FDA Updates

If approved, the combination would be the first chemotherapy-free, Bruton tyrosine kinase inhibitor–based regimen for adults with newly diagnosed mantle cell lymphoma (MCL).

The provider-administered IL-23 inhibitor improved ACR20 response vs placebo after 2 doses in the phase 3 INSPIRE-1 and INSPIRE-2 trials.

Doruxapapogene ralaplasmid and lalikinogene sifuplasmid-gxfd (Voceryq) targets HPV-6 and HPV-11 to reduce the surgical burden for adults with recurrent respiratory papillomatosis, a rare airway disease.

The FDA expanded approval of talazoparib plus enzalutamide to HRR-mutated metastatic androgen pathway modulation-naïve or -sensitive prostate cancer following a significant radiographic PFS benefit in TALAPRO-3.

The approval offers a once-weekly basal insulin treatment option for patients, reducing the frequency of injections.

The approval gives patients 12 years and older a once-daily oral pellet alternative to capsules for preventing hereditary angioedema (HAE) attacks.

Ravulizumab is now the first C5 complement inhibitor approved for adults with primary IgA nephropathy at risk of disease progression.

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

In the phase 3 B-Well trials, about 1 in 5 adults reached functional cure after a fixed course, but a boxed warning makes monitoring key for pharmacists.

The once-nightly drop, previously approved to reverse pupil dilation, met its primary end point in 2 phase 3 trials, with conjunctival hyperemia most common.

The once-daily device, bioequivalent to the existing prefilled syringe, gives adults with AChR antibody-positive generalized MG a second self-injection option.

Rather than requiring a new trial, the FDA extended evidence from carcinoma in situ to high-grade papillary-only disease, a new approach for this setting.

The FDA approval of pritelivir offers an oral treatment for refractory HSV lesions in immunocompromised adults, with drug interactions creating an important role for pharmacists.

Weltruza, the first subcutaneous long-acting injectable (LAI) formulation of olanzapine, requires no loading doses, oral supplementation, or postinjection monitoring.

BRUIN CLL-313 showed significantly longer progression-free survival vs bendamustine plus rituximab, bringing a noncovalent BTK inhibitor to first-line care.

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.

Ataxia-telangiectasia (A-T) is a rare neurodegenerative disorder that primarily affects the nervous system, causing progressive loss of muscle control and coordination.

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.