FDA Updates

The acceptance is based on positive data from the phase 3 ADORING 1 and ADORING 2 pivotal trials, as well as interim results from the phase 3 ADORING 3 open-label, long-term extension trial.

The indication is for patients aged 12 years of age and older, and the treatment is intended to increase the number of mature neutrophils and lymphocytes in patients.

Tovorafenib is the first systemic therapy to be approved for the treatment of pediatric patients who have low-grade glioma with BRAF rearrangements or fusions.

The approval is based on data from Study 5310, evaluating the pharmacokinetics, safety, and efficacy of Biktarvy (Gilead Sciences Inc) in pregnant individuals.

Dostarlimab plus chemotherapy is the only immuno-oncology-based therapy that showed statistically significant and clinically meaningful survival benefit in the overall patient population.

Naloxone hydrochloride (Amneal Pharmaceuticals Inc) is the generic equivalent to OTC Narcan (Emergent).

Pivmecillinam (Pivya; UTILITY therapeutics Ltd) tablets were approved for female adults with uncomplicated urinary tract infection caused by Escherichia coli, Proteus mirabilis, and Staphylococcus saprophyticus.

This approval makes lutetium Lu177 the first therapy to be approved for the treatment of gastroenteropancreatic neuroendocrine tumors in pediatric patients.

The approval was based on the QUILT-3.032 study, which included 77 adults with carcinoma in situ with or without papillary tumors after a transurethral resection.

With this approval, alectinib is the first anaplastic lymphoma kinase inhibitor to be approved for patients with ALK-positive, early-stage non-small cell lung cancer.

The product is currently being investigated alongside TLX101 in the IPAX-Linz and IPAX-2 clinical trials, and have previously shown efficacy in the IPAX-1 trial.

Ustekinumab (Stelara; Janssen Immunology) is a human monoclonal antibody that treats immune-mediated diseases such as psoriasis and psoriatic arthritis.

If approved, the test could provide more timely and accurate diagnosis, hopefully mitigating the impact of Alzheimer disease (AD) on individuals and the community.

The 5-in-1 meningococcal ABCWY vaccine candidate has an assigned Prescription Drug User Fee Act action date of February 14, 2025.

Although the vaccine has not yet been tested in humans, it demonstrated 100% efficacy in protecting primates who were injected with human Marburg virus disease.

Treatment with LYT-200 is currently being assessed in a phase 1/2 adaptive design trial in advanced/metastatic solid tumors and in a phase 1b clinical trial.

The designation was granted based on overall survival data from an ongoing randomized phase 2 clinical trial.

In addition to oral administration, patients with partial-onset seizures can now take the antiseizure medication via a nasogastric tube if the tablet is crushed and mixed with water.

Benralizumab was previously approved by the FDA in 2017 as an add-on maintenance therapy for patients 12 years and older.

The indication is for adult patients with multiple myeloma who are refractory to lenalidomide and have previously received at least 1 line of therapy.

The treatment is indicated for adolescents at least 12 years of age who weigh at least 25 kg without a previous treatment history of antiretroviral therapies.

This is the fifth indication for fam-trastuzumab deruxtecan-nxki (Enhertu; Daiichi Sankyo and AstraZeneca) approved by the FDA.

The approval expands the prior indication of idecabtagene vicleucel (Abecma; Bristol Myers Squibb), which will make the drug available to patients in earlier lines.

Bimekizumab-bkzx (Bimzelx; Union Chimique Belge) is a humanized monoclonal immunoglobulin G1 antibody that inhibits interleukin (IL)-17A and IL-17F.

New indications include Staphylococcus aureus bloodstream (SAB) infections, acute bacterial skin and skin structure infections, and community-acquired bacterial pneumonia.

The decision follows phase 3 trial results that demonstrated improved progression-free survival and confirmed the safety of Dato-DXd in patients with breast cancer.

Positive phase 3 trial results showed that danicopan was more effective than placebo when treating extravascular hemolysis in paroxysmal nocturnal hemoglobinuria.

The ODD comes after AP303 presented meaningful improvements in renal survival in an ADPKD and the completion of the first study that evaluated healthy human participants.