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Notable FDA Approvals: Jan. 2018

Jan 31, 2018

Spark Therapeutics’ Luxturna (voretigene neparvovec-rzyl) is the first gene therapy the FDA has approved for treatment of a disease caused by mutations in a specific gene. The therapy is indicated for patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy, an inherited form of vision loss that can lead to blindness. Luxturna is a one-time treatment that uses a modified adeno-associated virus to deliver a normal copy of the RPE65 gene into the patient’s retinal cells, after which the cells produce a protein that restores vision. The cost of treatment for both eyes is $850,000. According to FiercePharma, Spark is making outcomes-based payment arrangements with Harvard Pilgrim and Express Scripts affiliates and is developing a proposal with CMS that would allow payments over multiple years.

Merck & Co. and Pfizer’s Steglatro (ertugliflozin) has been approved to improve glycemic control in adults with type 2 diabetes. The drug is approved as a monotherapy in 5 mg and 15 mg tablet form, and as part of two fixed-dose combinations—Steglujan, which combines ertugliflozin with Merck’s Januvia (sitagliptin), and Segluromet, which combines ertugliflozin with metformin. Steglatro is the fourth approved drug in a class known as SGLT2 inhibitors. As such, it will compete with Janssen’s Invokana (canagliflozin), AstraZeneca’s Farxiga (dapagliflozin) and Eli Lilly and Boehringer Ingelheim’s Jardiance (empagliflozin). The list prices are $8.94 per day for Steglatro and Segluromet and $17.45 per day for Steglujan.

AstraZeneca and Merck’s Lynparza (olaparib) is the first drug to receive FDA approval as a treatment for patients with metastatic breast cancer who have a specific BRCA gene mutation. An estimated 10 percent to 15 percent of all patients with breast cancer have a BRCA mutation, according to the FDA. Specifically, Lynparza is approved for use in patients with deleterious or suspected deleterious germline BRCA-mutated, HER2-negative metastatic breast cancer who have previously been treated with chemotherapy. A companion diagnostic, Myriad Genetic Laboratories’ BRACAnalysis CDx, has also received expanded FDA approval for the new indication.

The drug, a PARP inhibitor, was already approved as a treatment for germline BRCA-mutated advanced ovarian cancer in patients who have previously been treated with at least three lines of chemotherapy. It is also indicated for maintenance use in patients with recurrent epithelial ovarian, fallopian tube or primary peritoneal cancer whose tumors have completely or partially responded to chemotherapy.

Last July, AstraZeneca and Merck & Co. signed a development and commercialization agreement for Lynparza that could be worth as much as $8.5 billion.

Novartis received approval from the FDA for Lutathera (lutetium Lu 177 dotatate), a first-in-class radioligand therapy that is indicated for adults with somatostatin receptor-positive gastroenteropancreatic neuroendocrine tumors (GEP-NETs), a type of cancer that affects the pancreas or gastrointestinal tract. According to the FDA, GEP-NETs are diagnosed in approximately 1 out of every 27,000 people in the U.S. each year. Lutathera binds to a tumor cell’s somatostatin receptor and then enters the cell, where it allows radiation to damage the cell. Novartis gained Lutathera with the acquisition of Advanced Accelerator Applications last fall for $3.9 billion.

The FDA approved Baxter International’s ready-to-use bivalirudin in 0.9 percent sodium chloride injection. The drug is an anticoagulant used for patients who are undergoing percutaneous coronary intervention. Bivalirudin is available in multiple generic formulations and is marketed by The Medicines Company under the brand name Angiomax, but according to Baxter, its version of bivalirudin is “the first and only available in a convenient frozen premixed solution.” Baxter said its premixed bivalirudin would be available early this year in two dosages: 250 mg per 50 mL, and 500 mg per 100 mL.


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