The FDA granted accelerated approval to AstraZeneca's Etcamah (camizestrant) combined with a CDK4/6 inhibitor (palbociclib, ribociclib or abemaciclib) for adults with HR-positive, HER2-negative locally advanced or metastatic breast cancer whose ESR1 mutations were detected during aromatase inhibitor and CDK4/6 inhibitor therapy. The FDA also approved a companion diagnostic for detecting emerging ESR1 resistance mutations in ctDNA. The approval rests on positive results from the pivotal Phase III SERENA-6 study.
The FDA has approved Eli Lilly's Mounjaro (tirzepatide) to reduce the risk of major adverse cardiovascular events (MACE-3), including cardiovascular death, nonfatal myocardial infarction or nonfatal stroke, in adults with type 2 diabetes at high risk of cardiovascular events. The approval rests on the phase 3 SURPASS-CVOT trial, which showed Mounjaro was noninferior to active comparator Trulicity in reducing MACE-3 risk.
China's NMPA has approved Zhuhai Tainomab's fully human anti-RSV monoclonal antibody Ruiwei Badan (formerly TNM001) for preventing RSV lower respiratory tract infections in neonates and infants entering or born during their first RSV season. A single 120 mg intramuscular injection protects for up to 9 months, with phase 3 efficacy at day 270 of 69.9% against medically attended RSV LRTI and 90.2% against severe cases, versus roughly 5 to 6 months for the imported nirsevimab. This is the company's second self-developed new drug after its tetanus antibody approved in 2025.
Japan's Ministry of Health, Labour and Welfare has approved GSK's antisense oligonucleotide therapy Hibsago (bepirovirsen) as the first treatment in Japan aimed at achieving a functional cure for chronic hepatitis B. It is indicated for adults who have received at least six months of nucleos(t)ide analogue therapy and meet predefined virological marker criteria. The approval adds an ASO-based option alongside recent siRNA advances in functional cure efforts.
The FDA issued an immediately-in-effect guidance on temporary policies for compounding certain starter parenteral nutrition drug products for neonates, after children's hospitals warned of a supply gap caused by two outsourcing facilities permanently shutting down. The guidance sets regulatory and enforcement priorities for state-licensed pharmacies, federal facilities and outsourcing facilities compounding under sections 503A and 503B. The FDA will keep monitoring supply needs and may take further action.
The FDA granted accelerated approval to AstraZeneca's Etcamah (camizestrant), an oral tablet, combined with a CDK4/6 inhibitor (abemaciclib, palbociclib or ribociclib) for adults with HR-positive, HER2-negative locally advanced or metastatic breast cancer whose tumors show an ESR1 mutation during aromatase inhibitor and CDK4/6 inhibitor therapy, detected by the FDA-authorized Guardant360 CDx companion diagnostic. In the pivotal trial, estimated median progression-free survival was 16 months versus 9.2 months for continuing an aromatase inhibitor plus a CDK4/6 inhibitor. The label carries a boxed warning for irregular heart rhythm, and confirmatory trials are required to verify clinical benefit.
On August 31, 2026, the US Court of Appeals for the Federal Circuit affirmed a Delaware federal court judgment, holding that claims in Exelixis patents US 11,091,439, US 11,091,440 and US 11,098,015 covering the crystalline salt cabozantinib (L)-malate, its formulations and treatment methods meet the written description requirement of 35 U.S.C. §112(a). As a result, the earliest launch of MSN's generic of Cabometyx is pushed to 2030.
On August 26, 2026, the FDA approved Revolution Medicines' Rasonque (daraxonrasib, formerly RMC-6236) for adults with metastatic pancreatic adenocarcinoma who have received at least one prior line of systemic therapy. It is the first FDA-approved targeted therapy aimed at the active-state RAS(ON) protein family. The RAS(ON) molecular glue approval marks one of the biggest clinical advances in pancreatic cancer in nearly a decade.
The FDA has approved Ionis' drug for Alexander disease, a rare and often fatal neurological condition, giving the company a pillar for its rare disease pipeline. The approval follows diagnoses like that of Elise Pearson, whose seizures and MRI changes led to a genetic confirmation of the disease at under three years old. Ionis now has an approved therapy in a indication with no prior treatment named in the news.
Ionis has secured approval for its Alexander disease treatment, a decision the company sees as establishing a pillar of its rare disease pipeline. Alexander disease is a rare genetic condition; in the case described, a toddler diagnosed after seizures and MRI changes had no prior treatment option indicated. The approval positions Ionis to build further in rare diseases.
EMA's Pharmacovigilance Risk Assessment Committee has begun a review of all injectable iron-containing medicines over risks of hypophosphataemia and related bone problems such as osteomalacia. The review was triggered by reports questioning the effectiveness of current risk-minimisation measures for ferric carboxymaltose products, including serious cases after only one or two doses in people without recognised risk factors. PRAC will assess the benefit-risk balance and decide whether changes to marketing authorisations are needed.
Bristol Myers Squibb won accelerated approval for iberdomide (Zenbexus) in combination with daratumumab and hyaluronidase (Darzalex Faspro) and dexamethasone for multiple myeloma patients who have received at least one prior line of therapy. Iberdomide is the newest of BMS's molecular glue degraders to reach the market.
The FDA approved Ionis Pharmaceuticals' Zanvastro as the first disease-modifying therapy for Alexander disease, a rare and deadly neurological condition, for use in children and adults. In a pivotal trial, walking speed stayed stable in treated patients while the control group declined 33%, and the data hinted that treating young children may improve motor function. The drug was generally safe, with serious adverse events more common in the control group.
The FDA has approved Zanvastro (zilganersen) injection from Ionis Pharmaceuticals for Alexander disease in pediatric and adult patients, the first treatment for this rare neurological disorder and the first to target the abnormal GFAP protein buildup that drives it. Efficacy rested on a 49-patient randomized controlled study plus a 4-patient open-label substudy in infants under 2, with treated patients aged 5 and older showing better walking speed at 61 weeks. The antisense oligonucleotide is injected into the spinal canal every three months.
China's NMPA has approved Sanofi's BTK inhibitor rilzabrutinib tablets for adults with persistent or chronic primary immune thrombocytopenia (ITP) who responded poorly to or cannot tolerate corticosteroids, immunoglobulin or similar treatments. Sanofi gained the drug through its $3.68 billion acquisition of Principia Biopharma in September 2020. The FDA approved rilzabrutinib for ITP in August 2025, making it the first BTK inhibitor cleared for that indication.
China's National Medical Products Administration approved Culeivibartamab Injection, brand name Yingmule, from Ruiyang (Shandong) Biopharmaceutical, via priority review. It prevents RSV lower respiratory tract infection in neonates and infants with gestational age of at least 35 weeks entering or born in their first RSV season, excluding children with chronic lung disease including bronchopulmonary dysplasia or clinically significant congenital airway abnormalities. The approval adds a new option for this population.
The FDA approved Revolution's RASONQUE (daraxonrasib), a once-daily oral tablet, for adults with metastatic pancreatic ductal adenocarcinoma who have received at least one prior systemic therapy or are not candidates for multi-drug combination systemic therapy. The approval on 26 August 2026 came about 6.5 months ahead of the original FDA target date. Daraxonrasib is the first approved RAS(ON) multi-selective inhibitor and the first RAS-targeted drug for metastatic PDAC that broadly inhibits multiple RAS mutations.
On September 3, China's NMPA approved two domestic long-acting RSV monoclonal antibodies through the priority review pathway. Tai Nuo Mai Bo's antibody is indicated for newborns and infants entering or born in their first RSV season to prevent RSV lower respiratory tract infection. Rui Yang Bio's antibody covers newborns with gestational age of at least 35 weeks in the same setting, excluding children with chronic lung disease, including bronchopulmonary dysplasia, or clinically significant congenital airway abnormalities. Studies cited show RSV mortality in infants under 1 year old is about 2.5 times higher than with influenza.
The FDA has approved Eli Lilly's tirzepatide to reduce the risk of cardiovascular events in adults with type 2 diabetes who are at high cardiovascular risk. Tirzepatide, a GIP/GLP-1 receptor agonist, is described as the first and currently only agent of its class shown to lower the risk of myocardial infarction, stroke or cardiovascular death in this population, while also providing glycemic control and weight management. The indication has not yet been approved in China.
China's CDE has granted proposed priority review to MediLink Therapeutics' B7-H3 ADC YL201 (Tam-Peli) for recurrent small cell lung cancer after failure of initial platinum-based therapy, with the marketing application filed the same day, September 2. The filing, based on the phase 3 TAISHAN-302 trial, which met its overall survival primary endpoint, makes YL201 the first ADC to secure acceptance of a small cell lung cancer application on phase 3 data. Full efficacy and safety results will be presented orally at the 2026 WCLC Presidential Symposium; a prior priority-review filing in July covered third-line nasopharyngeal carcinoma.