FDA approvals, CRLs, advisory committees, clinical holds and guidance — every shift in the U.S. regulatory posture.
Scholar Rock's injectable monoclonal antibody has received FDA approval, making it the first therapy to treat muscle atrophy in patients with spinal muscular atrophy (SMA).
Why it matters: It is the first FDA-approved therapy targeting muscle atrophy in SMA, giving readers a baseline against existing SMA treatments.
Takeda said the FDA has accepted for priority review its new drug application for zasocitinib (TAK-279), an oral TYK2 inhibitor in development for adults with moderate to severe plaque psoriasis. Takeda acquired the drug from Nimbus Therapeutics in 2022 for $4 billion upfront and up to $6 billion in total. A regulatory decision from the FDA is the next step.
Why it matters: FDA priority review sets a decision date for a $6 billion TYK2 inhibitor in plaque psoriasis.
GSK is preparing to submit a first-line FDA application for Jideytro, its first lung cancer drug. The filing is supported by data showing an unprecedented complete response rate. Jideytro is positioned as GSK's first lung cancer asset.
Why it matters: Not given in the Chinese brief.
The FDA has approved Scholar Rock's Isembyld, the first therapy to target muscle loss in spinal muscular atrophy, for adults and children aged 2 and older already receiving SMA therapies that target SMN2. In a late-stage trial, Isembyld combined with an SMN2-targeting drug improved young patients' motor skills over a year while placebo recipients declined, a statistically significant difference. Approval was granted on Friday.
Why it matters: First FDA-approved drug targeting SMA muscle loss adds a new mechanism to existing SMN2 therapy.
Definium Therapeutics' LSD formulation significantly alleviated anxiety symptoms in a third phase 3 trial, completing three straight phase 3 wins and paving the way for an FDA submission next year.
Why it matters: Third consecutive phase 3 win sets up an FDA filing next year.
Labcorp will offer Roche Diagnostics' Elecsys pTau-217 blood test in the US, the first and only FDA-approved single-biomarker blood test for Alzheimer's-related amyloid pathology. It measures pTau-217 in blood to help clinicians identify or rule out Alzheimer's-related pathology in people aged 55 and older with cognitive decline or related symptoms, in both general and specialty care settings.
Why it matters: First FDA-approved single-biomarker Alzheimer's blood test becomes commercially available through Labcorp.
The FDA has accepted Intellia Therapeutics' BLA for lonvoguran ziclumeran (lonvo-z), an in vivo CRISPR therapy for hereditary angioedema, with priority review and a PDUFA goal date of March 10, 2027. The agency does not plan to convene an advisory committee. If approved, lonvo-z would be the first approved in vivo CRISPR therapy and the only single-treatment option for HAE.
Why it matters: FDA acceptance brings the first in vivo CRISPR therapy within reach of approval.
The FDA has approved AstraZeneca's camizestrant (Etcamah), a selective oestrogen receptor degrader, as part of a combination therapy for a subset of breast cancer patients. The drug targets patients whose tumours have acquired resistance mutations to aromatase inhibitors. It expands the SERD class of therapies.
Why it matters: FDA approval of a new SERD widens treatment options for aromatase inhibitor-resistant breast cancer.
The FDA has placed a hold on enrollment in trials of Biohaven's epilepsy drug candidate BHV-7000 after findings from rodent tests. The candidate is covered by a pending $350 million deal announced days before the hold. Biohaven has not disclosed how it will respond to the FDA.
Why it matters: FDA halts enrollment in trials of a drug candidate tied to a pending $350 million deal.
The FDA has approved Moderna's mFlusiva, an mRNA-based seasonal flu vaccine, for people aged 50–64. It is the first seasonal influenza vaccine to use mRNA production, encoding full-length hemagglutinin from three WHO-recommended strains delivered in lipid nanoparticles. In two clinical trials, mFlusiva outperformed GlaxoSmithKline's four-strain vaccine Fluarix in preventing influenza infection or eliciting antibody responses.
Why it matters: First mRNA-based seasonal flu vaccine approval sets a regulatory precedent for Moderna's mRNA platform beyond its earlier vaccines.
The FDA approved Replimune's oncolytic immunotherapy Tudriqev (vusolimogene oderparepvec), formerly RP1, in August for unresectable advanced melanoma that has progressed after PD-1 immunotherapy. The engineered HSV-1 virus encodes GM-CSF and the fusogenic GALV-GP-R glycoprotein to spread through tumours. It is the second oncolytic virus approved, after Amgen's Imlygic (talimogene laherparepvec).
Why it matters: Only the second oncolytic virus ever approved by FDA, expanding options in post-PD-1 advanced melanoma.
The FDA has approved Bayer's tyrosine kinase inhibitor Hyrnuo for first-line treatment of patients with HER2-mutant non-small cell lung cancer. The approval comes roughly half a year after the FDA cleared Boehringer Ingelheim's rival TKI Hernexeos for a similar setting. The two drugs now compete in the same indication.
Why it matters: First-line FDA approval in HER2-mutant NSCLC sets up direct competition between Bayer and Boehringer Ingelheim TKIs.
AstraZeneca's camizestrant, combined with a CDK4/6 inhibitor (palbociclib, ribociclib or abemaciclib), received FDA accelerated approval on September 4 for adults with HR-positive, HER2-negative locally advanced or metastatic breast cancer who developed ESR1 mutations during aromatase inhibitor and CDK4/6 inhibitor treatment. The FDA also approved a companion diagnostic that detects emerging ESR1 resistance mutations in circulating tumor DNA. The approval rests on the pivotal Phase III SERENA-6 trial.
Why it matters: FDA accelerated approval of AstraZeneca's camizestrant plus CDK4/6 inhibition for ESR1-mutant breast cancer, with a ctDNA companion diagnostic.
The Department of Health and Human Services has named Michael Davis, M.D., Ph.D., as permanent director of CDER and Karim Mikhail as permanent director of CBER; both had been serving in acting roles. The appointments come as the agency awaits Senate confirmation of a new FDA commissioner. The two centers oversee drug and biologic reviews respectively.
Why it matters: Permanent leadership at FDA's drug and biologics centers affects review priorities for industry and clinicians.
The Trump administration permanently filled four FDA leadership roles on Tuesday, following its late-August nomination of Heidi Overton to lead the agency. Michael Davis becomes director of the Center for Drug Evaluation and Research, Karim Mikhail, a former Merck official, takes the Center for Biologics Evaluation and Research, and Bret Koplow heads the Center for Tobacco Products. Jared Seehafer becomes the FDA's first deputy commissioner for technology and artificial intelligence.
Why it matters: Permanent leadership at the FDA's drug and vaccine review centers affects how therapeutics are evaluated.
The FDA approved Priovant Therapeutics' Lisraya (brepocitinib), a first-in-class selective dual TYK2/JAK1 inhibitor, for adult dermatomyositis on August 27, 2026. Priovant was set up by Roivant and Pfizer, and the drug, originally coded PF-06700841, was discovered and taken through early-phase I/II trials by Pfizer. It is the only approved targeted oral therapy for dermatomyositis, received orphan drug designation and priority review, and is now available in the US.
Why it matters: First approved targeted oral therapy for dermatomyositis, relevant to clinicians treating inflammatory muscle and skin disease.
The FDA on August 25, 2026 approved Ziihera (zanidatamab-hrii) for first-line unresectable locally advanced or metastatic HER2-positive gastroesophageal adenocarcinoma: IHC 3+ and IHC 2+/ISH+ patients can receive it with tislelizumab plus chemotherapy, and IHC 3+ patients with chemotherapy alone. Under the BeOne Medicines collaboration, Zymeworks has received $81 million, with up to $144 million in further milestones and tiered royalties of up to 19.5%. The approval triggers the structure set up on March 2, 2026, when Zymeworks BC moved 30% of future Ziihera royalties into Zymeworks Royalty Limited Partnership, which borrowed $250 million in non-recourse notes from Royalty Pharma secured solely on those royalties.
Why it matters: FDA approval of a bispecific antibody in first-line gastric cancer activates milestone payments and a $250 million royalty-backed financing.
FDA approval of Rasonque, Revolution Medicines' pancreatic cancer drug that nearly doubles median overall survival, closed the company's expanded access program, leaving patients mid-process without coverage. Commercial insurers like Aetna and Medicare Part D plans take weeks or months to set up coverage, with one Medicare denial reversed only after OptumRx approved prior authorization. Patients such as Juan Solano, who has metastatic disease, remain caught in the gap.
Why it matters: Approval of Rasonque exposes a coverage gap that blocks pancreatic cancer patients from a survival-doubling drug.
Boston Scientific recalled all unused Infinion CX Leads, components of a spinal cord stimulator that delivers mild electrical pulses to block pain signals, because the leads may break. The FDA classified the action as its most serious category, and as of May 27 the company reported 1,081 serious injuries and no deaths. Already implanted devices are not affected; the recall covers models SC-2317-50 and SC-2317-70.
Why it matters: A high-volume serious-injury recall of a widely used chronic-pain implant directly affects patient management.
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.
Why it matters: Accelerated FDA approval of a new endocrine therapy option for ESR1-mutant advanced breast cancer matters to oncology prescribers.
