Monthly News Roundup - December 2017
Luxturna Gene Therapy OK’d for Rare Form of Vision Loss
A historic approval, Luxturna (voretigene neparvovec-rzyl) from Spark Therapeutics is the first directly administered gene therapy that targets a disease caused by specific gene mutations. In December the U.S. Food and Drug Administration (FDA) approved Luxturna, used in patients with biallelic RPE65-mediated inherited retinal disease (IRD), a form of vision loss that can lead to blindness. Luxturna is an adeno-associated viral (AAV) vector gene therapy given by subretinal Injection to directly deliver a normal copy of the RPEE65 gene. In a Phase III study, patients had significant improvements in completing an obstacle course at low light levels as compared to the control group.
FDA Clears Giapreza for Dangerous Low Blood Pressure
The FDA has approved Giapreza (angiotensin II) for intravenous infusion to increase blood pressure in adults with septic or other distributive shock. Giapreza, from La Jolla Pharmaceutical, is a synthetic human angiotensin II vasoconstrictor. In studies of 321 patients with shock and a critically low blood pressure, significantly more patients responded to treatment with Giapreza compared to placebo. Giapreza increased blood pressure when added to conventional treatments (fluids and vasopressor therapy such as catecholamines and/or vasopressin) also used to raise blood pressure. Giapreza can cause dangerous blood clots with serious consequences; prophylactic treatment for blood clots should be used.
Rhopressa Approved for Open-Angle Glaucoma or Ocular Hypertension
Glaucoma is often caused by an abnormally high pressure in the eye due to fluid build-up that can damage the optic nerve and lead to blindness. The FDA has now approved Rhopressa (netarsudil ophthalmic solution) once daily eye drops to lower elevated intraocular pressure (IOP) in patients with open-angle glaucoma or ocular hypertension. From Aerie Pharmaceuticals, Rhopressa is a Rho kinase inhibitor, and reduces IOP by increasing the outflow of aqueous humor (the fluid inside the eye). Rhopressa is expected to be available on the market in the second quarter of 2018.
Novo Nordisk Gains FDA Approval of Ozempic for Type 2 Diabetes
Type 2 diabetes affects more than 28 million people in the U.S. In response, the FDA has approved Novo Nordisk’s Ozempic (semaglutide), a glucagon-like peptide-1 (GLP-1) analog administered once-weekly for the treatment of adults with type 2 diabetes. Ozempic is approved for use in two doses, 0.5 mg and 1 mg, and will be launched in the Ozempic pre-filled pen. In Phase 3 studies, Ozempic showed significant reductions in A1c compared with placebo, sitagliptin (Januvia) and exenatide extended-release (Bydureon); weight loss was also seen. The most common side effects include nausea, vomiting, diarrhea, abdominal pain and constipation.
FDA Approves Three Ertugliflozin-Containing Medicines for Type 2 Diabetes
Patients with type 2 diabetes often need a combination of medications to control their blood sugar. This month, the FDA approved Pfizer and Merck’s new SGLT2 inhibitor Steglatro (ertugliflozin), as well as two new combination agents that contain ertugliflozin: Segluromet (ertugliflozin with the biguanide metformin), and Steglujan (ertugliflozin with the dipeptidyl peptidase-4 inhibitor sitagliptin). In Phase 3 studies, Steglatro was studied as monotherapy (alone) and in combination with metformin and/or sitagliptin, as well as with insulin and a sulfonylurea. Steglatro on top of metformin plus sitagliptin resulted in significant A1C reductions of 0.7% and 0.8% compared with 0.2% for placebo, and weight loss of roughly 6.2 to 6.6 pounds.
Posted: December 2017