Sapropterin (Kuvan - BioMarin) has been approved by the FDA to reduce phenylalanine plasma concentrations in patients with phenylketonuria (PKU). PKU is a rare genetic disorder (it occurs in 1 in 8000 Caucasian and 1 in 50,000 African-American newborns) caused by a deficiency of phenylalanine hydroxylase (PAH) the resulting high levels of the amino acid in blood and urine lead to cognitive, behavioral and other neurological complications.

Linagliptin (Tradjenta – Boehringer Ingelheim/Lilly), a third oral dipeptidyl peptidase-4 (DPP-4) enzyme inhibitor, was recently approved by the FDA for treatment of type 2 diabetes, either alone or in combination with metformin, a sulfonylurea or pioglitazone

The FDA has approved a fixed-dose combination of the integrase strand transfer inhibitor (INSTI) elvitegravir, the pharmacokinetic enhancer cobicistat, and the nucleoside/nucleotide reverse transcriptase inhibitors (NRTIs) emtricitabine and tenofovir disoproxil fumarate (Stribild – Gilead) for treatment of HIV-1 infection in antiretroviral treament-naïve adults. Elvitegravir is the second INSTI to be approved by the FDA for use in HIV treatment; raltegravir (Isentress) was the first.

The FDA has approved two new oral kinase inhibitors for treatment of unresectable or metastatic melanoma: dabrafenib (Tafinlar – GSK) for melanomas with BRAF V600E mutations and trametinib (Mekinist – GSK) for melanomas with either BRAF V600E or V600K mutations. Dabrafenib is not recommended for patients with wild-type BRAF (BRAF-negative) melanoma, and trametinib is not recommended for patients who have received prior BRAF-inhibitor therapy.

The FDA has approved vortioxetine (vor" tye ox' e teen; Trintellix – Takeda/Lundbeck), a new serotonergic drug, for treatment of major depressive disorder. Like vilazodone (Viibryd), another serotonergic antidepressant, it has been claimed to have a low incidence of sexual side effects and no significant effect on weight.

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;...