Former FDA commissioners Robert Califf, Scott Gottlieb and Ned Sharpless criticized current leadership at the Department of Health and Human Services, speaking at the STAT Summit. Their remarks come amid wider debate over how Congress should stabilize the FDA and protect U.S. biotech innovation. The full conversation is available to STAT+ subscribers.
The FDA on Oct. 14, 2026 unveiled the "Rare Disease Framework Initiative," a plan to "reimagine" how it regulates rare disease therapies by setting standards for individual conditions rather than a one-size-fits-all approach. The initiative will review policies from preclinical engagement through post-marketing requirements, seek patient and expert input, and, per Bloomberg News, targets initial recommendations for February. The agency will take feedback at an Oct. 28 meeting on its Silver Spring, Maryland campus.
The FDA launched its Rare Disease Framework Initiative to standardize how clinical trial requirements are applied across rare diseases while tailoring evidence expectations to what small patient populations can realistically generate. Only 5% of an estimated 10,000 rare conditions affecting 30 million Americans have an FDA-approved treatment. The framework builds on programs such as the Rare Disease Innovation Hub, CDER's ARC program and the RDEA pilot, and follows the November 2025 plausible mechanism pathway. The FDA will hold a meeting on Oct. 28 to gather feedback from patients, advocates and industry.
Why it matters: FDA policy shift sets consistent rules for rare disease drug development, a core issue for sponsors of gene and cell therapies.
A year after the FDA announced the first Commissioner's National Priority Vouchers, the agency has granted 22 of them. The update was reported by Nature Reviews Drug Discovery. No further details on recipients or indications were given.
Former FDA commissioners Rob Califf, Scott Gottlieb and Ned Sharpless criticized DOGE at the STAT Summit, calling the Musk-led initiative's cuts of 3,500 workers "one of the biggest disasters in American history" and saying the resulting loss of institutional knowledge will take years to rebuild. Marty Makary resigned as commissioner in May, and White House official Heidi Overton has been nominated to replace him but has not faced a Senate confirmation vote. The ex-officials said acting commissioner Kyle Diamantas and policy lead Grace Graham are restoring rational decision-making, and advised Overton to recruit, support and defend the workforce if confirmed.
The FDA Rare Disease Innovation Hub released its Strategic Agenda on February 2, 2026, setting out its goals and priorities for the coming year for the rare disease community. The Hub, co-led by the directors of CBER, CDER and CDRH with the Oncology Center of Excellence and Office of Orphan Products Development, will focus on products for smaller populations and diseases with variable, poorly understood natural history. It has scheduled public meetings for October 16 and October 28, 2026.
Par Health USA is voluntarily recalling 22 lots of Argatroban Injection 250 mg/2.5 mL (100 mg/mL) to the hospital/user level after particulate matter identified as cellulose, polystyrene, polyethylene terephthalate, stopper material and possibly stainless steel was detected. The affected lots, distributed nationwide to wholesalers and hospitals between December 30, 2024 and May 5, 2026, carry a risk of pulmonary emboli, blood vessel occlusion and vein wall inflammation. No adverse events have been reported; distributors and hospital pharmacies should stop use and return inventory through Inmar, Inc.
The FDA, with the Reagan-Udall Foundation for the FDA, will hold a hybrid public meeting on October 28, 2026, from 1:00 p.m. to 5:00 p.m. ET at the FDA White Oak Campus in Silver Spring, Maryland, and via Zoom. The meeting seeks input from patients, advocates, sponsors and others on the FDA's Rare Disease Framework Initiative, which aims to build a fit-for-purpose approach to rare disease drug development in small patient populations. Public remarks will inform the agency's work on the Framework, with input sought on fit-for-purpose review, regulatory predictability, external engagement and CMC considerations.
The FDA launched the Rare Disease Framework Initiative to build a fit-for-purpose framework for developing drugs and biologics for rare diseases, outlined in a new white paper dated October 14, 2026. The initiative, led by the Rare Disease Innovation Hub with the Commissioner's office, CBER and CDER, will tailor regulatory requirements to small patient populations, with only about 5% of rare conditions having an FDA-approved treatment. A public meeting on October 28 at the White Oak campus, facilitated by the Reagan-Udall Foundation, opens a feedback process that will inform new guidance, regulation revisions, training and organizational changes.
The FDA, with the Reagan-Udall Foundation for the FDA, will hold a hybrid public meeting on October 28, 2026, to gather input on its Rare Disease Framework Initiative, which aims to reimagine the agency's approach to rare disease drug development and review. The framework would tailor regulatory advice and decision-making to the evidence reasonably attainable for each disease, embed the patient voice at defined milestones, leverage external expertise, and apply risk-based approaches to manufacturing review and inspections. Changes could come through regulations, guidance documents and internal processes; FDA set out its aims in a white paper released October 14, 2026.
Why it matters: FDA's rare disease development and review overhaul could change regulatory expectations for sponsors of orphan drugs.
The FDA expanded approval of Anktiva (nogapendekin alfa inbakicept-pmln), given with BCG, to adults with BCG-unresponsive, high-grade papillary-only non-muscle invasive bladder cancer, the first FDA-approved treatment for that disease. The decision relied on evidence from Anktiva's earlier approval for carcinoma in situ, where a trial showed a majority of adults achieved a complete response and many maintained it for at least 12 months. The FDA is finalizing draft guidance to let developers use CIS study evidence for papillary-only NMIBC approvals.
Why it matters: FDA extends Anktiva to BCG-unresponsive papillary-only NMIBC and sets a precedent for cross-indication evidence use.
On October 14, 2026, the FDA expanded approval of nogapendekin alfa inbakicept-pmln (Anktiva, Altor BioScience, an ImmunityBio subsidiary) with BCG to cover adults with BCG-unresponsive non-muscle invasive bladder cancer with high-grade papillary-only tumors, beyond the prior indication limited to carcinoma in situ. The agency extrapolated efficacy from Cohort A of QUILT-3.032, a single-arm trial of 77 patients, which showed a complete response rate of 62%. The recommended dose is 400 mcg intravesically with BCG weekly for 6 weeks induction, followed by maintenance for up to 37 months.
FDA's Center for Drug Evaluation and Research runs Prescribe with Confidence, an educational campaign to help health care providers recognize and treat opioid use disorder and prescribe medications for opioid use disorder when appropriate. The campaign aligns with the Great American Recovery Initiative and lists SAMHSA, NIDA, CDC, HRSA, NRHA, PCSS, the Reagan-Udall Foundation and the American Academy of Family Physicians among its partners. FDA offers free downloadable materials, provider video testimonials and sample social media posts under #PrescribeWithConfidence for organizations to share.
An analysis published in Health Affairs Scholar found that nearly 40% of FDA advisory committee meetings over a recent 20-year period included a patient or consumer representative with active ties to a pharma-funded advocacy group, and on several committees such reps exceeded 50%. More than one in 10 meetings involved a rep affiliated with an organization funded by the drugmaker whose medicine was under review, yet most of these ties were not disclosed at the meetings. The analysis says FDA conflict-of-interest policies for patient and consumer representatives are less developed than those for scientific members, and it raises questions about voting behavior.
FDA is implementing BsUFA II, the Biosimilar User Fee Act programme covering fiscal years 2018 through 2022, under the Biosimilar User Fee Amendments of 2017, Title IV of the Food and Drug Administration Reauthorization Act. The prior BsUFA authority, authorized in 2012 by FDASIA, expired in September 2017, and the FD&C Act required FDA to hold public meetings and negotiate with industry on the reauthorization, including an October 20, 2016 public meeting and steering and finance committee discussions during 2016. The page lists the BsUFA II commitment letter, performance goals, hiring data and implementation plans for enhanced review transparency and communication.
FDA issued a warning letter, reference 320-26-130, to Jiangmen Shuizirun Sanitary Articles Co., Ltd. of Guangdong, China, after a November 3 to 7, 2025 inspection of its drug manufacturing facility documented CGMP violations for finished pharmaceuticals. The firm, whose OTC products are labeled sterile but not sterilized and lack sterility testing at release, also failed to conduct identity testing on incoming components and to validate supplier certificates of analysis. The letter, sent September 23, 2026, asks for an action plan covering customer notifications, recalls or market withdrawals for sterile-labeled batches in U.S. distribution.
FDA issued a warning letter, reference 320-27-03, to Hanlim Pharm Chairman Jungjin Kim on October 2, 2026, citing significant CGMP violations at the company's sterile drug manufacturing facility in Yonginsi, Gyeonggi-do, inspected from April 16 to April 24, 2026. The letter details poor aseptic practices, inadequate smoke studies and cleanroom design flaws risking product sterility, and calls the company's May 15, 2026 response inadequate. Hanlim must now submit a systematic plan covering aseptic practices, cleanroom behavior and independent assessments, or face seizure or injunction.
Why it matters: FDA warning letter over CGMP violations at a sterile drug manufacturing site affects Hanlim Pharm's US supply prospects.
FDA issued a warning letter dated October 6, 2026 to Eurofins SF Analytical Laboratories, a contract testing laboratory in Horsham, Pennsylvania, after a June 1-3, 2026 inspection documented CGMP violations under 21 CFR parts 210 and 211. FDA found analysts selectively omitted too-numerous-to-count results, recorded counts from cracked and desiccated plates, and lacked secondary verification of zero-CFU readings; the firm admitted inconsistent reporting practices dating back to February 2025. FDA deemed the firm's June 22, 2026 response inadequate and demanded an independent laboratory assessment, a remediation plan, and a CAPA plan, warning that failure to comply may bring seizure or injunction.
FDA issued a warning letter dated October 5, 2026, to Earl Lawson, president of Silgan Dispensing Systems, after a March 26 to April 2, 2026 inspection of the Silgan Unicep Packaging facility in Spokane, Washington, which contract manufactures non-sterile over-the-counter drug products. FDA cited CGMP violations including invalidated out-of-specification microbiological results obtained through averaging, an inadequate investigation linked to a customer's Class I recall over potential fungal contamination, and unapproved new drug claims found in product labeling reviewed in July 2026. FDA reviewed the firm's April 22, 2026 response and warned that failure to correct the violations may result in seizure or injunction.
FDA issued a Warning Letter to Lovelace Biomedical Research Institute in Albuquerque, citing Good Laboratory Practice violations found during an April 7-18, 2025 inspection under the Bioresearch Monitoring Program. The quality assurance unit failed to review final study reports so that results reflected raw data, omitting abnormal clinical observations in 20 of 34 animals in one protocol and dosing incidents such as pulled catheters and infiltration at the infusion site. FDA found the May 9, 2025 written response and the revised reports dated May 30, 2025 inadequate.
FDA issued a warning letter to SCA Pharmaceuticals, LLC, a 503B outsourcing facility in Little Rock, Arkansas, on September 14, 2026, following an inspection from October 20 to 30, 2025 that found sterile drug products prepared under insanitary conditions. Cited deficiencies include inadequate ISO 5 certification, poor response to microbial contamination, and aseptic technique failures during production of lots such as Fentanyl Citrate 2 mcg/mL Bupivacaine HCl 0.125% and Methadone HCl 10 mg/mL. FDA reviewed the firm's responses of November 21 and December 31, 2025, and deemed the products adulterated under section 501(a)(2)(A) of the FDCA.
Heidi Overton's confirmation as FDA commissioner faces a tight timeline, Republican divisions over abortion, and Trump administration vaccine policies. Senator Hawley's concerns about an FDA study of mifepristone safety complicate the process, and Republicans hold a one-seat majority on the Senate HELP Committee, so Overton cannot lose any Republican votes if all Democrats oppose her.
The FDA approved Revolution Medicines' Rasonque (daraxonrasib) in August for metastatic pancreatic adenocarcinoma, making it a first-in-class RAS inhibitor for the most common form of pancreatic cancer. Rasonque uses a molecular glue mechanism, binding cyclophilin A and RAS in its GTP-bound 'on' state, unlike KRAS G12C inhibitors such as Lumakras and Krazati. An early-stage trial in September showed promise in lung cancer, raising hopes for use in other hard-to-treat tumors.
Why it matters: First FDA approval of a pan-RAS inhibitor for metastatic pancreatic adenocarcinoma, with early lung cancer activity.
The FDA has approved Ionis Pharmaceuticals' Zanvastro (zilganersen), the first disease-modifying drug for Alexander disease, a rare progressive and often fatal neurological condition affecting about one person per 1-3 million. The antisense oligonucleotide targets RNA encoding glial fibrillary acidic protein (GFAP), reducing its overproduction in astrocytes, where excess GFAP forms clumps that disrupt myelination. Patients suffer impaired motor function, seizures, intellectual disability and developmental delays.
Why it matters: First disease-modifying therapy approved for a rare, previously untreatable neurological condition.
DEA issued two new Notices of Intent on October 9, 2026, based on updated HHS analysis, proposing threshold-based Schedule I control for 7-hydroxymitragynine (7-OH) and mitragynine pseudoindoxyl (MGPI). HHS recommends Schedule I when either substance, or their combined amount, exceeds 1 milligram per gram in solids or 1 milligram per milliliter in liquids, or when the mitragynine ratio to them falls below 100 to 1. DEA will reissue its Notice of Intent without changing the current scheduling approach for MGM-15 and MGM-16, which were temporarily placed in Schedule I on August 26, 2026; controls stay in effect during the 30-day notice period and until a new temporary scheduling order.
Why it matters: DEA sets quantitative thresholds that decide which kratom-derived opioid products fall under Schedule I control.
BoNTax decouples ANI-based species assignment from botulinum neurotoxin characterization for Clostridium botulinum Group I and Clostridium sporogenes. Analysis of 966 genomes informed selection of 46 references that resolved into two species clusters with within-species ANI >95% and between-species ANI <95%, supporting a 95% species threshold. Of 42 validation genomes, 29 of 32 reported assignments were concordant, three prompted re-evaluation, and ten unknown genomes received ANI-based assignments. BoNT typing, including bivalent strains, was fully concordant.
Why it matters: Gives public-health labs a standardized GalaxyTrakr workflow that separates species identity from toxin genotype in closely related clostridia.
FDA has approved Hovilpri (pritelivir) tablets for immunocompromised adults with mucocutaneous herpes simplex virus lesions who did not respond to acyclovir, valacyclovir or famciclovir. In the Phase 3 PRIOH-1 Part C trial of 101 adults, all lesions healed within 28 days in 63% of pritelivir patients versus 34% on investigator-chosen treatments. The drug received priority review, and headache was the most common adverse reaction.
Why it matters: First approval of a new mechanism option for immunocompromised HSV patients resistant or intolerant to existing antivirals.
The FDA's novel drug approvals page for 2026 lists 46 approvals to date, from relacorilant for platinum-resistant ovarian cancer on 3/25 through pritelivir for refractory mucocutaneous herpes simplex lesions in immunocompromised adults on 10/9. The list spans oncology, rare disease, metabolic and imaging agents, including orforglipron for obesity, tulisokibart for ulcerative colitis, and two FOP drugs, zilurgisertib and garetosmab. The list is updated as further approvals are granted.
The FDA has issued a final guidance for industry, "Postapproval Manufacturing Changes to Biosimilar and Interchangeable Biosimilar Products: Questions and Answers," from its drug and biologics centers. It answers common questions on postapproval manufacturing changes for biosimilar and interchangeable biosimilar products licensed under the PHS Act. The guidance finalizes a draft of the same title issued on July 23, 2024, under docket FDA-2024-D-2581, and comments may be submitted at any time.
FDA's CDER approved nine new molecular entities in September 2026, its highest monthly total of the year, lifting the year-to-date count to 45, one short of the full-year 2025 figure. Six of the nine approvals were treatments for rare diseases, including first approved therapies for MCT8 deficiency and Alexander disease. Companies behind the approvals include Abbvie, AstraZeneca, Bristol Myers Squibb, Curium Pharma, Egetis Therapeutics, Eisai and Elevar Therapeutics.
The Alliance for Regenerative Medicine (ARM) has published a playbook, building on FDA draft guidance from this summer, showing how gene-editing developers can reuse toxicity, manufacturing and other data across a platform to cut redundant studies and costs. Beam Therapeutics, which helped create the playbook, designs Phase 1 trials as registrational and aims to move from first-in-human studies to FDA approval in four to five years, versus eight to 10 traditionally. ARM also calls for a bifurcated regulatory framework allowing higher-risk, streamlined development for severe rare diseases with no standard of care, while William Blair analysts say more clarity on the plausible mechanism pathway is needed.
The FDA approved atezolizumab (Tecentriq, Genentech) in combination with a fluoropyrimidine and oxaliplatin on October 8, 2026, for adjuvant treatment of Stage III mismatch repair deficient colon cancer in patients two years of age and older, with the subcutaneous Tecentriq Hybreza approved for those 12 years and older weighing 40 kg or greater. In the ATOMIC trial of 711 adults and one pediatric patient, the hazard ratio for disease-free survival was 0.50 versus mFOLFOX6 alone, and median DFS was not reached in either arm. Reviews are ongoing at partner agencies under Project Orbis.
Why it matters: FDA approval of atezolizumab plus chemotherapy as adjuvant therapy for Stage III dMMR colon cancer, with a disease-free survival hazard ratio of 0.50.
Viatris is buying Pacira BioSciences for $1.65 billion, paying $36.50 per share in cash, a 45% premium, to gain the non-opioid pain drugs Exparel and Zilretta for post-surgical and osteoarthritis knee pain. Pacira's two main products booked $692 million in sales in 2025, up about 4% from 2024. The deal is expected to close by the end of 2026, with generics for Exparel likely from early 2030 and Zilretta patents expiring in 2031.
Why it matters: Large-cap pharma M&A in pain therapeutics, with near-term patent cliffs on the acquired products.
The FDA told a federal court that its safety review of mifepristone, the nation's main abortion pill, will continue into next year, with a safety study due by mid-December and a report no later than March 2027 before any decision on new restrictions. The reevaluation was ordered by Health Secretary Robert F. Kennedy Jr., and the update came in a case brought by Louisiana over mail delivery of the drug. The Supreme Court has blocked changes to mifepristone until it sees the case again.
The FDA has released a set of guidance documents for industry and agency staff issued from July to September 2026. The list includes final guidances on blood collection and storage systems for blood components using the buffy coat method, formal PDUFA meetings, cancer clinical trial eligibility criteria on laboratory values, washout periods and performance status, and FAQs for cellular and gene therapy products. Draft guidances cover premarket approval electronic submission templates, potency assessment of active immunotherapy products, container closure systems for drugs, biologicals and biosimilars.
The FDA approved tucatinib (Tukysa, Seagen, a Pfizer subsidiary) with trastuzumab and pertuzumab on October 7, 2026, for maintenance treatment of adults with unresectable locally advanced or metastatic HER2-positive breast cancer after induction therapy. In the 654-patient HER2CLIMB-05 trial, median progression-free survival was 24.9 months with tucatinib 300 mg twice daily versus 16.3 months with placebo (HR 0.64, p<0.0001). The label carries a boxed warning for hepatotoxicity; overall survival data were immature.
Why it matters: FDA approval extends tucatinib to first-line maintenance, with PFS results from a randomized phase 3 trial.
The FDA's Center for Drug Evaluation and Research issued a draft guidance on studying pharmacokinetics, and where appropriate pharmacodynamics, in patients with impaired hepatic function, including for therapeutic biological products. The non-binding recommendations cover study design, data analysis and impact on dosing for sponsors and applicants. Comments are due by 12/01/2026 under docket FDA-2026-D-8693 before work begins on the final version.
Why it matters: Draft FDA guidance sets expectations for hepatic impairment PK studies that shape sponsors' clinical development plans.
A Netherlands court blocked sales of Merck's subcutaneous Keytruda Qlex in eight European countries, siding with Halozyme in a patent dispute over its under-the-skin drug delivery technology. Separately, Enliven Therapeutics reached agreement with the FDA on a late-stage trial of relcobitinib in chronic myeloid leukemia, enrolling 450 people to test superiority at spurring a major molecular response. Halozyme is also challenging Merck in a U.S. court after a win in Germany.
Why it matters: Patent ruling blocks subcutaneous Keytruda across Europe and FDA-aligned CML trial design sharpens competition for Novartis and Merck.
FDA's CDER lists recent rare disease drug approvals on its Accelerating Rare Disease Cures Program page. In 2026 alone these include Camzyos for an inherited heart condition in children, Emcitate for MCT8 deficiency, Isembyld for muscle loss in spinal muscular atrophy, and Zanvastro for Alexander Disease, among others. The list spans 2021 to 2026 and links to the underlying FDA news releases.
A JAMA piece examines how amyloid PET imaging defines amyloid positivity on the Centiloid scale in Alzheimer disease research and clinical care. Amyloid PET has shaped understanding of disease natural history, guided patient selection and served as a trial outcome measure, supporting FDA approval of amyloid-targeting therapies as the first disease-modifying class. Clinically it aids diagnosis in mild cognitive impairment and confirms plaques before treatment.