases and increased total bilirubin in a patient who was dosed with nex-z in the MAGNITUDE trial. The company earlier this
Biologics
FDA Office of Therapeutic Biologics and Biosimilars (OTBB) director Sarah Yim said the agency is focused on making biosimilar development faster and more predictable — without undermining scientific rigor. Referencing recent analyses on rising development costs, Yim warned: “If biosimilar development becomes too costly, that’s a problem,” and stressed a
Biologics
FDA has accepted for priority review a BioMarin Pharmaceutical supplemental BLA for Palynziq (pegvaliase-pqpz) to expand the therapy’s use for treating adolescents aged 12–17 with phenylketonuria (PKU). The agency has set a user fee review action target date of 2/28/2026. The submission is supported by
Biologics
FDA is proposing a major shift in how biosimilars are evaluated, signaling that many products may no longer need traditional comparative clinical efficacy studies (CES) to demonstrate biosimilarity. A new draft guidance, Scientific Considerations in Demonstrating Biosimilarity to a Reference Product: Updated Recommendations for Assessing the Need for Comparative Efficacy
Biologics
FDA has issued Regeneron a complete response letter for its pre-filled Eylea HD syringe supplemental BLA due to unresolved inspection findings at third-party manufacturer Catalent’s troubled Indiana facility. Regeneron now says it plans to submit by 1/2026 an application to include a new pre-filled syringe
Biologics
FDA has accepted for priority review an Astellas supplemental BLA for Padcev (enfortumab vedotin-ejfv) in combination with Merck’s Keytruda (pembrolizumab) for treating cisplatin-ineligible patients with muscle-invasive bladder cancer (MIBC). The submission seeks approval for the regimen as both a neoadjuvant treatment before surgery and an adjuvant