Rozanolixizumab-noli (Rystiggo – UCB), a neonatal Fc receptor (FcRn) blocker, has been approved by the FDA for treatment of generalized myasthenia gravis in adults with anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibodies. It is the first drug to be approved for treatment of generalized myasthenia gravis in patients with anti-MuSK antibodies. Four other biologic drugs – the FcRn blocker efgartigimod alfa (Vyvgart; Vyvgart Hytrulo) and the complement inhibitors eculizumab (Soliris), ravulizumab (Ultomiris), and zilucoplan (Zilbrysq) – are...

Acetaminophen (paracetamol; Tylenol, and others) taken in overdosage can cause severe hepatic injury and death. Acetaminophen poisoning is treated with (N-)acetylcysteine. Available for many years in the US as a solution for inhalation therapy (Mucomyst, and others), it subsequently became available for oral use and was recently marketed in the US for intravenous (IV) use as Acetadote (Cumberland).

The motor symptoms of Parkinson's disease (PD) are caused primarily by degeneration of dopaminergic neurons in the substantia nigra. The nonmotor symptoms of the disease are thought to be caused by degeneration of other neurotransmitter systems. No disease-modifying drugs are available for treatment of PD.

Ocrevus Zunovo (Genentech), a subcutaneous (SC) formulation of the anti-CD20 monoclonal antibody ocrelizumab plus human recombinant hyaluronidase-ocsq has been approved by the FDA for treatment of primary progressive and relapsing forms of multiple sclerosis (MS). Intravenous ocrelizumab (Ocrevus), which was approved for the same indications in 2017, is one of the most commonly prescribed drugs for treatment of MS. Ocrelizumab remains the only drug approved for treatment of primary progressive MS.

Ganciclovir sodium (Cytovene - Syntex), a synthetic antiviral nucleoside analog, is now available in the USA for intravenous treatment of cytomegalovirus (CMV) retinitis in immunocompromised patients. CMV retinitis occurs in 20% to 25% of patients with AIDS.

Deferasirox (Exjade - Novartis), an oral chelating agent, recently received accelerated approval from the FDA as an orphan drug for oral treatment of chronic iron overload due to blood transfusions (transfusional hemosiderosis) in patients >2 years old. Deferasirox is a tridentate (2 molecules of deferasirox bind to one atom of iron) chelating agent with high affinity for iron. It has much lower affinity for zinc and copper.

The FDA has approved idelalisib (Zydelig – Gilead), an oral kinase inhibitor, for use in combination with rituximab (Rituxan) for treatment of relapsed chronic lymphocytic leukemia (CLL). It is also approved as monotherapy for treatment of relapsed follicular lymphoma and relapsed small lymphocytic lymphoma (both are subtypes of indolent non-Hodgkins lymphoma) in patients who have received at least two prior systemic therapies.

The FDA recently approved ixazomib (Ninlaro – Takeda), daratumumab (Darzalex – Janssen Biotech), and elotuzumab (Empliciti – BMS) for treatment of relapsed and/or refractory multiple myeloma.

Nesiritide, a recombinant human B-type natriuretic peptide (BNP), has been approved by the FDA of intravenous (IV) treatment of patients with decompensated congestive heart failure who have dyspnea at rest or with minimal exertion (New York Heart Association class IV).

An IV formulation of pantoprazole sodium (Protonix IV - Wyeth-Ayerst), a benzimidazole proton pump inhibitor (PPI), has been approved by the FDA for short-term treatment of Zollinger-Ellison Syndrome (ZES) and gastroesophageal reflux disease (GERD) in patients who cannot take oral drugs. Pantoprazole is the first PPI to be approved for IV use in the US.