Product Approvals and Launches

Accelerated approval was originally granted in June 2023 for patients aged 4 to 5 years, indicating there was an unmet clinical need for a potentially life-saving treatment for the rare genetic muscle disorder.

The FDA approved pembrolizumab (Keytruda) plus carboplatin and paclitaxel, followed by single-agent pembrolizumab, in adult patients with primary advanced or recurrent endometrial carcinoma.

The FDA has approved durvalumab combined with carboplatin and paclitaxel, followed by single-agent durvalumab.

Bringing the bispecific T-cell engager into the frontline consolidation phase "redefines the standard of care" for patients with B-ALL, an investigator said.

Neurotrophic tyrosine receptor kinase (NTRK)–positive locally advanced or metastatic solid tumors can now be treated with the first FDA-approved treatment option.

Children aged 10 years and older with type 2 diabetes can now improve their glycemic control with a drug previously only approved for adults.

The approval brings a new treatment option to pediatric patients with polyarticular juvenile idiopathic arthritis, which is a form of arthritis affecting multiple joints at the same time.

The latest approval of lisocabtagene maraleucel (liso-cel) in mantle cell lymphoma provides another treatment option and continues the tremendous advances in treatment for patients, said Christopher Flowers, MD, of MD Anderson Cancer Center.

The FDA approved mRNA-1345, a Moderna vaccine for the treatment of respiratory syncytial virus (RSV). This single-dose vaccine offers protection against lower respiratory tract disease in adults over 60 years, with data suggesting long-term effectiveness.

Mantle cell lymphoma is the fourth subtype of non-Hodgkin lymphoma that can be treated with this CAR T-cell therapy.

In the final part of our interview with Binod Dhakal, MD, lead investigator of the CARTITUDE-4 study, he looks to the future of earlier treatment for relapsed/refractory multiple myeloma.

The approval makes belimumab (Benlysta) the first at-home treatment in its class for patients 5 years and older with systemic lupus erythematosus (SLE).

Vamorolone (Agamree) is a first-in-class corticosteroid to treat Duchenne muscular dystrophy (DMD) in patients 2 years and older.

The FDA approved the first 2 biosimilars referencing aflibercept (Eylea). However, patent litigation has left it unclear when the biosimilars will come to market.

Amy Shapiro, MD, medical director, Indiana Hemophilia and Thrombosis Center, continues her discussion on plasminogen deficiency type 1 by highlighting the recent FDA approval of plasminogen (Ryplazim; Kedrion Biopharma), the first treatment specifically indicated for this disorder.

The FDA has approved tarlatamab (Imdelltra; Amgen), a novel bispecific T-cell engager, to treat patients with extensive-stage small cell lung cancer (SCLC).

FDA has given accelerated approval for lisocabtagene maraleucel (liso-cel; Breyanzi) for patients with relapsed or refractory (R/R) follicular lymphoma.

In part 2 of our interview with Binod Dhakal, MD, he addresses how CARTITUDE-4 study findings help to advance the clinical understanding of ciltacabtagene autoleucel (cilta-cel) and the importance of vigilance and education on the treatment’s adverse effects.

The FDA granted full approval for tisotumab vedotin-tftv to treat recurrent or metastatic cervical cancer that progressed after chemotherapy, offering patients improved overall survival and a manageable safety profile.

The FDA has approved fidanacogene elaparvovec-dzkt (Beqvez; Pfizer), a gene therapy administered in a one-time dose, for certain patients with moderate to severe hemophilia B.

The FDA recently approved an antibiotic for the treatment of urinary tract infections (UTIs) in women; a Senate committee recently launched an investigation into the prices of Novo Nordisk’s diabetes and weight loss drugs; US births fell last year, resuming a national slide after a previous increase during the pandemic.

Binod Dhakal, MD, associate professor, Medical College of Wisconsin, is lead investigator of the CARTITUDE-4 study, from which data were used by the FDA to approve ciltacabtagene autoleucel (cilta-cel) for patients with relapsed/refractory multiple myeloma (RRMM) who have received at least 1 prior line of therapy.

The principal investigator of the study leading to approval said this new immunotherapy could be a "game changer" in bladder cancer.

The approval of benralizumab (Fasenra) is supported by evidence from the phase 3 TATE study.

Most recently, the FDA’s Oncologic Drugs Advisory Committee unanimously voted to recommend ciltacabtagene autoleucel (Carvykti; Johnson & Johnson) in patients who have received at least 1 line of treatment for relapsed/refractory multiple myeloma (RRMM).

Idecabtagene vicleucel (ide-cel) was able to triple progression-free survival and reduce the risk of disease progression or death in a phase 3 trial.

Vanda Pharmaceuticals announces the FDA approval of iloperidone (Fanapt) for the acute treatment of bipolar 1 disorder.

Eko Health's artificial intelligence (AI) tool can detect low ejection fraction in seconds via an Eko stethoscope, bringing earlier heart failure detection to routine checkups.

The first-in-class oral factor D inhibitor danicopan (Voydeya) has been approved for the management of extravascular hemolysis in patients with paroxysmal nocturnal hemoglobinuria (PNH).