News|Articles|September 25, 2026

5 Notable FDA Approvals From the First Half of September

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Key Takeaways

Ropeginterferon alfa-2b outperformed anagrelide for durable modified ELN response in hydroxyurea-refractory/intolerant essential thrombocythemia, introducing long-acting interferon with putative disease-driving clone targeting.
Camizestrant plus CDK4/6 inhibition enables ctDNA-guided endocrine switching upon emergent ESR1 mutations before clinical progression, improving PFS but raising questions absent overall survival confirmation.
PrecivityAD2 clearance signals mainstreaming of blood-based amyloid biomarkers, offering less invasive support for Alzheimer pathology assessment while remaining adjunctive to comprehensive cognitive and neurologic evaluation.
Finerenone became the first new option for CKD in type 1 diabetes in >30 years, lowering UACR but increasing hyperkalemia risk, necessitating structured potassium surveillance.
Rebisufligene etisparvovec-hopf established first-in-class therapy for Sanfilippo A, using AAV9 SGSH gene delivery with supportive CSF heparan sulfate reductions and cognition comparisons to natural history.

The regulatory decisions reflect how advances in biomarkers and molecular medicine are reshaping both diagnosis and treatment.

The first few weeks of September brought a diverse collection of FDA decisions spanning hematology, oncology, neurology, nephrology, and rare disease. Although the therapies address very different conditions, they share the common goal of moving care toward earlier intervention, more precise disease targeting, and treatments that address underlying biology rather than simply managing symptoms.

The approvals also highlight how biomarkers, molecularly targeted therapies, and gene therapy are increasingly influencing treatment and diagnosis. Together, they illustrate a regulatory landscape increasingly focused on detecting disease earlier, identifying molecular changes before a clinical deterioration, and intervening closer to the underlying cause of disease.

FDA Approves Ropeginterferon Alfa-2b in Essential Thrombocythemia

On September 1, the FDA approved ropeginterferon alfa-2b-njft (Besremi; PharmaEssentia) for adults with essential thrombocythemia (ET), making it the first new treatment for the rare blood cancer in nearly 3 decades.¹ The approval expands the drug’s use beyond polycythemia vera and introduces a long-acting interferon into a treatment landscape historically dominated by hydroxyurea and anagrelide.

The approval was supported by data from the phase 3 SURPASS ET trial (NCT04285086), which randomized 174 adults with inadequate response to or intolerance of hydroxyurea to ropeginterferon or anagrelide. Durable modified European Leukemia Net responses at months 9 and 12 occurred in 37.4% of patients receiving ropeginterferon vs 3.6 receiving anagrelide. Unlike therapies primarily focused on controlling platelet counts and symptoms, ropeginterferon is designed to target disease-driving cells in the bone marrow.

**FDA Approves Camizestrant for ER+, HER2– mBC With ESR1 Mutations**

The FDA’s September 4 accelerated approval of camizestrant (Etcamah; AstraZeneca) introduced a different form of earlier intervention. The therapy is approved with a CDK4/6 inhibitor for adults with ER-positive (ER+), HER2-negative (HER2–) locally advanced or metastatic breast cancer when an ESR1 mutation emerges during aromatase inhibitor (AI) and CDK4/6 inhibitor therapy.²

The SERENA-6 trial (NCT04964934) screened more than 3200 patients and randomized 315 with an ESR1 mutation detected through circulating tumor DNA but without clinical disease progression. Median progression-free survival (PFS) was 16.0 months with camizestrant vs 9.2 months with continued AI therapy. The decision followed an FDA advisory committee vote against approval,³ with panelists questioning whether the PFS benefit represented a clinically meaningful benefit without overall survival data.

FDA Clears First Alzheimer Blood Test for Adults as Young as 40

On September 14, the FDA cleared PrecivityAD2, a blood test from C2N Diagnostics that helps identify amyloid pathology associated with Alzheimer disease in adults as young as 40 years who have signs or symptoms of cognitive impairment.⁴ The test uses a single blood draw and is intended to complement, rather than replace, a broader diagnostic evaluation.

The clearance reflects the rapid expansion of blood-based biomarkers in Alzheimer disease. PrecivityAD2 can provide a less invasive alternative to approaches such as cerebrospinal fluid analysis and PET imaging, potentially making biomarker assessment more accessible. Importantly, clinicians are expected to interpret the results alongside medical history, cognitive testing, neurological examination, and other appropriate assessments.

FDA Expands Finerenone Approval to CKD Associated With Type 1 Diabetes

Indications for finerenone grew on September 17 to adults with chronic kidney disease associated with type 1 diabetes, making it the first new FDA-approved treatment for this population in more than 30 years.⁵ The once-daily oral nonsteroidal mineralocorticoid receptor antagonist is approved to reduce urinary albumin-to-creatinine ratio (UACR), a marker associated with kidney disease progression.

The FINE-ONE phase 3 trial (NCT05901831) enrolled 242 adults and found that finerenone reduced UACR by 22% vs placebo at month 3 and 28% at month 6. Hyperkalemia occurred more frequently with finerenone, affecting 10.1% of treated patients vs 3.3% of those receiving placebo, highlighting the need for potassium monitoring.

FDA Approves First Treatment for Sanfilippo Syndrome Type A

Also announced on September 17, the FDA said “yes” to rebisufligene etisparvovec-hopf (Fayuvi; Ultragenyx), making it the first approved treatment for pediatric patients with Sanfilippo syndrome type A, or mucopolysaccharidosis type IIIA.⁶ Previously available care was limited to symptom management, and this disease progressively damages the brain and nervous system.

Fayuvi is a single-infusion intravenous gene therapy that uses an adeno-associated virus 9 vector to deliver a functional copy of the SGSH gene. The goal is to restore production of the sulfamidase enzyme and reduce accumulation of heparan sulfate. In the Transpher A study (NCT02716246), cognitive outcomes favored treatment compared with an external natural-history cohort, while reductions in cerebrospinal fluid heparan sulfate provided evidence of biological activity.

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