FDA Updates

The FDA has approved rebisufligene etisparvovec-hopf (Fayuvi), the first treatment designed to alter the course of mucopolysaccharidosis type IIIA.

This update came with no changes to the safety sections of labeling, with no new signals identified with longer follow-up.

The update shortens required observation to 6 to 8 hours from 22 to 24, easing tarlatamab administration in community settings for ES-SCLC.

The approval is supported by data from the phase 3 FINE-ONE clinical trial.

Apitegromab-mstn is cleared as an add-on to SMN2-targeted treatment for patients aged 2 and older, directly addressing muscle loss.

The expanded approval of leniolisib makes it the first approved treatment for pediatric patients with activated phosphoinositide 3-kinase delta syndrome.

The FDA expanded the accelerated approval of sevabertinib to include treatment-naive adults with locally advanced or metastatic nonsquamous NSCLC harboring HER2 tyrosine kinase domain-activating mutations.

Camizestrant is the first cancer therapy guided by a ctDNA resistance mutation detected before imaging shows disease progression.

Zilganersen, an antisense oligonucleotide targeting glial fibrillary acidic protein, is cleared for pediatric and adult patients across all ages.

Rusfertide gives adults with polycythemia vera a first-in-class option to cut phlebotomy dependence and control hematocrit.

Bictegravir/lenacapavir (Bixlenvo) offers the first single-tablet option for many patients with HIV currently receiving complex antiretroviral regimens.

Mounjaro becomes the first GIP/GLP-1 receptor agonist cleared to lower MACE risk, based on the SURPASS-CVOT head-to-head trial.

The updated formula targets the JN. 1-lineage XFG variant and begins shipping immediately to pharmacies, hospitals, and clinics nationwide.

Brepocitinib (Lisraya), a once-daily TYK2-JAK1 inhibitor, is the first oral therapy approved for adults with the rare autoimmune disease.

Early-phase trial data showed antitumor activity in heavily pretreated metastatic colorectal cancer.

If Heidi Overton is confirmed, the FDA nominee's views on abortion pills, vaccines, and drug review could reshape pharmacists' daily work.

CK0803 is an investigational allogeneic cord blood–derived regulatory T-cell therapy designed to target neuroinflammation in amyotrophic lateral sclerosis.

Daraxonrasib, an oral RAS inhibitor, was approved for adults with metastatic pancreatic adenocarcinoma after prior systemic therapy or when multiagent systemic therapy is not appropriate.

The FDA has expanded the approval of dolutegravir expands access to a second-generation integrase inhibitor for infants in the neonatal period.

Abbott's Libre Duo 10 Day tracks both glucose and ketones to flag rising DKA risk in people with diabetes 2 years and older.

The FDA has approved 2 zanidatamab-hrii–based regimens following phase 3 results demonstrating significant improvements in progression-free survival.

The first single-biomarker blood test is cleared for both rule-in and rule-out of amyloid pathology in primary and specialty care.

Nipocalimab is the first therapy approved specifically for wAIHA, a rare, life-threatening autoantibody disease driving red blood cell destruction.

The FDA has granted fast track designation to ERAS-0015, an investigational oral pan-RAS molecular glue that has shown early antitumor activity in previously treated KRAS G12X pancreatic ductal adenocarcinoma.

The FDA-granted priority review raises the possibility of an immunotherapy-first approach that could allow some patients to avoid chemotherapy, radiation, and surgery.

The FDA granted fast track designation to safusidenib, an investigational oral IDH1 inhibitor that produced durable responses in a phase 2 study of patients with treatment-naive grade 2 IDH1-mutant glioma.

Cardiomyopathy, hepatitis B, breast cancer, hypertension, and myeloma decisions land in the FDA's final-quarter queue.

Its targeted PDE4 mechanism, favorable safety profile, and supportive pediatric data make it a meaningful long-term treatment addition.