The FDA is warning that the methylphenidate patch (Daytrana) can lead to permanent depigmentation of the skin (chemical leukoderma). The patch is used to treat ADHD. The area of depigmentation can range up to 20 cm (8 inches) in diameter. Chemical leukoderma is not physically harmful but can be disfiguring. The FDA is warning patients or caregivers to watch for areas of lighter skin, especially under the patch, and report it immediately to their healthcare provider.
The FDA has approved cangrelor, an intravenous antiplatelet drug that is used as an adjunct to percutaneous coronary intervention (PCI) to reduce the risk of periprocedural thrombotic events such as myocardial infarction (MI) or stent thrombosis. Approval was based on a trial of more than 10,000 patients undergoing PCI in which cangrelor was compared to clopidogrel. Cangrelor reduced the incidence of MI and the need for further PCI or stent thrombosis, but was also associated with significantly more serious bleeding events. Cangrelor is manufactured by the Medicines Company and marketed as Kengreal.
The FDA has approved a novel new drug to treat cystic fibrosis (CF). Lumacaftor/ivacaftor is approved for CF patients 12 years and older who have two copies (homozygous) of the F508del gene mutation, the leading cause of CF. Having two copies of the mutation (one from each parent) leads to disruption in how water and chloride are transported. The drug was given breakthrough status and priority review as well as designation as an orphan drug, all of which speed the approval process for the drug and reduce the cost of development. Approval was based on two double-blind, placebo-controlled trials of more than 1100 patients with CF and the F508del mutation. In both studies, patients who took lumacaftor/ivacaftor (2 pills every 12 hours) demonstrated improved lung function compared to those who took placebo. Lumacaftor /ivacaftor is manufactured by Vertex Pharmaceuticals and is marketed as Orkambi. The drug is expected to cost more than $250,000 per year.
The FDA has approved a new combination drug for the treatment of heart failure. The new drug combines valsartan, an angiotensin II receptor antagonist, and the new chemical entity sacubitril, which exerts its action by inhibiting neprilysin, an enzyme that degrades atrial and brain natriuretic peptide. Sacubitril/valsartan was studied in the PARADIGM-HF trial of more than 8000 patients with symptomatic heart failure and was shown to reduce the rate of cardiovascular death and hospitalization related to heart failure when compared to the ACE inhibitor enalapril. Most patients were also receiving other drugs for heart failure, including beta-blockers, diuretics, and mineralocorticoid antagonists. The most common side effects were hypotension, hyperkalemia, and renal impairment. Like valsartan and other ARBs and ACEIs, the drug was also associated with angioedema. Sacubitril/valsartan is manufactured by Novartis as Entresto. The New York Times reports that the drug will cost about $4500 per year, and Novartis is telling shareholders the drug could eventually achieve more than $5 billion in sales.