Vyalev (Abbvie), a solution for continuous subcutaneous infusion containing the prodrugs foscarbidopa and foslevodopa, has been approved by the FDA for treatment of motor fluctuations in adults with advanced Parkinson's disease (PD). Vyalev is the first subcutaneously administered product to become available in the US for treatment of PD symptoms. Duopa, a carbidopa/levodopa enteral suspension infused via a nasojejunal tube or percutaneous gastrostomy, was approved in for the same indication 2015.

The dopamine agonist pramipexole (Mirapex - Boehringer Ingelheim) is the second drug approved by the FDA for treatment of moderate to severe restless legs syndrome (RLS). Ropinirole (Requip), another dopamine agonist, was approved for RLS in 2005. Both of these drugs were first approved for treatment of Parkinson's disease.

The FDA has approved erenumab-aooe (Aimovig – Amgen/Novartis), a once-monthly, subcutaneously-injected, monoclonal antibody against the calcitonin gene-related peptide receptor, for preventive treatment of migraine in adults. It is the first drug in its class to be approved by the FDA.

The FDA has approved pitolisant (Wakix – Harmony), a histamine-3 (H₃)-receptor antagonist/inverse agonist, for treatment of excessive daytime sleepiness (EDS) or cataplexy in adults with narcolepsy. It is the first H₃-receptor antagonist/inverse agonist to become available in the US and the first non-controlled substance to receive FDA approval for these indications. Pitolisant has been available in Europe since 2016.

Atrial fibrillation (AF) is the most common arrhythmia in the world. Risk factor modification, anticoagulation, rhythm control, and rate control are the four pillars of its management. American College of Cardiology/American Heart Association (ACC/AHA) guidelines on management of AF were updated recently.

The FDA has approved a 0.016% gel formulation of the nitrogen mustard mechlorethamine (Valchlor – Actelion) for second-line topical treatment of patients with stage IA or IB mycosis fungoides, the most common type of cutaneous T-cell lymphoma. Topical nitrogen mustard has been used off-label for decades for this indication, usually as a pharmacy-compounded ointment, but no clinical trials evaluating its efficacy and safety have been done. Mechlorethamine is also available in an injectable formulation (Mustargen – Recordati) for the same and other indications.FDA approval of Valchlor was...

In 2007, an FDA advisory committee asked that placebo-controlled, dose-ranging trials be conducted to establish the efficacy of the oral decongestant phenylephrine (Sudafed PE, and others), which is sold over the counter (OTC) as a single agent and in combination with other drugs for treatment of cold and allergy symptoms. Phenylephrine replaced pseudoephedrine (Sudafed, and others) in many OTC formulations when access to pseudoephedrine-containing products was restricted in an effort to reduce their use in the synthesis of methamphetamine.CLINICAL STUDIES — In a randomized, open-label,...

The FDA has approved Lonsurf (Taiho Oncology), a combination of the thymidine-based nucleoside analog trifluridine and the thymidine phosphorylase inhibitor tipiracil, for oral treatment of metastatic colorectal cancer. Trifluridine is incorporated into DNA, interfering with DNA synthesis and inhibiting cell proliferation. Tipiracil inhibits the metabolism of trifluridine. The combination is only approved for use in patients who were previously treated with a fluoropyrimidine (fluorouracil or capecitabine), oxaliplatin, irinotecan, an anti-VEGF biological such as bevacizumab, and, if the tumor...

The FDA has approved an oral suspension formulation of nitisinone (Orfadin – Sobi) for treatment of hereditary tyrosinemia type 1 (HT-1). Orfadin has been available in a capsule formulation in Canada since 1994 and in the US since 2002.HT-1 is an autosomal recessive genetic disorder (estimated birth prevalence: 1:100,000) caused by mutations in the gene responsible for formation of fumarylacetoacetate hydrolase (FAH), which catalyzes the last step in tyrosine metabolism. FAH deficiency results in accumulation of upstream toxic metabolites, causing liver and kidney failure and developmental...

The FDA has approved adjunctive treatment with Dartisla ODT (Edenbridge), a new orally disintegrating tablet (ODT) formulation of the anticholinergic drug glycopyrrolate, to reduce symptoms of a peptic ulcer. Glycopyrrolate oral tablets (Robinul, Robinul Forte, and generics) were approved for the same indication in 1961.