The FDA has approved emicizumab-kxwh (Hemlibra – Genentech), a subcutaneously injected, factor IXa- and X-directed antibody, for routine prophylaxis to prevent or reduce bleeding episodes in patients with hemophilia A. Emicizumab is not recommended for treatment of bleeding.

The FDA has approved onasemnogene abeparvovec-xioi (Zolgensma – Avexis), an adeno-associated virus vector-based gene therapy, for one-time IV treatment of children <2 years old who have spinal muscular atrophy (SMA) and bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. It is the first gene therapy and the second drug to be approved in the US for SMA; nusinersen (Spinraza) was approved in 2016.

The FDA has approved Duaklir Pressair (Circassia), a fixed-dose combination of the long-acting muscarinic antagonist (LAMA) aclidinium bromide and the longacting beta₂-adrenergic agonist (LABA) formoterol fumarate, for maintenance treatment of chronic obstructive pulmonary disease (COPD). Aclidinium/formoterol is the fifth LAMA/LABA combination to be approved in the US for this indication.

In recently published clinical trials, once-weekly subcutaneous injection of the glucagon-like peptide 1 (GLP-1) receptor agonist semaglutide (Ozempic – Novo Nordisk), which is FDA-approved for treatment of type 2 diabetes and to reduce the risk of major adverse cardiovascular events in adults with type 2 diabetes and established cardiovascular disease, has reduced body weight significantly in patients with and without type 2 diabetes when given in addition to lifestyle intervention. Liraglutide (Saxenda), another GLP-1 receptor agonist, has been FDA-approved for chronic weight...

Ifosfamide (Ifex - Mead Johnson), a synthetic analog of cyclophosphamide (Cytoxan; and others), has been approved by the US Food and Drug Administration (FDA) for concurrent use with other drugs in third-line chemotherapy of metastatic germ-cell testicular cancer. Given concomitantly, a synthetic sulfhydryl compound known as mesna (Mesnex - Asta) acts in the urine to detoxify metabolites of ifosfamide that cause hemorrhagic cystitis (H Burkert, Cancer Treat Rev, 10 suppl A:175, 1983). Ifosfamide and mesna are available together from Bristol-Myers Oncology Division.

A 0.05% ophthalmic solution of levocabastine hydrochloride (Livostin - Iolab), a histamine H 1 -receptor antagonist, has been approved by the US Food and Drug Administration for treatment of seasonal allergic conjunctivitis. The new drug is a cyclohexylpiperidine derivative with no structural relationship to any other antihistamine.

Irbesartan (Avapro - Sanofi/Bristol-Myers Squibb) is the third angiotensin II receptor antagonist to become available in the USA for oral treatment of hypertension. Losartan (Cozaar) and valsartan (Diovan) were marketed earlier. Eprosartan (Teveten - SmithKline Beecham) has been approved by the FDA but not marketed.

Tamoxifen, anti-estrogen used for many years in the treatment of breast cancer, has now been approved by the FDA for reducing the incidence of breast cancer in women at increased risk of developing the disease.

The angiotensin receptor blocker (ARB) azilsartan medoxomil (Edarbi – Takeda) was recently approved by the FDA for oral treatment of hypertension, either alone or combined with other drugs. It is the eighth ARB approved for this indication.

The FDA has approved Nuedexta (Avanir), a fixed-dose combination of the cough suppressant dextromethorphan hydrobromide and the antiarrhythmic quinidine sulfate, for oral treatment of pseudobulbar affect. The combination is the first treatment approved by the FDA for this indication. Studies to support the effectiveness of Nuedexta were performed in patients with underlying amyotrophic lateral sclerosis (ALS) or multiple sclerosis (MS); the drug has not been shown to be safe or effective in other types of emotional lability.