FDA Updates

NRX-100 receives FDA fast track designation, offering hope for treating suicidal ideation in depression and bipolar disorder, addressing a critical health crisis.

The action follows 2 FDA-issued complete response letters.

The action is supported by positive safety and efficacy findings from the phase 1a/1b first-in-human Beamion LUNG-1 trial.

Birelentinib gains FDA fast track designation, offering hope for relapsed CLL/SLL patients resistant to current BTK and BCL2 therapies.

The FDA approves dordaviprone, the first systemic therapy for H3 K27M-mutant diffuse midline glioma, offering hope for patients with limited options.

The FDA expands tocilizumab-anoh's approval for treating cytokine release syndrome (CRS), enhancing treatment options for patients aged 2 and older.

The FDA approved fremanezumab for pediatric migraine prevention, offering a new treatment option for children and adolescents aged 6 to 17 years.

The FDA approves aceclidine ophthalmic solution 1.44%, the first aceclidine eye drop for presbyopia, offering a new solution for millions struggling with near vision loss.

FDA approves inclisiran as a monotherapy to effectively lower low-density lipoprotein cholesterol in adults with hypercholesterolemia, enhancing heart disease prevention strategies.

The treatment proved efficacy across all 3 key markers of C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN).

FDA places clinical hold on Sarepta's Elevidys gene therapy after safety concerns, but ambulatory patients regain access amid ongoing investigations.

Sepiapterin treatment allowed patients to liberalize their diet while reaching or exceeding recommended daily protein allowances in patients living with phenylketonuria.

Durvalumab continues to demonstrate efficacy in a variety of cancers, with new data from the phase 3 MATTERHORN trial solidifying its use with chemotherapy in gastric cancer.

FDA approves lonapegsomatropin for once-weekly treatment of growth hormone deficiency, offering a promising alternative for improved patient adherence and outcomes.

Avatrombopag gains FDA approval for pediatric ITP treatment, offering a new oral option for managing chronic thrombocytopenia in children.

The approval represents a major advancement for patients with moderate-to-severe chronic hand eczema, as delgocitinib becomes the first treatment available for this population in the US.

Results from the ZENITH trial show sotatercept's promise for pulmonary arterial hypertension treatment.

Debra Patt, MD, PhD, MBA; and Houston Holmes, MD, explore how the removal of risk evaluation and management strategies (REMS) for chimeric antigen receptor (CAR) T-cell (CAR T) therapies can improve health equity, expand access in community settings, and elevate the pharmacist’s role in care coordination.

The complete response letter (CRL) did not raise safety concerns, but stated the IGNYTE trial was inadequate and did not provide substantial evidence of effectiveness.

The advisory committee cited concerns around ocular toxicity and overall tolerability, raising serious questions about the drug's benefit-risk profile ahead of its scheduled Prescription Drug User Fee Act action date on July 23, 2025.

The CRL states that the STARGLO data do not provide sufficient evidence to support the second-line indication in the US patient population.

ImCheck's ICT01 gains an orphan drug designation from the FDA, offering hope for acute myeloid leukemia (AML) treatment in older patients with limited options.

The FDA recently approved gepotidacin oral tablets for the treatment of uncomplicated urinary tract infections (UTIs).

The FDA approves a prefilled syringe for the zoster vaccine recombinant, enhancing shingles vaccination convenience and effectiveness for adults 50 years and older.

The agent, Kirsty, represents a more affordable alternative to Novolog.

The expanded instructions for use labeling expand on the Farawave and Farawave Nav Pulsed Field Ablation (PFA) catheters.

Debra Patt, MD, PhD, MBA; and Houston Holmes, MD, discuss the ramifications of the FDA's removal of REMS requirements for approved chimeric antigen receptor (CAR) T-cell (CAR T) therapies.

This designation is significant because there are no FDA-approved treatments for transplant recipients with or preventing antibody-mediated rejection (AMR).