The FDA has approved a fixed-dose combination of lumacaftor and ivacaftor (Orkambi – Vertex) for oral treatment of cystic fibrosis (CF) in patients ≥12 years old who are homozygous for the F508del mutation. About 50% of patients in the US with CF are homozygous for the F508del (also called Phe508del) mutation. This is the first approved indication for lumacaftor; ivacaftor is available alone as Kalydeco for treatment of CF in patients ≥2 years old with other mutations. Orkambi is the first drug to be approved in the US for treatment of patients with the F508del...

The FDA has approved a rapidly disintegrating tablet formulation of the antiepileptic drug levetiracetam (Spritam – Aprecia) for adjunctive treatment of partialonset, myoclonic, and primary generalized tonicclonic seizures. Oral and intravenous formulations of levetiracetam (Keppra, and generics) have been available for years. Although approved by the FDA only as adjunctive therapy, levetiracetam is commonly used as monotherapy for partial-onset and generalized seizures and may also be effective in treating absence seizures and seizures of Lennox-Gastaut syndrome.

The FDA has approved vutrisiran (Amvuttra – Alnylam), a subcutaneously injected small interfering RNA (siRNA), to reduce cardiovascular hospitalizations, urgent heart failure visits, and cardiovascular death in adults with wild-type or variant transthyretin amyloid cardiomyopathy (ATTR-CM). Vutrisiran is the first siRNA to be approved in the US for this indication; it was approved earlier for treatment of polyneuropathy associated with hereditary transthyretin-mediated amyloidosis.

Acute toxic reactions to drugs of abuse continue to be important problems. Some patients may have mixed intoxications with complex combinations of signs and symptoms.

The erythropoiesis-stimulating agents (ESAs) epoetin alfa (Epogen, Procrit) and darbepoetin alfa (Aranesp) are widely used for treatment of anemia and to reduce the need for red blood cell transfusions. Based on the results of recent clinical trials indicating an increased risk of serious adverse events and death associated with ESAs, particularly when used to achieve a hemoglobin concentration ≥12 g/dL, the FDA has revised the prescribing information for these drugs to include a black box warning.

Proton pump inhibitors (PPIs), which are used for treatment of gastroesophageal reflux disease (GERD) and for prevention of upper gastrointestinal adverse effects caused by NSAIDs and aspirin, are one of the most commonly prescribed classes of drugs in the US. All PPIs are similarly effective and generally well tolerated, but their long-term use has been associated with a number of safety concerns. Recommendations addressing these concerns have recently been published.

The FDA has approved the marketing of another extended-release brand-name formulation of the serotonin and norepinephrine reuptake inhibitor (SNRI) desvenlafaxine (Khedezla – Par/Osmotica) for treatment of depression. It is the third extended-release formulation of desvenlafaxine to become available in the US. Khedezla was approved using a 505(b)(2) application, a new drug application (NDA) that relies upon the FDA's findings of safety and/or effectiveness for a previously approved drug.Khedezla does not appear to offer any advantage over the other extended-release formulations of...

The FDA is requiring a new warning in the prescription and over-the-counter labels of the oral second-generation H₁-antihistamines cetirizine (Zyrtec, and others) and levocetirizine (Xyzal, and others) about the risk of severe pruritus following discontinuation of treatment.

A new effervescent formulation of alendronate (Binosto – Mission) was recently approved by the FDA for treatment of osteoporosis. The new 70-mg effervescent tablet is considered bioequivalent to the usual 70-mg tablet formulations of alendronate (Fosamax, and generics), which are difficult to swallow and can cause esophageal injury.1 No published studies of the new formulation are available.LABELING — The new strawberry-flavored effervescent tablet should be dissolved over at least 5 minutes in 4 ounces of water (not mineral or flavored water) and stirred for 10 seconds before drinking. As...

The FDA has approved the pyrimidine analog uridine triacetate (Vistogard – Wellstat Therapeutics) for emergency treatment of a fluorouracil (5-FU) or capecitabine (Xeloda, and generics) overdose or severe toxicity that occurs within 96 hours following administration of one of these drugs. Fluorouracil is a cytotoxic antimetabolite used to treat breast, colorectal, and other cancers; capecitabine is an oral prodrug of fluorouracil.Uridine triacetate, a prodrug, is deacetylated to uridine after oral administration. Excess circulating uridine is converted into uridine triphosphate, which...