Latest 10 published clinical updates, shown newest first. Previous updates remain visible until newer updates replace them.
2026-09-18 Oncology
FDA approves imlunestrant plus abemaciclib for ESR1-mutated advanced breast cancer
- FDA approved imlunestrant with abemaciclib for adults with ER-positive, HER2-negative, ESR1-mutated advanced or metastatic breast cancer after progression on at least one endocrine-therapy line.
- An FDA-authorized test is required to identify the ESR1 mutation.
- This adds another mutation-directed endocrine strategy for treatment-resistant HR-positive breast cancer.
Source: U.S. FDA
Further Reading ↗2026-09-17 Genetics / Pediatrics
FDA approves first gene therapy for Sanfilippo syndrome type A
- Fayuvi (rebisufligene etisparvovec-hopf) became the first FDA-approved treatment for pediatric mucopolysaccharidosis type IIIA.
- The one-time gene therapy is intended to address the underlying inherited enzyme deficiency rather than provide supportive care alone.
- Long-term efficacy and safety surveillance remain important for gene-therapy recipients.
Source: U.S. FDA
Further Reading ↗2026-09-16 Hematology / Pediatrics
Blinatumomab studied as a chemotherapy-replacing strategy in high-risk pediatric B-ALL
- A randomized trial in children with newly diagnosed high-risk B-cell ALL compared two cycles of blinatumomab with two cycles of conventional chemotherapy after consolidation.
- The study evaluated whether a CD19-directed bispecific T-cell engager can replace selected chemotherapy exposure while maintaining or improving disease control.
- The full trial should be reviewed before translating the strategy into protocol-level practice.
Source: New England Journal of Medicine
Further Reading ↗2026-09-11 Neurology
FDA approves first therapy targeting muscle loss in spinal muscular atrophy
- Isembyld (apitegromab-mstn) was approved for adults and children aged 2 years and older with spinal muscular atrophy who are receiving an SMN2-targeted treatment.
- The therapy targets muscle biology and is used in addition to an SMN2-directed treatment rather than replacing it.
- The approval introduces a complementary treatment approach focused on motor function and muscle loss.
Source: U.S. FDA
Further Reading ↗2026-09-11 Neurology / Imaging
FDA approves F-18 PET tracer to help assess suspected recurrent or progressive glioma
- Pixclara (floretyrosine F 18) was approved for PET imaging to help differentiate recurrent or progressive glioma from treatment-related change.
- The scan is intended to be interpreted together with other diagnostic evaluations rather than as a stand-alone diagnosis.
- The approval expands molecular imaging options in post-treatment glioma assessment.
Source: U.S. FDA
Further Reading ↗2026-09-09 Oncology / Pulmonology
FDA expands accelerated approval of sevabertinib for HER2-mutated NSCLC
- FDA granted accelerated approval to sevabertinib for adults with locally advanced or metastatic non-squamous NSCLC harboring HER2 (ERBB2) tyrosine-kinase-domain activating mutations.
- Patient selection requires an FDA-authorized test.
- Because this is accelerated approval, confirmatory evidence remains relevant to continued approval and long-term positioning.
Source: U.S. FDA
Further Reading ↗2026-09-04 Oncology
FDA grants accelerated approval to camizestrant plus a CDK4/6 inhibitor
- Camizestrant was granted accelerated approval with a CDK4/6 inhibitor for HR-positive, HER2-negative advanced or metastatic breast cancer when an ESR1 mutation emerges during aromatase-inhibitor plus CDK4/6 therapy.
- The approval incorporates molecular monitoring with an FDA-authorized ESR1 test into treatment selection.
- This is a clinically important example of adapting endocrine therapy at molecular progression.
Source: U.S. FDA
Further Reading ↗2026-09-03 Neurology / Genetics
FDA approves first disease-targeted treatment for Alexander disease
- Zanvastro (zilganersen) was approved for pediatric and adult patients with Alexander disease.
- The therapy targets the disease mechanism related to abnormal GFAP protein accumulation.
- It represents the first FDA-approved treatment for this rare progressive neurological disorder.
Source: U.S. FDA
Further Reading ↗2026-09-03 Pediatrics / Endocrinology
Phase 3 trial reports increased growth velocity with oral infigratinib in achondroplasia
- In a randomized phase 3 trial involving children with achondroplasia, once-daily oral infigratinib for one year increased annualized height velocity compared with placebo.
- The result supports FGFR3-pathway modulation as a targeted strategy in achondroplasia.
- Growth benefit, safety and longer-term functional outcomes should be interpreted from the complete trial report.
Source: New England Journal of Medicine
Further Reading ↗2026-09-02 Hematology
Azacitidine–venetoclax compared with induction chemotherapy in fit adults with AML
- A multicenter randomized phase 2 trial compared azacitidine plus venetoclax with conventional induction chemotherapy in previously untreated adults with AML who were eligible for intensive induction.
- Selected molecular AML subgroups were excluded, so applicability depends on disease biology and patient selection.
- The study is relevant to ongoing efforts to define when lower-intensity venetoclax-based therapy can substitute for intensive induction.
Source: New England Journal of Medicine
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