Biologics
FDA has approved Amgen’s Uplizna (inebilizumab-cdon) for adults with generalized myasthenia gravis (gMG) who test positive for anti-acetylcholine receptor (AChR) or anti-MuSK antibodies, giving patients a new twice-yearly biologic option aimed at sustained disease control. Uplizna, already approved for treating neuromyelitis optica spectrum disorder (NMOSD)
Biologics
FDA has accepted for priority review a Bristol Myers Squibb supplemental BLA for Opdivo (nivolumab) and its use in combination with AVD chemotherapy for adults and adolescents with previously untreated Stage III or IV classical Hodgkin lymphoma. The agency set a user fee review action date of 4/8/2026.
Biologics
A 6/2-6/6 FDA inspection at Lux Therapeutics, doing business as Ponya Therapeutics, in Brookhaven, GA, documented significant violations of the regulations for human cells, tissues, and cellular and tissue-based products (HCT/P). An 11/25 Warning Letter says the specific violations were: * failure to validate and
Biologics
Children’s Health Defense (CHD), which was co-founded by now-HHS secretary Robert F. Kennedy, Jr., is petitioning FDA to take several actions against the mRNA Covid-19 vaccines produced by Moderna and Pfizer/BioNTech. The petition specifically asks that the agency: * revoke all BLAs for every mRNA Covid-
Biologics
FDA is signaling a major policy shift for cancer CAR T-cell therapies, moving away from reliance on single-arm studies toward randomized controlled trials (RCTs) that demonstrate superiority over existing treatments. Writing in a new JAMA Perspective piece, CBER director Vinay Prasad and other Center officials say that many
Biologics
The FDA has opened a broad investigation into reports of deaths in adults that may be linked to Covid-19 vaccines, expanding a review that previously appeared focused only on pediatric cases. The agency says multiple divisions are now examining deaths “potentially related to Covid vaccines” across all age groups,
Biologics
A leading biosimilars scholar has filed an extensive citizen petition urging FDA to dramatically streamline and modernize its biosimilar approval requirements, arguing that current regulations impose unnecessary clinical testing, inflate development costs, and delay access to lower-cost biologic products. The petition, submitted by Sarfaraz K. Niazi, an adjunct professor
Biologics
FDA has approved Gamida Cell’s Omisirge, the first cellular therapy authorized to treat patients with severe aplastic anemia (SAA), a rare and life-threatening bone marrow disorder. The action expands the use of Omisirge to adults and children as young as six who lack a compatible stem cell donor
Biologics
CDC’s Advisory Committee on Immunization Practices (ACIP) has voted 8 to 3 to discontinue the longstanding policy that all newborns receive the Hepatitis B vaccine within 24 hours of birth — a recommendation in place since 1991. Under the new CDC guidance, only infants born to mothers with a positive
Biologics
CBER director Vinay Prasad addressed a 12/4 closed-door investor conference in New York and defended his leadership and attributed criticism to "misleading media narratives," according to a report by STAT News. His appearance comes the day after 12 former FDA commissioners issued an extraordinary public warning,
Biologics
CBER says that a common deficiency that is driving a significant share of BLA-related complete response letters (CRLs) is manufacturing facility readiness, and companies are routinely misjudging when their facilities are actually prepared for an FDA inspection. In remarks delivered to the Food and Drug Law Institute’s annual
Biologics
Capricor Therapeutics says Deramiocel, a cell therapy candidate targeting cardiomyopathy associated with Duchenne muscular dystrophy (DMD), achieved statistically significant improvements in both skeletal muscle function and heart performance in the pivotal Phase 3 HOPE-3 trial. Data from the trial will support a response to a complete response letter (CRL)
Biologics
Leading vaccine experts are sharply criticizing a newly revealed FDA memo that claims ten children died “after and because of” receiving Covid-19 vaccines between 2021 and 2024. The memo, written by CBER director Vinay Prasad and obtained by several media outlets, states that the deaths were drawn from reports
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