Eflornithine (Iwilfin – US WorldMeds), an oral ornithine decarboxylase inhibitor, has been approved by the FDA to reduce the risk of relapse in children and with high-risk neuroblastoma who had at least a partial response to prior multiagent, multimodality therapy, including anti-GD2 immunotherapy. About 40-50% of neuroblastoma cases are classified as high-risk and they account for ~15% of all pediatric cancer deaths. Eflornithine is the first drug to be approved to reduce the risk of relapse in children with high-risk neuroblastoma. Eflornithine was previously available in the US...

A synthetic form of the protein apoaequorin is the active ingredient in the over-the-counter dietary supplement Prevagen (Quincy Bioscience), which is heavily marketed to improve memory.

Low-intensity extracorporeal shockwave therapy similar to that used to fragment kidney stones or gallstones is a new option for treatment of erectile dysfunction that is now being advertised extensively in the US. It has not been approved by the FDA for treatment of erectile dysfunction.

The FDA has approved cemiplimab-rwlc (Libtayo – Regeneron), an immune checkpoint inhibitor, for use in combination with platinum-based chemotherapy for first-line treatment of locally advanced or metastatic non-small cell lung cancer (NSCLC) in adults with no epidermal growth factor receptor (EGFR), anaplastic lymphoma kinase (ALK), or ROS1 aberrations and who are not candidates for surgical resection or chemoradiation. The drug was previously approved for first-line treatment of NSCLC in patients whose tumors have high PD-L1 expression and no genomic tumor aberrations....

Enfortumab vedotin-ejfv (Padcev – Astellas), a nectin-4-directed antibody and microtubule inhibitor conjugate, has received accelerated approval from the FDA for use with the immune checkpoint inhibitor pembrolizumab (Keytruda) for treatment of locally advanced or metastatic urothelial cancer in adults who are ineligible for cisplatin-containing chemotherapy. Accelerated approval was based on tumor response rates and the durability of response.

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.

Isatuximab-irfc (Sarclisa – Sanofi), a CD38-directed cytolytic antibody, has been approved by the FDA for treatment of newly diagnosed multiple myeloma in adults who are not eligible for autologous stem cell transplantation (ASCT). The drug was approved earlier for treatment of relapsed or treatment-refractory multiple myeloma.

Zolbetuximab-clzb (Vyloy – Astellas), a claudin 18.2 (CLDN18.2)-directed cytolytic antibody, has been approved by the FDA for use in combination with fluoropyrimidine- and platinum-containing chemotherapy for first-line treatment of locally advanced unresectable or metastatic human epidermal growth factor receptor 2 (HER2)-negative gastric or gastroesophageal junction (GEJ) adenocarcinoma in patients who have CLDN18.2-positive tumors. It is the first monoclonal antibody to be approved in the US that selectively targets CLDN18.2.

The FDA has approved ensartinib (Ensacove – Xcovery), an oral kinase inhibitor, for treatment of anaplastic lymphoma kinase (ALK)-positive, locally advanced or metastatic non-small cell lung cancer (NSCLC) in adults who have not previously received an ALK inhibitor (e.g, crizotinib [Xalkori], certinib [Zykadia], alectinib [Alecensa], brigatinib [Alunbrig], lorlatinib [Lorbrena]). All previously approved ALK inhibitors are also approved for first-line use.

Golodirsen (Vyondys 53 — Sarepta), an antisense oligonucleotide, has received accelerated approval from the FDA for treatment of Duchenne muscular dystrophy (DMD) in the ~8% of patients who have mutations of the dystrophin gene that are amenable to exon 53 skipping. It is the first drug to be approved for this indication and the third to be approved for treatment of DMD; the antisense oligonucleotide eteplirsen (Exondys 51) and the oral corticosteroid deflazacort (Emflaza) were approved earlier.