Perampanel (per am’ pa nel; Fycompa – Eisai), a first-in-class noncompetitive AMPA receptor antagonist, has been approved by the FDA for adjunctive treatment of partial-onset seizures in patients ≥12 years old. New drugs for epilepsy are often initially approved by the FDA as adjunctive treatment for partial seizures.

The FDA has approved a fixed-dose combination (Harvoni [har voe' nee] – Gilead) of sofosbuvir and ledipasvir (led' i pas' vir), two oral direct-acting antiviral agents, for treatment of chronic hepatitis C virus (HCV) genotype 1 infection. Genotype 1 is responsible for 70-80% of HCV infections in the US. Sofosbuvir (Sovaldi) was approved earlier for use in combination with other antiviral drugs for treatment of HCV infection. Ledipasvir is a new drug.

The FDA has approved eluxadoline (Viberzi – Actavis), a mu-opioid receptor agonist and delta-opioid receptor antagonist, for oral treatment of adults with irritable bowel syndrome with diarrhea (IBS-D).

The FDA has approved nusinersen (Spinraza – Biogen) for treatment of spinal muscular atrophy (SMA), a hereditary neurodegenerative disease that occurs in about one in every 10,000 births. It is the first drug to be approved in the US for this indication.

The oral cyclin-dependent kinase (CDK) 4/6 inhibitor ribociclib (Kisqali – Novartis) has been approved by the FDA for use in combination with an aromatase inhibitor for first-line endocrine-based therapy in postmenopausal women with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative advanced or metastatic breast cancer. Ribociclib is also available copackaged with the aromatase inhibitor letrozole (Femara, and generics) as Kisqali Femara Co-Pack. Ribociclib is the second CDK 4/6 inhibitor to be approved in the US for this indication;...

The FDA has approved Haegarda (CSL Bering), a human plasma-derived C1 esterase inhibitor (C1INH), for prophylaxis against hereditary angioedema (HAE) attacks in adolescents and adults. Haegarda is the fourth C1INH to be approved by the FDA and the second to be approved for HAE prophylaxis. Unlike the other C1INHs, Haegarda is self-administered subcutaneously rather than intravenously.

The FDA has approved an extended-release tablet formulation of amantadine (Osmolex ER – Vertical/Osmotica) for once-daily treatment of Parkinson's disease (PD) and drug-induced extrapyramidal symptoms (EPS) in adults. An extended-release capsule formulation of amantadine (Gocovri) was approved in 2017 for treatment of levodopa-induced dyskinesia in patients with PD.

The FDA has approved glucagon nasal powder (Baqsimi – Lilly) for treatment of severe hypoglycemia in patients ≥4 years old with diabetes. Baqsimi is the first noninjectable glucagon formulation to become available in the US. Injectable glucagon emergency kits (GlucaGen Hypokit, and generic) have been available for years, but they require reconstitution of the lyophilized powder by the caregiver immediately before injection. An injectable glucagon formulation that does not require reconstitution was recently approved by the FDA (Gvoke) and is expected to become available...

The FDA has approved a progestin-only oral contraceptive ("minipill") containing drospirenone (Slynd – Exeltis). All other progestin-only oral contraceptives available in the US contain norethindrone (Camila, and others). Progestin-only oral contraceptives are similar in efficacy to combination oral contraceptives. They are used predominantly by breastfeeding women and by those in whom estrogen is poorly tolerated or contraindicated. Combination oral contraceptives containing drospirenone and ethinyl estradiol have been available for years.

Caplacizumab-yhdp (Cablivi – Sanofi /Genzyme), a von Willebrand factor-directed antibody fragment, has been approved by the FDA for use in combination with plasma exchange and immunosuppressive therapy for treatment of immune-mediated thrombotic thrombocytopenic purpura (iTTP; formerly called acquired thrombotic thrombocytopenic purpura [aTTP]) in adults. It is the first drug to be approved in the US for this indication.