Approvals, filings, CRLs, breakthrough designations and advisory committees.
The UK's MHRA has approved lurbinectedin for the treatment of adults with extensive-stage small cell lung cancer. The MHRA says it will continue to closely monitor the drug's safety and effectiveness and track its clinical performance on an ongoing basis.
Why it matters: New regulatory approval of an oncology drug by the UK MHRA.
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.
Why it matters: FDA now allows validated non-animal methods to replace animal testing for preclinical drug safety.
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.
Why it matters: FDA and NIH policy changes will directly alter nonclinical testing requirements for drug and biologics developers.
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.
Why it matters: Marks the approval of a sirolimus-coated balloon for coronary in-stent restenosis, a comparator to paclitaxel-coated balloon data.
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.
Why it matters: FDA approval of a gene therapy with one of the world's highest drug prices is a daily-relevant industry event.
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.
Why it matters: First FDA-approved therapy for Alexander disease, a rare indication with no prior treatment.
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.
Why it matters: FDA approval of a Lilly combination therapy for advanced breast cancer directly affects oncology treatment options.
IntraBio's Aqneursa has won approval for a second indication, becoming the first treatment approved worldwide for a rare ataxia. The approval marks another advance for IntraBio in rare neurological diseases, where the company has achieved consecutive successes.
Why it matters: First approved treatment worldwide for a rare ataxia.
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.
Why it matters: FDA rulemaking directly changes nonclinical testing options for drug developers preparing human trials.
Suzhou Zhonghua Pharmaceutical's Weilening, a 100 mg sacubitril/valsartan sodium tablet, has won NMPA approval in China for adults with chronic heart failure with reduced ejection fraction (NYHA class II-IV, LVEF of 40% or less). The company positions the dual-target drug as addressing an unmet need in standard treatment for heart failure, the end stage of cardiovascular disease.
Why it matters: NMPA approval of a generic-type heart failure combination tablet adds a competitor in a large cardiologymarket.
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.
Why it matters: First new FDA-approved treatment for type 1 diabetes-related CKD in over 30 years.
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.
Why it matters: A new gene therapy approval with one of the highest drug prices ever set.
The first prescription for Jie'entai, written by Professor Zhou Jun, has been dispensed in China, moving the drug into clinical use. It is described as the first targeted therapy approved worldwide for biliary tract cancer that has progressed after FGFR inhibitor treatment, giving Chinese patients with drug-resistant advanced disease a treatment option where none existed. The shift is from no available drug to an accessible therapy.
Why it matters: First clinical use of the first approved targeted drug for biliary tract cancer after FGFR inhibitor progression.
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.
Why it matters: First new treatment in over 30 years for type 1 diabetes-related chronic kidney disease, and finerenone's third US indication.
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.
Why it matters: First therapy approved for MPS IIIA and an FDA traditional approval relying on external controls.
EMA's CHMP issued positive opinions for eight new medicines at its September meeting, including two small molecules for ovarian cancer, a potential first daily oral therapy for some adults with hidradenitis suppurativa, and a GLP-2R agonist for short bowel syndrome that FDA previously rebuffed, plus two biosimilars and two generics. Among them is Lifyorli relacorilant, Corcept Therapeutics' selective glucocorticoid receptor antagonist, recommended with nab-paclitaxel for adults with platinum-resistant, high-grade epithelial ovarian, fallopian tube or primary peritoneal cancer who have had one to three prior systemic regimens. EU approval decisions from the European Commission follow.
Why it matters: Eight EU approval recommendations at once include new ovarian cancer options and a potential first oral hidradenitis suppurativa drug.
The FDA has approved Eli Lilly's Inluriyo in combination with Verzenio, entering the oral SERD market competition. Jake Van Naarden, president of Lilly Oncology, told Fierce that the combination shows longer progression-free survival (PFS) than oral SERD monotherapy and that most patients are expected to choose the combination.
Why it matters: FDA approval of Inluriyo plus Verzenio gives readers a PFS anchor for comparison against oral SERD monotherapy in the same indication.
Ascentage Pharma's APG-2575 (lisatocla) has been proposed for breakthrough therapy designation in China, in combination with acalabrutinib for previously untreated adult patients with chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL). Lisatocla is an orally administered selective Bcl-2 inhibitor developed by Ascentage, described as the second worldwide and first in China to show clear efficacy and reach pivotal registration-stage trials as a Bcl-2 inhibitor. The combination regimen showed an overall response rate of 97.7%.
Why it matters: A Chinese breakthrough therapy proposal for a first-in-China Bcl-2 inhibitor with 97.7% ORR in frontline CLL/SLL.
China's NMPA has approved Eli Lilly's selpercatinib tablets, a 5.1-class new drug, for marketing in China, as shown on the agency's website on September 18, 2026. This is the first approval of the tablet formulation of selpercatinib in China. Selpercatinib is the world's first approved highly selective RET inhibitor.
Why it matters: First China approval of the tablet form of the first-in-class RET inhibitor selpercatinib.
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.
Why it matters: First new FDA-approved therapy for type 1 diabetes-related CKD in over 30 years.