The FDA has approved voretigene neparvovec-rzyl intraocular suspension (Luxturna – Spark), an adeno-associated virus vector-based gene therapy, for treatment of confirmed biallelic RPE65 mutation-associated retinal dystrophy in patients who have viable retinal cells. It is the first gene replacement therapy approved in the US that targets a disease caused by mutations in a specific gene and the first pharmacologic treatment to be approved for this disorder.

The FDA has approved siponimod (Mayzent – Novartis), a sphingosine 1-phosphate (S1P) receptor modulator, for oral treatment of adults with relapsing forms of multiple sclerosis (MS), including clinically isolated syndrome (initial neurological episode), relapsing-remitting disease, and active secondary progressive MS (SPMS). Siponimod is the second S1P receptor modulator to be approved in the US; fingolimod (Gilenya), which is approved for oral treatment of relapsing forms of MS in patients ≥10 years old, was the first. The purine antimetabolite cladribine (Mavenclad) was also...

The FDA has approved ocrelizumab (Ocrevus – Genentech), a humanized anti-CD20 monoclonal antibody, for treatment of adults with primary progressive or relapsing multiple sclerosis (MS). It is the first anti-CD20 monoclonal antibody to be approved for treatment of MS and the first disease-modifying drug to be approved in the US for primary progressive MS.

Palivizumab (Synagis - MedImmune), an injectable monoclonal antibody, has been marketed in the USA since 1999 for prevention of respiratory syncytial virus (RSV) disease in high-risk infants and children.

The FDA has approved the interleukin-1 (IL-1) beta inhibitor canakinumab (Ilaris – Novartis) for treatment of systemic juvenile idiopathic arthritis (sJIA; formerly called juvenile rheumatoid arthritis or Still’s disease) in children ≥2 years old. Canakinumab was approved earlier for treatment of cryopyrin-associated periodic syndromes (CAPS). Tocilizumab (Actemra), an interleukin-6 (IL-6) inhibitor that has been available since 2010 for treatment of rheumatoid arthritis in adults, was also recently approved by the FDA for sJIA. Canakinumab is the only IL-1 inhibitor approved...

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 severity and rapid spread of COVID-19 (caused by SARS-CoV-2) have raised questions about the use of some drugs in patients with the disease and whether currently available drugs could be effective in treating it. Definitive answers are lacking, but some recommendations can be made. For additional information on specific drugs, see our table Some Drugs Being Considered for Treatment of COVID-19.

Romiplostim (Nplate - Amgen), a recombinant fusion protein injected subcutaneously, and eltrombopag (Promacta - GlaxoSmithKline), a non-peptide taken orally, have been approved by the FDA for treatment of chronic immune thrombocytopenic purpura (ITP) refractory to corticosteroids, immunoglobulins and/or splenectomy.

Eculizumab (Soliris - Alexion) has been approved by the FDA for reduction of hemolysis in patients with paroxysmal nocturnal hemoglobinuria (PNH), a rare form of hemolytic anemia. A recombinant humanized monoclonal antibody, eculizumab is the first drug to be marketed for this indication.

The FDA has approved tofacitinib (toe” fa sye’ ti nib; Xeljanz – Pfizer), an oral Janus kinase (JAK) inhibitor, for treatment of adults with moderately to severely active rheumatoid arthritis (RA) who have had an inadequate response to or are intolerant of methotrexate. Ruxolitinib (Jakafi) is the only other JAK inhibitor available in the US; it is FDA-approved for treatment of myelofibrosis.