FDA Update: March 2017

Mar 7, 2017
Express Scripts Office of Clinical Evaluation and Policy tracks some recent updates to the drug pipeline.
  • Immune Deficiency
  • Orphan Drugs
  • Inflammatory Conditions

New Pill Bottle

Over the past month, Express Scripts’ Emerging Therapeutics team focused on four new drug approvals:

Gammaplex 10% Approved for Immune Deficiencies

On February 6, the U.S. Food and Drug Administration (FDA) approved Gammaplex® 10% (immune globulin intravenous [human], 10% liquid). It is indicated to treat adults who have primary immunodeficiency (PI) or chronic immune thrombocytopenic purpura (ITP). The manufacturer, Bio Products Laboratory, also markets Gammaplex® 5% (immune globulin intravenous [human], 5% liquid in the U.S. Intravenous immunoglobulins (IVIGs), are used as replacement for patients who have immunoglobulin deficiencies. They are dosed by weight. For treating PI, one Gammaplex 10% infusion is recommended every three to four weeks. Recommended dosing for the treatment of ITP is one infusion on each of two successive days when needed to increase platelet count. In clinical studies, the average infusion time for the 10% strength was about one hour – approximately one-half the average time for an infusion of Gammaplex 5%. Labeling for all IVIG products includes warnings that they may cause blood clots, kidney damage or kidney failure. Complete prescribing information for Gammaplex 10% can be found here.

Parsabiv Approved to Treat Secondary Hyperparathyroidism

Amgen announced on February 7, that the FDA had approved Parsabiv™ (etelcalcetide). It is indicated to treat hyperparathyroidism (HPT) caused by hemodialysis for adults with chronic kidney disease (CKD). Secondary HPT is a condition that results from over production of parathyroid hormone (PTH) from the parathyroid glands, which are stimulated by low kidney function. In response, additional calcium and other minerals are lost from bones into the blood. Parsabiv acts like calcium at receptors on the parathyroid glands to decrease PTH release. Given as an intravenous (IV) infusion, it can be injected into the tubing used for dialysis after each dialysis treatment ends. Recommended initial dose is 5mg three times a week. Maintenance doses range from 2.5mg to 15mg three times a week as long as calcium blood levels are too high. Prescribing information is available here.

Emflaza Approved for Duchenne Muscular Dystrophy

On February 9, the FDA approved Marathon Pharmaceuticals’ Emflaza™ (deflazacort). It is indicated to treat patients who have Duchenne muscular dystrophy (DMD) and who are five years of age and older. Emflaza is an oral corticosteroid that decreases both immune and inflammatory activity to relieve symptoms of DMD and also to delay progression of the disease. The recommended dose is 0.9mg/kg once daily. Emflaza is expected to be launched in four to six weeks. Full prescribing information can be found here.

Siliq Approved for Psoriasis

Siliq™ (brodalumab), a monoclonal antibody that selectively binds to the interleukin (IL)-17 receptor, was approved by the FDA on February 15. It is indicated to treat adults who have moderate-to-severe plaque psoriasis. Siliq should be used in patients who are eligible for systemic (oral or injected) treatment or phototherapy, but already have tried systemic treatments that either stopped working or did not work at all. Recommended dosing is one subcutaneous (SC) injection (210mg) once a week for three doses, and then once every two weeks. Siliq has a boxed warning that it may cause patients to consider or try suicide. Its use will be regulated under a Risk Evaluation and Mitigation Strategy – Siliq REMS. Patients, physicians and pharmacists all must be authorized before using, prescribing or dispensing Siliq. Valeant plans on launching Siliq in the second half of 2017. Complete prescribing information is available here.

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