FDA Update: September 2017

Sep 12, 2017
Express Scripts Office of Clinical Evaluation and Policy tracks some recent updates to the drug pipeline.

New Pill Bottle

The Emerging Therapeutics team took note of the following new drug, new indication and new generic approvals, last month:

First CAR-T Therapy, Kymriah, Approved

On Aug. 30, 2017, the U.S. Food and Drug Administration (FDA) approved Kymriah™ (tisagenlecleucel – Novartis), the first gene therapy approved for use in the U.S. It is personalized treatment for patients under the age of 25 years who have B-cell acute lymphoblastic leukemia (ALL) that has relapsed at least twice or no longer responds to standard treatments. In a specialized laboratory, genes containing a chimeric antigen receptor (CAR) are attached to T-cells harvested from each individual patient. When the modified CAR-T cells are infused back into the patient, they seek out CD19 (a protein overexpressed by ALL cells), attach to it and destroy the cancer cells. Kymriah will be administered as a one-time intravenous (IV) infusion only at certified treatment centers that have staff members trained to collect the T-cells, give the infusions and recognize signs of adverse reactions. Novartis announced that it will price Kymriah at $475,000, but will adjust cost according to outcomes for patients who respond to treatment in the first month. Kymriah will be delivered directly to the facilities where it is given, not through pharmacies. Prescribing information is available here.

New Form of Pitavastatin Approved

Lupin’s Nikita™ (pitavastatin sodium) tablets were approved by the FDA on Aug. 4, 2017. It will be used, in combination with diet changes and exercise, to regulate cholesterol, lipoprotein and triglyceride levels for patients who have primary hyperlipidemia or mixed dyslipidemia. Nikita was approved through FDA’s 505(b)(2) pathway, allowing the submitting company to use documentation from a similar product that already is FDA approved rather than requiring a repeat of the clinical trials. In this case, the reference product is Livalo® (pitavastatin calcium – Kowa Company). The two drugs, however, are not A-rated or automatically interchangeable. Livalo currently has patents protecting it from direct generic competition until at least the end of 2020. Recommended dosing for Nikita is once daily. It will be manufactured in 1mg, 2mg and 4mg tablets. Complete prescribing information can be found here.

Generic Approved for Fosrenol

Natco Pharma announced on Aug. 16, 2017, that the FDA has approved its generics for Shire’s Fosrenol® (lanthanum carbonate). The drug reduces hyperphosphatemia (high blood phosphate levels) for patients with end-stage renal disease (ESRD). Recommended dosing is 1,500mg to 3,000mg per day divided into three doses and taken with food. Although Natco’s generic is the first to be FDA approved, its exclusivity status currently is unclear. According to QuintilesIMS, sales for Fosrenol amounted to approximately $122 million for the year ending on June 30, 2017. Natco plans to launch lanthanum carbonate 500mg, 750mg and 1,000mg tablets shortly and market it in a partnership with Lupin Pharmaceuticals.

Besponsa Approved for Acute Lymphoblastic Leukemia

On Aug. 17, 2017, Pfizer received FDA approval of Besponsa® (inotuzumab ozogamicin) for the treatment of adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL). Besponsa is administered as a one-hour intravenous (IV) infusion. Recommended dosing for the first cycle is 0.8mg/m2 on Day 1, 0.5mg/m2 on Day 8 and 0.5mg/m2 on Day 15. Dosing for subsequent cycles varies based on response to therapy. Pfizer is launched Besponsa on Aug. 21, 2017 through open distribution. Full prescribing information can be found here.

New Approvals for Lynparza

The FDA granted full approval to Lynparza® (olaparib – AstraZeneca/Merck) on Aug. 17, 2017, for treating patients with BRCA mutated (gBRCAm) advanced ovarian cancer that has been treated with chemotherapy (chemo) at least three times. Lynparza is a poly ADP-ribose polymerase (PARP) inhibitor, which prevents cancer cells from repairing chemo-caused damage to their DNA. The original indication, granted under accelerated approval, has now been finalized following successful results from further clinical studies. In addition, FDA approved a new indication – for maintenance treatment of patients who have recurrent epithelial ovarian, fallopian tube or primary peritoneal cancers. Treatment is not dependent on BRCA status, but patients must have a complete or partial response after platinum-based chemo. FDA also approved a new oral tablet dosage form (in 100mg and 150mg strengths), which was launched immediately after its approval. Although Lynparza’s capsule form will be discontinued gradually, it is not directly interchangeable with the tablets. Recommended dosing of the tablets for both indications is 300mg taken twice a day. Full prescribing information for Lynparza tablets is available here.

Gocovri Approved for Parkinson’s Disease

On Aug. 24, 2017, the FDA approved Adamas Pharmaceuticals’ Gocovri™ (amantadine) extended-release capsules for treating dyskinesia (involuntary muscle movement), in patients receiving levodopa-based therapy for Parkinson’s disease. Levodopa is a standard treatment of Parkinson’s disease, but it can cause levodopa-induced dyskinesia (LID). Approximately 200,000 people, or 20% of the estimated 1 million Parkinson’s disease patients in the U.S., suffer from LID. Administered once-daily at bedtime, Gocovri dosing is 137mg per day for one week, and then increased to the recommended daily dose of 274mg. It is an extended-release formulation of generically-available amantadine, which has been used off-label to treat Parkinson’s dyskinesia for many years. According to Adamas, the new extended-release properties allow for consistently high levels of amantadine throughout the day. Adamas plans on making Gocovri 68.5mg and 137mg capsules available in the fourth quarter of 2017 with a formal product launch in Jan 2018. Prescribing information is available here.

FDA Approves Cyltezo, a Second Biosimilar to Humira

On Aug. 25, 2017, the FDA approved Boehringer Ingelheim’s Cyltezo™ (adalimumab – adbm), a biosimilar to AbbVie’s Humira®. Like the previously approved Humira biosimilar, Amjevita™ (adalimumab-atto – Amgen), Cyltezo is approved for treating adults with rheumatoid arthritis, plaque psoriasis, psoriatic arthritis, ankylosing spondylitis, Crohn’s disease and ulcerative colitis. Both also are approved for treating children four years of age and older who have polyarticular juvenile idiopathic arthritis. The recommended dose of Cyltezo varies depending on the indication. While Boehringer Ingelheim’s launch plans for Cyltezo are unknown, its release could be delayed for two or more years due to patent litigation. Full prescribing information can be found here.

Expanded Indication for Victoza

Novo Nordisk announced on Aug. 25, 2017, that the FDA had approved Victoza® (liraglutide) to decrease the risk of major adverse cardiovascular (CV) events for adults who have both type 2 diabetes and documented CV disease. A glucagon-like peptide-1 (GLP-1) receptor agonist, it first was FDA approved in 2010, along with diet changes and exercise, to improve blood sugar control for adults with type 2 diabetes. Recommended dosing is 0.6mg, 1.2mg or 1.8mg once a day delivered subcutaneously (SC) through a pre-filled, multi-dose pen-like device. Victoza is known to cause some types of thyroid tumors, so it carries a boxed warning against its use by patients or close relatives of patients who have medullary thyroid carcinoma (MTC) or multiple endocrine neoplasia syndrome type 2 (MEN2). Complete prescribing information is available here.

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