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FDA

FDA approvals, CRLs, advisory committees, clinical holds and guidance — every shift in the U.S. regulatory posture.

264 picksRelatedEMA / CHMPNMPA / CDERegulatory

Latest picks

61–80 of 264
Oct 6Tue
  1. U.S. FDA55

    Walmart, Inc. - 736557 - 10/01/2026

    FDA's Center for Drug Evaluation and Research issued a warning letter dated October 1, 2026 to Walmart President and CEO John Furner over three products sold on walmart.com: HClear for him, Herstat GST, and Terragen HG Formula. Purchased by FDA in July 2026 and shipped via Walmart Fulfillment Services, the products are marketed for genital herpes and genital sore symptoms and are unapproved new drugs under section 505(a) of the FD&C Act. FDA notes no OTC drug product is approved for treating any STI and warns that failure to correct the violations may bring seizure or injunction without further notice.

    Why it matters: FDA targets a major retailer for selling unapproved STI products, a direct compliance risk for online drug marketplaces.

  2. U.S. FDA56

    Amazon.com, Inc. - 736006 - 10/01/2026

    FDA's Center for Drug Evaluation and Research sent a warning letter dated October 1, 2026 (reference 736006) to Amazon CEO Andy Jassy, citing nine products sold on amazon.com and fulfilled via Fulfillment by Amazon, including FemiClear, HClear for him, HerpCare Ointment and Spray, and RoyceDerm Herpes Relief Cream, as unapproved new drugs for genital herpes under section 505(a) of the FD&C Act. FDA, which purchased the products in July 2026, notes no OTC drug is approved for treating any STI and warns that reliance may delay care and risk infertility, sepsis and death. Amazon must correct the violations or face regulatory or legal action including seizure and injunction.

    Why it matters: FDA warning letter puts Amazon's marketplace and Fulfillment by Amazon service directly in the regulatory line for unapproved STI drugs.

  3. BioCentury80

    Nobel Prize for optogenetics arrives as first therapy nears FDA decision

    The 2026 Nobel Prize in Physiology or Medicine goes to Karl Deisseroth, Peter Hegemann and Georg Nagel for their discoveries of light-gated ion channels and optogenetics. FDA is expected to decide by mid-2027 on the first optogenetic therapy, a retinal gene therapy. Nanoscope leads direct therapeutic applications, while MapLight uses the technology to guide psychiatric drug discovery.

    Why it matters: Pairs the Nobel Prize for basic optogenetics research with the first retinal gene therapy nearing FDA review, showing the timeline from mechanism discovery to translation.

  5. WeChat48

    HHS Launches SURPASS Program Through ARPA-H to Speed Clinical Trials

    On September 30, the US Department of Health and Human Services announced the launch of SURPASS (Simulation-augmented, Real-time Platform Adaptive Seamless Trials) through ARPA-H, along with three supporting programs. The initiative aims to redesign how clinical trials are run, streamline trial processes and speed products to market. Congress has authorized ARPA-H to pursue breakthrough technologies that conventional research and commercial activity cannot achieve.

    Why it matters: HHS is restructuring how clinical trials are designed and conducted, affecting drug development timelines industry-wide.

  6. U.S. FDA64

    M15 Resource Center

    The FDA has posted a resource center for ICH M15, a guidance on general principles for model-informed drug development (MIDD). The guidance describes MIDD principles, including planning, model evaluation and evidence documentation, sets a harmonized framework for assessing MIDD-derived evidence, and gives recommendations on MIDD-related regulatory interactions, reporting and submission. FDA recommends sponsors use it when preparing meeting requests, background packages and regulatory submissions.

    Why it matters: FDA now expects ICH M15 principles to shape MIDD evidence in submissions and meetings.

  7. U.S. FDA54

    Expedited Investigational New Drug Pilot Program - Application Instructions

    The FDA has issued application instructions for its Expedited IND Pilot Program. Sponsors and prospective Qualified Research Institutions each complete a form, then the sponsor submits both through the FDA ESG Gateway as one package. The application window stays open for 45 days with possible extension, and FDA scientific experts will review submissions and select 8 to 10 Sponsor-QRI pairs. Participating parties must form their business relationship outside FDA, which will only confirm that an official agreement exists.

    Why it matters: FDA sets out how sponsors and QRIs apply for the Expedited IND Pilot Program and how many pairs will be selected.

Oct 5Mon
  1. Pharmaceutical Technology83

    Scientists claim Nobel Prize for approach behind light-activated gene therapy

    Karl Deisseroth of Stanford University, Peter Hegemann of Humboldt University and Georg Nagel of the University of Würzburg will share the 2026 Nobel Prize in Physiology or Medicine, worth SKr 12m ($1.19m), for discovering and developing optogenetics. Drugmakers are applying the light-activated approach in ophthalmology: Nanoscope Therapeutics' Mogenry (sonpiretigene isteparvovec), whose BLA in retinitis pigmentosa US regulators accepted in September, has shown late-stage promise in RP and Stargardt disease, while GenSight Biologics' GS030 and Ray Therapeutics' RTx-01 are in RP trials. Luxturna remains the only FDA-approved vision-restoring therapy for RP, with GlobalData estimating sales near $400m in 2032.

    Why it matters: The Nobel award highlights optogenetics, the basis for gene therapies now in late-stage development for blinding eye diseases.

  2. STAT43

    STAT+: Lilly’s Jaypirca approved as first-line CLL therapy

    The FDA has approved Eli Lilly's Jaypirca as a first-line therapy for chronic lymphocytic leukemia (CLL), according to STAT News. The approval moves Jaypirca, already known in CLL treatment discussions, into the earlier-line setting. Details of the decision are behind the STAT+ paywall, with further coverage available to subscribers.

    Why it matters: First-line CLL approval expands the eligible patient population for Lilly's Jaypirca.

  3. Nature73

    Gene therapy for rare lysosomal storage disorder nabs FDA approval

    The FDA has approved Ultragenyx Pharmaceutical's gene therapy rebisufligene etisparvovec (Fayuvi) for paediatric patients with mucopolysaccharidosis type IIIA (MPS IIIA), also known as Sanfilippo syndrome type A. It is the first FDA-approved treatment option for this rare neurodegenerative lysosomal storage disorder.

    Why it matters: First FDA-approved therapy for a rare neurodegenerative lysosomal storage disorder.

  4. Pharmaceutical Technology71

    FDA approves expanded indication for Lilly’s Jaypirca

    The FDA has approved an expanded indication for Eli Lilly's Jaypirca (pirtobrutinib) as a first-line treatment for adults with previously untreated CLL or SLL who lack a 17p deletion. The decision is supported by the Phase III BRUIN CLL-313 trial, in which 282 treatment-naive patients received pirtobrutinib or bendamustine plus rituximab; over a median follow-up of 28 months, pirtobrutinib showed a progression-free survival hazard ratio of 0.20, with median PFS not reached versus 33.5 months and an overall response rate of 94% versus 81%. Jaypirca is already listed in NCCN guidelines as a Category 1 preferred option in relapsed or refractory CLL/SLL after prior covalent BTK inhibitor therapy.

    Why it matters: First-line approval expands Lilly's non-covalent BTK inhibitor into initial CLL/SLL treatment based on strong Phase III data.

  5. WeChat65

    FDA Approves Revolution Medicines' Daraxonrasib for Metastatic Pancreatic Cancer

    On August 26, 2026, the FDA approved daraxonrasib (brand name RASONQUE), developed by Revolution Medicines, for adults with metastatic pancreatic ductal adenocarcinoma who have received at least one prior systemic therapy or are unsuitable for multi-drug combination systemic therapy. It is the first FDA-approved RAS(ON) inhibitor worldwide, extending direct RAS inhibition beyond a single mutation subtype to a broader molecular population.

    Why it matters: First FDA approval of a RAS(ON) inhibitor shifts direct RAS targeting to a broader patient population.

  6. Reddit56

    The Lord of the Incretins: The Reign of Retatrutide... [Latest Research Update]

    Retatrutide, Eli Lilly's unapproved GLP-1/GIP/glucagon triple agonist, delivered mean weight reductions of 17.6%, 23.7% and 25.0% at 4, 9 and 12 mg once weekly versus 3.9% on placebo over 80 weeks in the 2,339-adult TRIUMPH phase 3 program, with a BLA planned for Q1 2027. Clinicians on r/doctorsUK flagged the real-world challenge of moving patients from unregulated gray-market vials, with injection-site infections and toxic reactions already reported, back to regulated supply once the drug is approved.

    Why it matters: Clinicians are publicly debating how to handle patients already self-sourcing retatrutide before approval.

  7. WeChat34

    FDA Transparency Roadmap Aims to Expand Drug Lifecycle Information Disclosure

    The FDA has released a transparency roadmap that would expand public information disclosure across the full product lifecycle for drugs, making transparency a core strategic priority. The roadmap follows the policy direction set in the 2025 Make America Healthy Again (MAHA) Commission report, which called for deep transparency at federal health agencies. It reviews milestones achieved in fiscal year 2026 and lists priority tasks for fiscal year 2027.

    Why it matters: Two sources reported the same event.

  8. WeChat64

    FDA Approves Priovant's LISRAYA as First Oral Therapy for Adult Dermatomyositis

    On August 27, 2026, the FDA approved Priovant Therapeutics' LISRAYA (brepocitinib) 30 mg tablets for adults with dermatomyositis. The FDA described it as the first approved oral treatment option for the indication and the first targeted therapy, being a small molecule directed at JAK/TYK2. Octagam 10%, an intravenous immunoglobulin, had been approved for adult dermatomyositis in 2021.

    Why it matters: First oral and first targeted therapy approved for adult dermatomyositis affects treatment options for clinicians.

  9. WeChat69

    Retatrutide achieves 20.8% weight loss at 80 weeks in TRIUMPH-2 topline results

    Retatrutide delivered an average weight loss of 20.8% at 80 weeks in the 12 mg group of the TRIUMPH-2 trial, which enrolled 1152 people with T2D across dose arms. A subgroup with baseline BMI of 35 or higher lost 23.4%. The topline results were published in The Lancet, and Eli Lilly plans to file with the FDA in Q1 2027.

    Why it matters: TRIUMPH-2 topline data show record weight loss with retatrutide in T2D, with an FDA filing planned.

  10. YouTube84

    Personalized DNA Neoantigen Vaccine Pipeline for Glioblastoma Enters First-in-Human Phase 1

    Elaine Mardis presented a CSHL lecture on a personalized DNA neoantigen vaccine pipeline for glioblastoma: tumor-versus-normal DNA comparison, RNA expression filtering, multi-algorithm neoantigen prediction, and manufacture as a DNA vector vaccine now in a first-in-human phase 1 trial. The design sequences tumors after resection during 6 weeks of radiotherapy, with PD-1 permitted only at progression per FDA requirements. Pre-treatment biopsies from patient 9 already contained tumor-specific T cells that expanded clonally after vaccination, unlike patient 2, and GBM showed greater TCR clonality than brain metastases despite lower T-cell infiltration.

    Why it matters: A leading genomics group describes a DNA neoantigen vaccine for glioblastoma and early phase 1 immune findings.

Oct 4Sun
  1. WeChat70

    FDA Approves Lilly's Pirtobrutinib for First-Line CLL/SLL

    The US FDA has approved a new indication for Eli Lilly's pirtobrutinib (100 mg and 50 mg tablets), announced October 2, as first-line treatment for adults with chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL) who have not received prior treatment and have no known 17p deletion. Pirtobrutinib is a highly selective, non-covalent BTK inhibitor, the first and only approved one, binding both wild-type and C481-mutant BTK.

    Why it matters: FDA approval extends pirtobrutinib into first-line CLL/SLL, expanding use of the only approved non-covalent BTK inhibitor.

  2. WeChat43

    Novo Nordisk Mim8 hemophilia A approval stalled in US over plant issue

    Novo Nordisk said on October 2 that FDA review of Mim8, its hemophilia A drug, has stalled. The company submitted the marketing application in September 2025 and had expected a decision in the third quarter of this year. The delay stems from an issue at a manufacturing facility, not from the drug itself.

    Why it matters: A regulatory delay affecting a hemophilia A candidate, with the cause being manufacturing rather than the drug.

Oct 3Sat
  1. WeChat43

    FDA Approves Edwards Lifesciences' Size-Adjustable AUTUS Pulmonary Valve for Children

    Edwards Lifesciences said on October 1, 2026 that the FDA has approved the AUTUS pulmonary valve, a size-adjustable valve designed for children with congenital heart disease that can expand as the child grows. Valve enlargement is done through minimally invasive transcatheter procedures, which may reduce the need for repeat open-chest surgeries that children traditionally face years after valve replacement.

    Why it matters: FDA approval of a first-of-kind growth-adaptable pediatric valve affects congenital heart disease practice.

CBACBA Frontier Insights (CFI)Chinese Biopharmaceutical Association (CBA)www.cba-usa.org