FDA Updates

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 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).

FDA fast-tracks TRE-515 for advanced prostate cancer, combining it with radiation therapy to enhance treatment outcomes and patient care.

Previously indicated to reduce the risk of end-stage kidney disease, cardiovascular death, and hospitalization for heart failure (HF) in chronic kidney disease, finerenone is now approved for patients with HF directly, offering a new treatment option.

mRNA-1273, marketed as Spikevax by Moderna, is now fully approved for individuals aged 6 months through 64 years with high-risk features for severe COVID-19.

FDA approves a new dosing schedule for donanemab, enhancing safety and efficacy in treating early symptomatic Alzheimer disease.

The FDA is warning of rare severe itching after stopping long-term use of popular allergy medications cetirizine and levocetirizine, urging patient education.

The new formulation contains little IgA, reducing possible risks associated with infusion in patients with primary immunodeficiency.

Sebetralstat becomes the first FDA-approved oral treatment for hereditary angioedema (HAE), offering rapid relief for patients experiencing acute attacks.

Sunvozertinib becomes the first and only targeted approval treatment for patients with non-small cell lung cancer (NSCLC) harboring epidermal growth factor receptor (EGFR) exon 20 insertion mutations.

The letter cited deficiencies previously identified at a third-party manufacturing vendor unrelated to oxylanthanum carbonate (OLC).

The treatment becomes the first approved BCMAxCD3 bispecific antibody with the potential to achieve biweekly or monthly dosing in patients with relapsed/refractory multiple myeloma.

The injection is indicated for the treatment of sepsis and other serious infections, enhancing treatment efficiency and patient care.