FDA Updates

The agency will focus on collaboration to protect public health; advancing regulatory approaches; developing standards, guidelines, and best practices; and supporting research that evaluates and monitors AI performance.

The decision to vote in favor of idecabtagene vicleucel comes after positive phase 3 trial results demonstrating its efficacy compared with standard regimens.

The FDA-approved topical and oral agent is currently indicated for adult patients with patterned alopecia.

In a phase 1/2 clinical trial, lisocabtagene maraleucel helped patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) achieve complete response rates.

The drug is the first FDA-approved agent for treating hepatorenal syndrome.

The platform is a first-of-its-kind therapeutic approach to promote remyelination among individuals with relapsing/remitting multiple sclerosis.

LSD d-tartrate (MM-120) showed rapid improvements in patients with generalized anxiety disorder by day 2 of the study.

Results from the phase 3 RATIONALE 302 trial showed tislelizumab-jsgr prolonged survival compared to chemotherapy in patients who received prior systemic treatment.

The accelerated approval of resmetirom, a once-daily, oral thyroid hormone receptor-β agonist, is the first treatment available for patients with this disease.

The drug is indicated for patients 5 years of age and older with progressive familial intrahepatic cholestasis and cholestatic pruritus in pediatric patients with Alagille syndrome.

At 4 months, 75% of participants achieved remission and no longer showed signs of depression symptoms in a phase 2 clinical trial.

Individuals included in the study reported positive changes in fatigue, and disease activity displayed endoscopic and histologic remission.

The IVIG therapies were previously approved for 4-week room temperature storage conditions of 25º Celsius during the first 24 months of shelf life.

Semaglutide (Wegovy; Novo Nordisk) reduces the risk of cardiovascular death, heart attack, and stroke in adults with cardiovascular disease and either obesity or overweight when combined with the standard-of-care.

The FDA is convening a committee to discuss phase 3 data after the planned action date, which delays drug availability to patients.

Patients with urothelial carcinoma who were treated with the nivolumab-based regimen had better OS and PFS compared with those who received cisplatin with gemcitabine alone.

The application was previously granted fast track designation and orphan drug designation.

The designation is for all patients with low-grade serous ovarian cancer, regardless of their KRAS status and after 1 or more prior lines of therapy.

Tocilizumab-aazg (Tyenne; Fresenius Kabi) is the first approved biosimilar to tocilizumab (Actemra; Genentech) as both intravenous and subcutaneous.

They also compared the approach of the European Medicines Agency and the FDA in drug approvals.

The device conducts blood biomarker tests that accurately diagnose amyloid pathology and can assist in diagnosing Alzheimer disease in patients with cognitive impairment.

The continuous glucose monitoring system will be available for purchase online and without a prescription starting in summer 2024.

Denosumab-bbdz (Wyost; Sandoz) and denosumab-bbdz (Jubbonti; Sandoz) are approved as interchangeable for all indications of denosumab (Xgeva and Prolia; Amgen).

This designation makes CBL-514 the first drug to receive both Orphan Drug Designation and Fast Track Designation for the treatment of Dercum disease.

The next-generation SRI was previously granted an Orphan Drug Designation in November 2023 and its safety and efficacy are currently being evaluated in clinical trials.

Juvéderm Voluma XC (AbbVie) is the first and only hyaluronic acid dermal filler to receive FDA approval for this indication with results lasting up to 13 months.

The FDA also granted traditional approval to amivantamab-vmjw for adults whose disease has progressed on or after platinum-based chemotherapy.

Hypomyelination with atrophy of the basal ganglia and cerebellum is the most severe form of TUBB4A leukodystrophy that currently has no curative treatment.

NVL-520 is a novel brain-penetrant ROS1-selective TKI being investigated for ROS1-positive metastatic non–small cell lung cancer in patients who have previously been treated with 2 or more ROS1 TKIs.

If approved, epcoritamab (DuoBody CD3xCD20; AbbVie, Genmab) would be the first and only subcutaneous bispecific antibody for treatment in this patient population.