Deflazacort (Emflaza – PTC Therapeutics), an oral corticosteroid, has been approved by the FDA for treatment of Duchenne muscular dystrophy (DMD) in patients ≥5 years old. It has been available outside the US for many years. Deflazacort is the second drug to be approved for treatment of DMD; eteplirsen (Exondys 51), an antisense oligonucleotide approved for IV administration in patients with mutations of the dystrophin gene amenable to exon 51 skipping (about 13% of DMD cases), was the first.

Caplacizumab-yhdp (Cablivi – Sanofi /Genzyme), a von Willebrand factor-directed antibody fragment, has been approved by the FDA for use in combination with plasma exchange and immunosuppressive therapy for treatment of immune-mediated thrombotic thrombocytopenic purpura (iTTP; formerly called acquired thrombotic thrombocytopenic purpura [aTTP]) in adults. It is the first drug to be approved in the US for this indication.

The oral kinase inhibitor capivasertib (Truqap – AstraZeneca), a first-in-class AKT inhibitor, has been approved by the FDA for use in combination with the selective estrogen receptor degrader (SERD) fulvestrant (Faslodex, and generics) for treatment of hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative, locally advanced or metastatic breast cancer in adults with one or more PIK3CA/AKT1/PTEN-alterations who had disease progression on at least one endocrine-based regimen for metastatic disease or recurrence on or within 12 months of completing...

The FDA has approved the oral tyrosine kinase inhibitor repotrectinib (Augtyro – BMS) for treatment of locally advanced or metastatic ROS1-positive non-small cell lung cancer (NSCLC) in adults. Repotrectinib is the third oral tyrosine kinase inhibitor to be approved for this indication in the US; crizotinib (Xalkori) and entrectinib (Rozlytrek) were approved earlier.

The FDA has approved the nonsteroidal mineralocorticoid receptor antagonist (MRA) finerenone (Kerendia) to reduce the risk of cardiovascular death, hospitalization for heart failure (HF), and urgent HF visits in adults with HF with a left ventricular ejection fraction (LVEF) ≥40%. Finerenone was approved in 2021 to reduce the risk of kidney disease progression and cardiovascular events in adults with chronic kidney disease (CKD) associated with type 2 diabetes.

The US Food and Drug Administration recently licensed a new Salk-type injectable polio vaccine (Poliovirus Vaccine Inactivated - Connaught) that is more potent in its antigenicity than any other inactivated polio vaccine (IPV) previously available in the USA. Live oral polio vaccine (OPV) has been the preferred vaccine for routine immunization in the USA since the early 1960's. Its advantages over IPV have included ease of administration, superior immunogenicity, induction of gastrointestinal immunity, increased immunization through spread of vaccine virus to unimmunized susceptible ...

Clomipramine (Anafranil - Ciba-Geigy), a tricyclic antidepressant, is now available for treatment of obsessive compulsive disorder (OCD) in accordance with the new US Food and Drug Administration (FDA) procedure for promising investigational drugs (FE Young et al, JAMA, 259:2267, 1988). In other countries, clomipramine has been widely used for many years for treatment of depression.

A topical formulation of 0.75% metronidazole gel (MetroGel - Curatek), an antibacterial, antiparasitic agent, was recently approved by the US Food and Drug Administration for treatment of rosacea.

Levamisole (Janssen), an old antiparasitic drug widely used for treatment of ascariasis in animals, is now available from the National Cancer Institute (NCI) for investigational use with fluorouracil (5-FU; Adrucil; and others) as an adjuvant for treatment of resectable colon cancer that has metastasized to regional lymph nodes (Dukes stage C).

An oral formulation of mesalamine (5-aminosalicylic acid, 5-ASA, Asacol - Proctor & Gamble), was recently approved by the US Food and Drug Administration (FDA) for treatment of mildly to moderately active ulcerative colitis; it has not been approved for maintenance of remissions. Olsalazine (Dipentum), a similar drug (Medical Letter, 32:105, 1990), is approved for maintenance of remissions but not for treatment.