FDA Updates

Congenital muscular dystrophy type 1a is a severe, early-onset condition that currently has no treatment, but MDL-101 may serve as a 1-time, durable treatment option.

The FDA announces a Class II recall for the lot #220128, which were reported to contain the presence of nitrosamine.

The approval marks Abrysvo as the first and only RSV vaccine designated for adults younger than 50 that are at increased risk for LRTD.

In April 2024, the FDA approved ustekinumab-aekn as a subcutaneous injection for the treatment of moderate to severe plaque psoriasis and active psoriatic arthritis.

The diagnostic test is a companion to vorasidenib, an isocitrate dehydrogenase inhibitor that received FDA approval this past summer.

Zolbetuximab is the first approved CLDN18.2-targeted treatment for gastric and gastroesophageal junction adenocarcinoma.

The additions include Dextrose 70% intravenous (IV) solution, Lactated Ringers IV Solution, and Peritoneal Dialysis Solution, which were affected by Hurricane Helene.

Patients aged 7 years and older can now be treated for their cataplexy or excessive daytime sleepiness with the extended-release oral formulation.

Gepotidacin could be first-in-class oral antibiotic treatment approved for uUTIs in over 20 years.

Imuldosa has received approval for all indications of its reference product, including ulcerative colitis, psoriatic arthritis, and Crohn disease.

This approval marks the first and only anti-tissue factor pathway inhibitor approved in the US for the treatment hemophilia A or B, and the first approved hemophilia treatment to be administered via a pre-filled, auto-injector pen.

As the first dual neurokinin-1 and 3 receptor antagonist, elinzanetant could provide benefit for women suffering from vasomotor symptoms.

The exemption allows more time for partners to adhere to enhanced distribution security requirements in the Food, Drug, & Cosmetics Act and prevents possible supply chain disruptions.

The FDA also approved the FoundationOne Liquid CDx assay as a companion diagnostic device to identify patients who would benefit from the treatment.

The regulatory action is based on a positive preliminary analysis of the VANTAGE trial.

Healgen Rapid Check COVID-19/Flu A&B Antigen Test is the first OTC test to detect influenza to be granted a marketing authorization outside of Emergency Use Authorizations.

Survodutide is a glucagon/glucagon-like peptide-1 (GLP-1) receptor dual agonist that activates the glucagon and GLP-1 receptors to better control metabolic function.

Setrusumab could offer a rapid and clinically meaningful decrease in fracture rate in individuals with osteogenesis imperfecta.

Nivolumab is the only PD-1 inhibitor to show statistically significant and clinically meaningful benefits in non-small cell lung cancer compared with chemotherapy.

The treatment aims to reduce prolonged QTc interval in patients with long QT syndrome.

The new screening test provides an easier, convenient option for patients seeking to screen themselves for colorectal cancer.

Currently, Danon disease has a poor prognosis and does not have any pharmaceutical therapeutics for treatment or management.

In a phase 1 trial, the nicotine replacement therapy (NRT) demonstrated a higher mean maximum plasma concentration was achieved faster than with an existing inhaled NRT.

Currently, VLS-1488 is being evaluated in a phase 1/2 trial (NCT05902988) in various advanced tumors, including high-grade serous ovarian cancer (HGSOC).

The designation was based on results from the DESTINY-Breast06 trial, evaluating fam-trastuzumab deruxtecan-nxki (Enhertu; AstraZeneca, Daiichi Sankyo) compared with chemotherapy.

Positive trial results laid the groundwork for the designation.

Navenibart was developed to aid treatment of hereditary angioedema (HAE) to provide swift and sustained HAE attack prevention through administration every 3 to 6 months.