FDA Updates

In a major policy shift, officials from the FDA announced a new regulatory framework for COVID-19 vaccinations, prioritizing adults 65 years and older and individuals with serious comorbidities putting them at high risk for severe COVID-19.

The approval expands the therapy’s indication to children as young as 7 years.

The Lumipulse G pTau217/ß-Amyloid 1-42 Plasma Ratio is the first in vitro diagnostic device that tests blood to aid in diagnosing Alzheimer disease.

The vaccine, indicated with a restriction on use for those younger than 65 years and without comorbidities, was proven safe and effective in a series of large clinical trials, and marks the first protein-based vaccine to be approved in the United States.

The FDA approves retifanlimab for advanced anal cancer, offering new hope with improved survival rates and treatment options for patients.

Cure Rare Disease's CRD-002 receives FDA orphan drug designation, advancing hope for SCA3 treatment with innovative oligonucleotide therapy.

The approved injection contains dihydroergotamine (DHE), which is the same medication used in hospitals, in a ready-to-use device.

Emrelis is a groundbreaking treatment for advanced non-small cell lung cancer, targeting high c-Met protein overexpression.

The FDA approved belzutifan, the first oral treatment for pheochromocytoma and paraganglioma, offering a new treatment pathway for patients with these rare tumors.

The new at-home test aims to increase comfort and screening timeliness through a more accessible method.

With the FDA approval, avutometinib/defactinib is now the first and only FDA-approved treatment for patients with recurrent low-grade serous ovarian cancer (LGSOC).

The agent demonstrated promising tolerability and manageable safety.

The 12.5-mg dose tablet enhances hypertension treatment options and reduces adverse effects for patients.

The agent targets B7-H3, a commonly overexpressed protein in diffuse intrinsic pontine glioma (DIPG).

Currently, the treatment is undergoing evaluation in a 3-part clinical trial to determine its safety, efficacy, and pharmacokinetics in patients with recurrent advanced/metastatic cancers.

In clinical trials, the dihydroergotamine (DHE) nasal powder had favorable safety profiles and rapid pain relief in patients with migraine with or without aura.

Nipocalimab the first and only FcRn blocker approved in anti-acetylcholine receptor– or anti-muscle-specific kinase adults and children with generalized myasthenia gravis.

FDA approves Zevaskyn, the first gene therapy for RDEB, offering hope for effective wound healing and pain relief in patients.

A 15-mg dose with a corticosteroid taper regimen was superior to placebo in sustained remission from week 12 to 52.

NS-229 is a Janus kinase 1 (JAK1) inhibitor being investigated in eosinophilic granulomatosis with polyangiitis, a rare autoimmune condition that can cause severe allergic and asthmatic symptoms through inflammation of nerve cells.

The treatment received indications as a monotherapy and in combination regimen with carboplatin or cisplatin and gemcitabine.

The in vivo gene editing program is designed to eliminate cccDNA and inactive integrated hepatitis B DNA.

An oral dose of 0.6 mg per 5 mL of colchicine could aid individuals who have trouble with swallowing pills.

The breakthrough designation is supported by clinical data from ongoing phase 1/2 trials.

Multiple phase 3 clinical trials demonstrated dupilumab’s (Dupixent; Regeneron, Sanofi) effectiveness in reducing hives and itch in patients with chronic spontaneous urticaria compared with placebo.

Some patients with aplastic anemia treated with CK0801 achieved durable transfusion independence lasting over 3 years.

The safety profile of diazepam nasal spray among pediatric patients was consistent with older patients, supporting its use in this age group.

The designation was supported based on data from the randomized, double-blind, active-controlled phase 3 interchangeability study.

The new indication expands administration methods for the combination of efgartigimod alfa and hyaluronidase-qvfc, which are already approved for the treatment of some immune-related conditions.