Vyalev (Abbvie), a solution for continuous subcutaneous infusion containing the prodrugs foscarbidopa and foslevodopa, has been approved by the FDA for treatment of motor fluctuations in adults with advanced Parkinson's disease (PD). Vyalev is the first subcutaneously administered product to become available in the US for treatment of PD symptoms. Duopa, a carbidopa/levodopa enteral suspension infused via a nasojejunal tube or percutaneous gastrostomy, was approved in for the same indication 2015.

Fidanacogene elaparvovec-dzkt (Beqvez – Pfizer), an adeno-associated virus (AAV) vector-based gene therapy, has been approved by the FDA for treatment of adults with moderate to severe hemophilia B who do not have neutralizing antibodies to AAV serotype Rh74var and currently receive factor IX prophylaxis therapy or have had life-threatening hemorrhage or repeated, serious, spontaneous bleeding episodes. Beqvez is the second single-dose gene therapy to be approved in the US for treatment of hemophilia B; Hemgenix was the first.

Etranacogene dezaparvovec-drlb (Hemgenix – CSL Behring), an adeno-associated virus vector-based gene therapy, has been approved by the FDA for treatment of hemophilia B in adults who currently receive factor IX prophylaxis therapy or have had life-threatening or repeated, serious, spontaneous bleeding episodes. It is the first gene therapy to be approved in the US for this indication.

Valoctocogene roxaparvovec-rvox (Roctavian – Biomarin), an adeno-associated virus (AAV) vector-based gene therapy, has been approved by the FDA for one-time treatment of severe hemophilia A in adults without pre-existing antibodies to AAV serotype 5. It is the first gene therapy to be approved in the US for treatment of hemophilia A.

The FDA has approved donislecel-jujn (Lantidra – CellTrans), an allogeneic pancreatic islet cellular therapy, for use in conjunction with immunosuppression for treatment of adults with type 1 diabetes who are unable to approach target HbA1c because of current repeated episodes of severe hypoglycemia despite intensive diabetes management and education. Donislecel is the first cell-based treatment to be approved in the US for type 1 diabetes.

The investigational neutralizing IgG1 monoclonal antibody bamlanivimab (LY-CoV555; Lilly) has been granted an FDA Emergency Use Authorization (EUA) for treatment of recently diagnosed mild to moderate COVID-19 in patients who are ≥12 years old, weigh at least 40 kg, and are at high risk for progressing to severe disease and/or hospitalization (see Table 1).

The FDA has approved idecabtagene vicleucel (Abecma – BMS), a B-cell maturation antigen (BCMA)-directed genetically-modified cellular product, for treatment of relapsed or refractory multiple myeloma in adults who received ≥4 prior lines of therapy, including a proteasome inhibitor, an immunomodulatory drug, and an anti-CD38 monoclonal antibody. Abecma is an individualized cellular product prepared from the patient’s own T cells, which are genetically modified to express chimeric antigen receptors (CAR) and then infused back into the patient. Ciltacabtagene autoleucel...

Teprotumumab-trbw (Tepezza - Horizon), an insulin-like growth factor-1 receptor (IGF-1R) inhibitor, has been approved by the FDA for IV treatment of thyroid eye disease. It is the first drug to be approved in the US for this indication.

The FDA recently approved axicabtagene ciloleucel (Yescarta – Kite), a CD19-directed genetically modified cellular product, for treatment of large B-cell lymphoma that is refractory to first-line chemoimmunotherapy or that relapses within 12 months of first-line treatment. It was previously approved for treatment of relapsed or refractory B-cell lymphoma after ≥2 lines of systemic therapy and for treatment of relapsed or refractory follicular lymphoma after ≥2 lines of systemic therapy. Yescarta is an individualized cellular product prepared from the patient's own T cells,...

The FDA has approved ciltacabtagene autoleucel (Carvykti – Janssen), a B-cell maturation antigen (BCMA)-directed genetically-modified cellular product, for treatment of relapsed or refractory multiple myeloma in adults who received ≥4 prior lines of therapy, including a proteasome inhibitor, an immunomodulatory drug, and an anti-CD38 monoclonal antibody. Carvykti is an individualized cellular product prepared from the patient's own T cells, which are genetically modified to express chimeric antigen receptors (CAR) and then infused back into the patient. Idecabtagene...