The FDA has approved vamorolone (Agamree – Catalyst), an oral corticosteroid, for treatment of Duchenne muscular dystrophy (DMD) in patients ≥2 years old. Vamorolone is the second oral corticosteroid to be approved in the US for treatment of DMD; deflazacort (Emflaza) was approved in 2017.

The FDA has approved Tonmya (Tonix), a sublingual tablet formulation of the skeletal muscle relaxant cyclobenzaprine, for treatment of fibromyalgia in adults. Cyclobenzaprine is available in immediate-release tablets and extended-release capsules for short-term treatment of muscle spasms and has been used off-label for treatment of fibromyalgia.

The oral second-generation antipsychotic drug lumateperone (Caplyta – Intra-Cellular Therapies), which was approved by the FDA in 2020 for treatment of schizophrenia, is now approved for use as monotherapy or as an adjunct to lithium or valproate for treatment of depressive episodes associated with bipolar I or II disorder in adults.

The FDA has approved Entadfi (Veru), a fixed-dose combination of the 5α-reductase inhibitor finasteride and the phosphodiesterase type 5 (PDE5) inhibitor tadalafil, for initial treatment of benign prostatic hyperplasia (BPH) in men with an enlarged prostate; use of the drug is limited to 26 weeks. Entadfi is the first 5α-reductase inhibitor/PDE5 inhibitor combination to become available in the US. Finasteride (Proscar, and generics) and tadalafil (Cialis, and generics) are also available separately.

The FDA has approved the intravenously administered synthetic vasopressin analog terlipressin (Terlivaz – Mallinckrodt) to improve kidney function in adults with hepatorenal syndrome (HRS) and a rapid decline in kidney function. Terlipressin is the first drug to be approved for this indication in the US; it has been available in Europe and elsewhere for year

Fezolinetant (Veozah – Astellas), a first-in-class neurokinin 3 (NK3) receptor antagonist, has been approved by the FDA for treatment of moderate to severe vasomotor symptoms (VMS) due to menopause. It is the second nonhormonal treatment to be approved in the US for this indication; a low-dose formulation of the selective serotonin reuptake inhibitor (SSRI) paroxetine mesylate (Brisdelle) was approved in 2013.

The FDA has approved an intranasal formulation of the opioid antagonist nalmefene (Opvee – Indivior) for emergency treatment of known or suspected opioid overdose in persons ≥12 years old. Nalmefene, which is available by prescription, is the second opioid antagonist to become available as a nasal spray for this indication; the first was naloxone, which is now available for sale over the counter (Narcan, and generic). Other nasal spray formulations of naloxone and injectable formulations of nalmefene and naloxone are available by prescription (see Table 2).

The FDA has approved the interleukin (IL)-6 inhibitor sarilumab (Kevzara – Sanofi/Regeneron) for treatment of polymyalgia rheumatica (PMR) in adults who had an inadequate response to corticosteroids or cannot tolerate a corticosteroid taper. Sarilumab is the first biologic drug to be approved for treatment of PMR; it was previously approved for treatment of rheumatoid arthritis.

Givinostat (Duvyzat – Italfarmaco), an oral histone deacetylase inhibitor, has been approved by the FDA for treatment of patients ≥6 years old with Duchenne muscular dystrophy (DMD), regardless of the DMD-causing mutation. It is the first nonsteroidal drug to be approved for this indication. The oral corticosteroids deflazacort (Emflaza) and vamorolone (Agamree) are also approved for treatment of patients with all genetic variants of DMD.

The FDA has approved Exblifep (Allecra), a fixed-dose combination of the cephalosporin cefepime and the beta-lactamase inhibitor enmetazobactam, for IV treatment of adults with complicated urinary tract infections (cUTIs), including pyelonephritis, caused by designated susceptible microorganisms (see Spectrum of Activity). Exblifep is the first product that contains enmetazobactam to be approved in the US.