Remestemcel-L (Ryoncil – Mesoblast), an allogeneic bone marrow-derived mesenchymal stromal cell therapy, has been approved by the FDA for treatment of steroid-refractory acute graft-versus-host disease (GVHD) in patients ≥2 months old. Steroid-refractory GVHD is associated with a poor prognosis, organ damage, and death, particularly in pediatric patients. Remestemcel-L is the first cellular therapy to be approved in the US for this indication and the only treatment of any kind to be approved for GVHD in children <12 years old.

The tissue plasminogen activator (tPA) tenecteplase (TNKase – Genentech) has been approved by the FDA for treatment of acute ischemic stroke in adults. It is the second tPA to be approved in the US for this indication; alteplase (Activase) was approved in 1996. Tenecteplase was approved in 2000 to reduce the risk of death associated with acute ST-elevation myocardial infarction (STEMI).

About 60% of men ≥60 years old have clinically relevant prostatic enlargement due to benign prostatic hyperplasia (BPH). The goals of treatment are to decrease lower urinary tract symptoms and to prevent disease progression and complications such as acute urinary retention. The American Urologic Association's guidelines for treatment of BPH were recently updated.

Doconsanol 10% cream, a long-chain saturated alcohol, has been approved by the FDA for over-the-counter treatment of herpes labialis.

The FDA has approved the interleukin-6 (IL-6) antagonist siltuximab (Sylvant – Janssen), a recombinant chimeric (human-mouse) monoclonal antibody, for treatment of multicentric Castleman's disease (MCD) in patients who are HIV negative and human herpesvirus-8 (HHV-8) negative. It is the first drug to be approved for this indication.

The FDA has approved idarucizumab (Praxbind – Boehringer Ingelheim) for urgent reversal of the anticoagulant effect of the direct thrombin inhibitor dabigatran etexilate (Pradaxa). Idarucizumab is the first specific reversal agent to become available for one of the new oral anticoagulants.

The FDA has approved the combined use of the programmed death receptor-1 (PD-1) blocking antibody nivolumab (Opdivo) and the anti-CLA-4 antibody ipilimumab (Yervoy) for treatment of BRAF V600 wild-type unresectable or metastatic melanoma. This is the first immunotherapy combination to be approved for treatment of any type of cancer.

The FDA has approved the oral poly (ADP-ribose) polymerase (PARP) inhibitor olaparib (Lynparza – Astra-Zeneca) as monotherapy for treatment of women with advanced ovarian cancer who have BRCA1/2 germline mutations and have received at least 3 prior lines of chemotherapy. Olaparib is the first PARP inhibitor to be approved in the US. It is approved outside the US for maintenance treatment of relapsed BRCA-mutated ovarian cancer.

Dichlorphenamide (Keveyis – Taro), an oral carbonic anhydrase inhibitor, has been approved by the FDA for treatment of primary hypokalemic and hyperkalemic periodic paralysis and related variants. Dichlorphenamide is the first drug to be approved in the US for this indication. It was approved as Daranide in 1958 for treatment of glaucoma, but had not been marketed since 2002.

The FDA has approved the atypical antipsychotic pimavanserin (Nuplazid – Acadia) for treatment of hallucinations and delusions associated with Parkinson's disease. It is the first drug to be approved in the US for this indication.