FDA Updates

In SCOUT-HCM, mavacamten reduced the Valsalva LVOT gradient by 48 mm Hg vs placebo in adolescents. REMS, echo monitoring, and CYP interactions still apply in the new population.

FDA clears tiratricol for MCT8 deficiency, lowering dangerous T3 and outlining dosing, lab testing, and pharmacy access.

Approval spans early and levodopa-treated disease; a 6-week titration and CYP3A interactions put pharmacists at the center of starting therapy.

The once-daily tablet is the third approved treatment for fibrodysplasia ossificans progressiva (FOP), with CYP3A4 and metformin interaction guidance.

The all-oral combination is supported by phase 3 LITESPARK-011 data showing improved progression-free survival compared with cabozantinib.

Insulin efsitora alfa-gobe (Onswik; Eli Lilly and Company) is designed to deliver steady basal insulin levels over a full 7-day duration.

HYPERION trial data now support sotatercept within a patient's first year of pulmonary arterial hypertension (PAH) diagnosis, cutting clinical worsening risk by 76% compared with placebo.

EMBER-3 data show switching to the combination at progression nearly doubled PFS versus imlunestrant alone in ESR1-mutated advanced breast cancer.

A new device and expanded label tie LDL-C lowering to reduced cardiovascular risk.

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.

The approval makes ustekinumab the first non-TNF-alpha monoclonal antibody available for both major forms of pediatric inflammatory bowel disease (IBD).

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.