Berotralstat (Orladeyo – Biocryst), an oral plasma kallikrein inhibitor, has been approved by the FDA for prevention of hereditary angioedema (HAE) attacks in adults and children ≥12 years old. The subcutaneously-injected plasma kallikrein inhibitor lanadelumab-flyo (Takhzyro) and the human plasma-derived C1 esterase inhibitors (C1INHs) Cinryze, which is given IV, and Haegarda, which is given SC, have been available for prophylaxis of HAE for years.

Recombinant human deoxyribonuclease I (rhDNase; dornase alfa; Pulmozyme - Genentech), an enzyme that hydrolyzes extracellular DNA, is now available as a purified solution to decrease the viscosity of sputum in patients with cystic fibrosis. It requires a nebulizer to produce an aerosol mist for inhalation.

The FDA has approved the use of nitroglycerin ointment 0.4% (Rectiv – ProStrakan/Aptalis) for treatment of moderate to severe pain associated with chronic anal fissure; the same drug is marketed as Rectogesic in Europe. Nitroglycerin ointment 2% (Nitro-Bid) is available in the US for prevention of angina.

Ado-trastuzumab emtansine (Kadcyla – Genentech), a human epidermal growth factor receptor 2 (HER2)-targeted antibody and microtubule inhibitor conjugate, has been approved by the FDA for intravenous (IV) treatment of HER2-positive metastatic breast cancer in patients previously treated with trastuzumab (Herceptin) and a taxane. The prefix was added to the new conjugate’s name at the request of the FDA to distinguish it from trastuzumab.

The FDA has approved telotristat ethyl (Xermelo – Lexicon), a tryptophan hydroxylase inhibitor, for use in combination with a somatostatin analog (SSA) for treatment of carcinoid syndrome diarrhea inadequately controlled with SSA therapy alone. Telotristat ethyl is the first oral drug to be approved in the US for this indication.

The Bruton's tyrosine kinase (BTK) inhibitor zanubrutinib (Brukinsa), which was previously approved by the FDA for treatment of mantle cell lymphoma, Waldenström's macroglobulinemia, and relapsed or refractory marginal zone lymphoma, has now been approved for treatment of chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) in adults. The BTK inhibitors ibrutinib (Imbruvica) and acalabrutinib (Calquence) were approved earlier for treatment of CLL and SLL.

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.

Telisotuzumab vedotin-tllv (Emrelis – Abbvie), a c-Met-directed antibody and microtubule inhibitor conjugate, has received accelerated approval from the FDA for treatment of locally advanced or metastatic nonsquamous non-small cell lung cancer (NSCLC) in adults with high c-Met protein overexpression [≥50% of tumor cells with strong (3+) staining] who received prior systemic therapy. Accelerated approval was based on the overall response rate and duration of response.

For patients with diabetes poorly controlled with a single oral drug, addition of a second drug with a different mechanism can be helpful. A fixed-dose combination (Actoplus met - Takeda) of two widely used antihyperglycemic drugs, the thiazolidinedione pioglitazone (Actos) and the biguanide metformin (Glucophage, and others), has been approved by the FDA for management of type 2 diabetes. It is indicated for patients already being treated with both pioglitazone and metformin or as second-line therapy for those not adequately controlled with either metformin or pioglitazone alone....

Posaconazole (Noxafil - Schering-Plough), an oral azole antifungal with a chemical structure similar to that of itraconazole (Sporanox), has been approved by the FDA to prevent Candida and Aspergillus infections in severely immunocompromised patients and for treatment of oropharyngeal candidiasis. It is likely also to be used off-label for treatment of other fungal infections, including those caused by Mucor and other Zygomycetes.