FDA News

For the first time, the FDA has accepted the initiation of the qualification of an NIT as a noninvasive surrogate endpoint in drug development for MASH.

The first patient in the ongoing Multi-Patient Expanded Access study has surpassed 7 months kidney function, and a second patient has been successfully transplanted.

Lexicon aims to address concerns from a 2024 complete response letter, which cited an increased risk of diabetic ketoacidosis.

Explore the latest advancements in diabetes technology, including FDA-approved insulin pumps and innovative CGMs for weight management.

FDA expands Vonvendi approval for all von Willebrand disease types, enhancing treatment options for adults and children with this rare condition.

Agios announces a 3-month extension for mitapivat's FDA review.

This announcement regarding the new sNDA for roflumilast cream in pediatric patients with psoriasis follows positive findings from the MUSE study.

FDA approval of weekly subcutaneous lecanemab-irmb offers at-home maintenance therapy for early Alzheimer’s after initial intravenous treatment.

Outlook Therapeutics has expressed plans to meet with the FDA to address regulatory issues, as well as expand into European markets.

The REMS label update reduces the frequency of liver function monitoring and removes the embryo-fetal toxicity monitoring requirement.

If approved, the investigative therapy could provide a frontline treatment for patients with SAA who do not have matched sibling donors for cell transplantation.

This episode highlights the groundbreaking approval of semaglutide for metabolic dysfunction-associated steatohepatitis, as well as GoodRx’s new Ozempic pricing.

The FDA has approved Ionis Pharmaceuticals' donidalorsen to prevent HAE attacks in adults and children over 12 years.

With positive topline phase 2 results, parent company Crinetics Pharmaceuticals, Inc., has announced plans to begin phase 3 testing in the second half of 2025.

The accelerated approval is based on findings from Part 1 of the ESSENCE trial and indicates the agent for noncirrhotic MASH with moderate to advanced fibrosis.

The therapy's, marketed as Papzimeos, approval is supported by data from a pivotal Phase 1/2 clinical trial.

Over 11,000 bottles have been voluntarily recalled in response to a consumer complaint of a foreign substance inside a tablet, later identified as aluminum.

The therapy was approved based off of positive data from the pivotal ASPEN trial.

FDA grants Fast Track to NRX-100, the first preservative-free IV ketamine, for treating suicidal ideation in depression.

After overcoming 2 FDA rejections, PharmaTher secures approval for ketamine (KETARX) in surgical pain.

Teva’s AJOVY (fremanezumab) becomes the first CGRP antagonist approved by the FDA for pediatric patients with episodic migraine.

Recent FDA approvals of pegcetacoplan and iptacopan revolutionize C3 glomerulopathy treatment, offering hope and targeted options for patients.

C3G treatment options expand with FDA approvals of pegcetacoplan and iptacopan, offering new hope for patient care.

This FDA News Month in Review provides a round-up of regulatory decisions from July 2025.

The therapy was originally approved under the name Alhemo for people with hemophilia ages 12 and up without inhibitors.