Expanded approval for cystic fibrosis drug
The FDA has expanded the approved use of the fixed-dose combination of lumacaftor and ivacaftor (Orkambi), the cystic fibrosis (CF) treatment, to children ages 6 to 11 years who have two copies of the F508del mutation in the CF transmembrane conductance regulator gene. The drug was approved last year for children age 12 and older with CF who have two copies of the F580del mutation, marking the first time a drug for CF was approved to address the underlying cause of the disease for individuals with this mutation.
The most common adverse reactions associated with Orkambi include dyspnea, nasopharyngitis, nausea, diarrhea, upper respiratory tract infection, rash, flatulence, and fatigue.
The FDA's approval of the expanded indication was based on data from an open-label phase 3 clinical safety study presented at the 39th European Cystic Fibrosis Society Conference in June 2016.
FDA approves new biologic for Crohn disease
The FDA approved ustekinumab (Stelara), a biologic, for the treatment of moderately-to-severely active Crohn disease in adults age 18 and older. The drug is indicated in patients who have failed or were intolerant of treatment with immunomodulators or corticosteroids but who never failed treatment with a tumor necrosis factor (TNF) blocker (or who failed or were intolerant of treatment with one or more TNF blockers). Ustekinumab is the first biologic approved for Crohn disease that targets interleukin-12 and interleukin-23 cytokines, which play key roles in inflammatory and immune responses.
Ustekinumab is the only medication for Crohn disease that begins with a weight-based, one-time I.V. infusion induction dose to help reduce symptoms (followed by 90-mg subQ maintenance injections every 8 weeks to help keep symptoms under control). The induction I.V. dose is administered under the supervision of a healthcare provider; subsequent maintenance doses are administered every 8 weeks either by a healthcare provider or self-injected by the patient after adequate training.
In clinical trials of patients who were either new to, experienced with, or failed biologic therapy, between 34% and 56% of patients experienced relief from their Crohn disease symptoms in 6 weeks after receiving the one-time I.V. infusion of ustekinumab, and noticeable improvement was seen as early as 3 weeks. Most of those who responded to induction dosing and continued treatment with ustekinumab subQ maintenance doses every 8 weeks were in remission at the end of 44 weeks.
Ustekinumab may lower the ability to fight infections and increase the risk of infections. Serious allergic reactions to ustekinumab may occur.
FDA approves Invokamet XR for type 2 diabetes
Invokamet extended-release (XR), a once-daily, fixed-dose combination therapy of a sodium-glucose cotransporter 2 (canagliflozin) and a biguanide (metformin hydrochloride XR), has been approved for first-line use as an adjunct to diet and exercise to improve blood glucose control in adults with type 2 diabetes mellitus when treatment with the two medications is appropriate. Invokamet XR offers the convenience of once-daily dosing and flexibility for tailoring treatment to the needs of patients, especially those with higher A1C levels.
Clinical studies of Invokamet XR show that the combination of canagliflozin and metformin reduces A1c significantly more than metformin alone, sitagliptin plus metformin, or glimepiride plus metformin.
Common adverse reactions associated with canagliflozin include frequent urination, female genital mycotic infections, and urinary tract infection. Common adverse reactions associated with metformin include diarrhea, nausea, vomiting, flatulence, asthenia, indigestion, abdominal discomfort, and headache. Invokamet XR contains a boxed warning concerning metformin-associated lactic acidosis. Invokamet XR should not be given to individuals with type 1 diabetes mellitus or diabetic ketoacidosis.