Obeticholic acid (Ocaliva – Intercept), a farnesoid X receptor agonist, has been approved by the FDA for treatment of primary biliary cholangitis; it is indicated for use in combination with ursodeoxycholic acid (ursodiol, UDCA; Urso, and others) in adults with an inadequate response to UDCA and as monotherapy in adults unable to tolerate UDCA. Obeticholic acid is the second drug to be approved for this indication; UDCA was the first.

The FDA has approved Arymo ER (Egalet), a new extended-release, abuse-deterrent tablet formulation of morphine sulfate, for management of pain severe enough to require daily, around-the-clock, long-term opioid treatment and for which alternative treatment options are inadequate. Arymo ER can only be marketed to deter abuse by injection because another single-entity, extended-release morphine product, MorphaBond, which has not been marketed to date, has marketing exclusivity for deterrence of abuse by the intranasal route through October 2018.

The FDA has approved elacestrant (Orserdu – Stemline), an oral estrogen receptor antagonist, for treatment of estrogen receptor (ER)-positive, human epidermal growth factor receptor 2 (HER2)-negative, estrogen receptor 1 (ESR1)-mutated advanced or metastatic breast cancer in postmenopausal women or men who had disease progression following endocrine therapy. Elacestrant is the first oral selective estrogen receptor degrader (SERD) to be approved for treatment of breast cancer; the injectable SERD fulvestrant (Faslodex, and generics) was approved more than 20 years...

View the Comparison Table: Some Drugs for Postmenopausal Osteoporosis

The FDA has approved pilocarpine 0.4% preservativefree ophthalmic solution (Qlosi – Orasis) for treatment of presbyopia in adults. Pilocarpine 1.25% ophthalmic solution (Vuity) was approved in 2021 for the same indication. Pilocarpine 1%, 2%, and 4% ophthalmic solutions (Isopto Carpine, and others) have been available for years for treatment of glaucoma, but local (e.g., blurry vision and instillation-site pain) and systemic (e.g., sweating and GI disturbances) adverse effects have limited their use.

The FDA has approved the complement factor B inhibitor iptacopan (Fabhalta – Novartis) for treatment of paroxysmal nocturnal hemoglobinuria (PNH) in adults. Iptacopan is the first oral drug to be approved in the US for this indication. Three parenterally administered drugs, the complement C5 inhibitors eculizumab (Soliris) and ravulizumab (Ultomiris) and the complement C3 inhibitor pegcetacoplan (Empaveli), are also approved for treatment of PNH.

Valacyclovir (Valtrex - Glaxo Wellcome), an L- valyl ester of acyclovir (Zovirax), has been approved by the US Food and Drug Administration (FDA) for oral treatment of herpes zoster (shingles) in immunocompetent adults. Famciclovir (Famvir - Medical Letter, 36:97, 1994) is also available for this indication.

Isotretinoin (Accutane - Roche), an effective oral drug for treatment of acne, will soon be available generically. Concerns about its adverse effects, particularly psychiatric symptoms in adolescents, have been widely reported in the media.

The FDA has issued an update (August 18, 2008; www.fda.gov) on occurrences of acute pancreatitis in patients with diabetes taking exenatide (Byetta – Amylin/Lilly). The latest update, which follows an FDA Alert in October 2007, reports 6 cases of hemorrhagic or necrotizing pancreatitis with 2 deaths in patients taking the drug. Whether pancreatitis occurs more often in patients taking exenatide than in patients with diabetes not taking exenatide is not clear.1Given by subcutaneous injection, exenatide is a synthetic peptide that stimulates release of insulin from pancreatic beta cells.2 It...

The FDA has approved ivacaftor (eye va kaf’ tor; Kalydeco – Vertex) for oral treatment of cystic fibrosis (CF) in patients ≥6 years old with the G551D mutation, which is found in about 5% of patients with CF. It is the first drug approved in the US that treats the cause of the disease.