The FDA sent Empower Clinic Services, which does business as Empower Pharmacy, a warning letter dated Sept. 18 citing insanitary conditions at a Houston site inspected in November 2025 and evidence that its compounded tirzepatide and semaglutide products are essentially copies of FDA-approved drugs in violation of Section 503A. As of Sept. 24, Empower still listed both products for sale. The company has 15 working days to respond in writing with steps taken to correct and prevent the violations.
An FDA expert committee voted on September 23 that the clinical benefits of Galleri, a multi-cancer early-detection blood test, outweigh its risks. The vote marks a key step toward formal FDA review of the product, which is positioned as a blood draw for early cancer screening. The agency's decision process now moves forward following the panel's endorsement.
FDA advisors have voted to recommend Grail's multi-cancer blood test Galleri, despite a major trial setback earlier this year and ongoing questions over its efficacy. Grail now awaits an FDA decision on the test. The outcome leaves the company positioned for a potential approval ahead.
An FDA advisory panel endorsed Galleri, Grail's blood test to screen for multiple types of cancer, voting 10 to 0 that there is reasonable assurance the test is safe. The panel split 6 to 4 on effectiveness and voted 7 to 2 with one abstention that benefits outweigh risks for patients meeting the proposed indication's criteria. The FDA is not obliged to follow the panel's advice but often does.
The FDA has issued a direct final rule stating that non-animal methods can be used to assess the preclinical safety of drugs and biological products intended for humans. The change formally recognizes emerging approaches as able to supplement or replace animal testing in human drug safety evaluation. Non-animal methods may be used when they are adequately validated and appropriate for the product and regulatory question.
For the second time this year, HHS announced strategies to shift drug development toward human-based research methods and reduce animal testing. The FDA issued a final rule allowing alternatives to animal testing when 'appropriate' for determining the safety of drugs and biologics, replacing terms like 'animal tests' with 'nonclinical tests.' The NIH will provide $88 million for 10 infrastructure projects encouraging methods such as cell-based assays, organs-on-a-chip, and computer models.
The FDA has approved Cordis' sirolimus drug-coated balloon for treating coronary in-stent restenosis. According to the source, it is the first and only sirolimus drug-eluting balloon for this indication and was cleared through the FDA's breakthrough device pathway.
On September 17, the FDA approved the gene therapy FAYUVI as a one-time treatment for mucopolysaccharidosis type IIIA (MPS IIIA), a rare and fatal childhood genetic disease. The company disclosed a list price of $3.95 million (about 26.45 million yuan), making it tied for the second most expensive drug in the world.
The FDA approved Ionis Pharmaceuticals' Zanvastro (zilganersen, development code ION373), an antisense oligonucleotide, for treating Alexander disease in children and adults. It is the first FDA-approved therapy for the disease and the first disease-modifying therapy directly targeting accumulation of the causal protein. The approval came on September 3, 2026, nearly three weeks ahead of its PDUFA date of September 22, 2026.
The FDA approved Eli Lilly's combination drug treatment for patients with an advanced form of breast cancer, reported Sept. 21, 2026. The decision gives Lilly a new option in the advanced breast cancer setting. Full details of the approval are available to STAT+ subscribers.
FDA and industry have negotiated a new PDUFA agreement, dated September 21, 2026, intended to speed patient access to new drugs and cut costs for developers and the agency by raising the share approved in a single review cycle. The deal targets CMC bottlenecks and FDA staffing, leaves past performance goals unchanged and adopts incremental process reforms. Negotiated while FDA was under political and institutional stress, it includes largely symbolic America First provisions.
The FDA issued a direct final rule on September 21, 2026 clarifying that non-animal methods, such as human cells, organs-on-chips and computer models, can be used where appropriate for safety testing of human drugs and biological products before trials in people. The rule replaces terms like "animal tests" with "nonclinical tests" and "nonclinical studies," as defined under FDORA 2022, and does not eliminate animal studies or change evidentiary standards. The FDA also launched a database with 25 examples of New Approach Methodologies from its review materials, and the public may comment; significant adverse comments would lead the agency to withdraw the rule and proceed via a companion proposed rule.
On September 17, 2026, Bayer announced that the FDA approved finerenone (Kerendia) for chronic kidney disease associated with type 1 diabetes in adults. It is the third US indication for finerenone and the first new FDA-approved treatment option in more than 30 years for CKD related to type 1 diabetes, a setting where treatment options have long been limited, adding drug therapy evidence targeting kidney progression risk.
Beacon Therapeutics' gene therapy for X-linked retinitis pigmentosa improved visual acuity in a pivotal trial, positioning the company to seek regulatory approval for the rare vision loss disease. The company plans to file based on the result, which comes in an indication where larger companies have not succeeded.
On September 17, the FDA approved FAYUVI (rebisufligene etisparvovec-hopf, development code UX111), a gene therapy from Ultragenyx Pharmaceutical, for children with mucopolysaccharidosis type IIIA, also called Sanfilippo syndrome type A. Ultragenyx set the US list price at $3.95 million per dose, second only to Lenmeldy at $4.25 million, making FAYUVI the world's second most expensive drug.
The US FDA has approved Bayer's Kerendia (finerenone) for chronic kidney disease associated with type 1 diabetes, making it the first entirely new therapy for this population in more than 30 years. The approval is finerenone's third in the US, following July 2021 approval for reducing cardiorenal risk in adults with CKD related to type 2 diabetes and July 2025 approval for symptomatic chronic heart failure with LVEF of at least 40%. Finerenone is now the only mineralocorticoid receptor antagonist approved for CKD related to both type 1 and type 2 diabetes.
FDA granted Ultragenyx traditional approval for rebisufligene etisparvovec, now Fayuvi, the first treatment for Sanfilippo syndrome Type A (MPS IIIA). CBER approved the gene therapy on clinical evidence that treated children maintained or improved cognitive function versus an untreated natural-history cohort, rather than on CSF heparan sulfate as a surrogate endpoint. The outcome exceeds the accelerated approval Ultragenyx had sought in its original application and resubmission, while CBER's biomarker stance remains unresolved.
The US FDA has approved Bayer's Kerendia (finerenone tablets) to reduce urinary albumin-to-creatinine ratio (UACR) in adults with chronic kidney disease (CKD) associated with type 1 diabetes. Announced on 17 September 2026, this is the first new treatment option FDA has approved for T1D-related CKD in more than 30 years, and the third indication for finerenone since its first approval in 2021. Around 20% to 30% of type 1 diabetes patients have CKD, and about a quarter of them progress to end-stage renal disease.
The FDA has approved FAYUVI, the first gene therapy for children with Sanfilippo syndrome type A (MPS IIIA), an ultra-rare, fatal lysosomal storage disease that mainly affects the central nervous system. FAYUVI is a one-time intravenous infusion that uses a modified, non-infectious AAV9 vector to deliver functional copies of the SGSH gene to patients' cells. The therapy had received orphan drug, fast track and breakthrough therapy designations from the FDA.
The FDA has approved Fayuvi (rebisufligene etisparvovec), a gene therapy developed by Ultragenyx Pharmaceutical, making it the first FDA-approved therapy for children with mucopolysaccharidosis type IIIA, also known as Sanfilippo syndrome type A. The rare inherited disease progressively damages the brain and nervous system, causing gradual loss of cognitive, speech and other developmental abilities.
