Drug Topics - December 12, 2008 - (Page 18)

Clinical ORPHAN-DRUG APPROVAL RX CARE Mona Nashed, PharmD, BCPS FDA approves Gamunex for treatment of neurologic disease Patients with chronic inﬂammatory demyelinating polyneuropathy (CIDP) ﬁnally have a treatment option that has been recognized by the U.S. Food and Drug Administration (FDA). Gamunex, an intravenous immune globulin (IVIG) pooled from human blood, recently received FDA approval for the treatment of CIDP. The product was previously approved for primary humoral immunodeﬁciency disease (PI) and idiopathic thrombocytopenic purpura (ITP). Although various immune globulins have been used over the years to treat CIDP on an off-label basis, Gamunex is the ﬁrst product to receive FDA approval as an orphan drug for this indication. The orphan-drug designation provides manufacturers with ﬁnancial incentives to develop treatments for rare diseases, deﬁned as those affecting fewer than 200,000 people in the United States. CIDP is a rare autoimmune disorder characterized by progressive weakness and impaired sensory function in the legs and arms. The disorder, also known as chronic relapsing polyneuropathy, is caused by damage to the myelin sheath (the fatty covering that protects nerve ﬁbers) of the peripheral nerves. CIDP is closely related to Guillain-Barré syndrome and is considered the chronic counterpart of that acute disease. Although it is typically more prevalent in young adults and in men, it can strike at any age and in either gender. The course of CIDP varies widely among patients, but many patients are left with residual numbness, weakness, fatigue, and other symptoms which can lead to reduced quality of life. CIDP typically goes unrecognized and untreated due to its heterogeneous presentation and diagnostic limitations. However, early treatment is crucial in preventing irreversible loss of nerve axons and improving functional recovery. Traditional treatment options for CIDP have included corticosteroids, which may be prescribed alone or in combination with immunosuppressant drugs, plasmapheresis, and IVIG. Theories about how to treat this condition include the hypothesis that Gamunex helps improve muscle function in patients with CIDP by modulating the immune system’s response to the inﬂammation that damages the myelin sheaths, but the exact mechanism remains unknown. “Research in this ﬁeld is lacking, and the approval of Gamunex signals a step forward for patients suffering from CIDP,” said Tarun Verma, PharmD, BCPS, Pharmacy Clinical Manager of Florida Hospital-Fish Memorial. Gamunex was approved for administration as a total loading dose of 2 g/ kg, given in divided doses over two to four consecutive days. For maintenance infusions, Gamunex may be administered either over one day at a dose of 1 g/kg or divided into two doses of 0.5 g/kg over two consecutive days. The dose is typically repeated every three weeks. Gamunex, which is available Network Clinical SEE INDEX PAGE 8 For more info on this topic, see www.drugtopics.com MONA NASHED is a writer and hospital pharmacist in New Jersey. W W W.D R U GTO P I C S .C O M 18 DRUG TOPICS D E C . 15, 2008 GETTY IMAGES / PHOTOALTO / LAURENCE MOUTON as a 10 percent liquid, is incompatible with saline but can be diluted with 5 percent dextrose in water (D5W). Side effects reported most frequently by patients were headache, fever, increased blood pressure, rash, joint pain, chills, back pain, nausea, and lightheadedness. Similar to other licensed IGIV products, Gamunex holds a black box warning about renal dysfunction, acute renal failure, osmotic nephrosis, and death in predisposed patients. Therefore, blood urea nitrogen, serum creatinine, and urine output should be closely monitored in patients at risk of developing renal failure. Renal dysfunction and acute renal failure have been reported more frequently in patients receiving IGIV products containing sucrose, which is typically used as a stabilizer. http://WWW.DRUGTOPICS.COM http://WWW.DRUGTOPICS.COM

Table of Contents Feed for the Digital Edition of Drug Topics - December 12, 2008

Drug Topics - December 12, 2008 Contents Up Front Up Front In Depth Letters Health-System Edition Cover Story: Controlled Substance Disposal a Growing Problem for Hospitals Pharmacists Hope for Speedy Approvals Deaths from C. Difficile Increasing PharmMD Teams with HealthSpring RX Care Commentary Ohio Hospital Automates Pharmacy The Business Outlook for 2009 OTC The Pharmacist's Duty to Warn: Implications for Pharmacy Practice New Products Viewpoint

Drug Topics - December 12, 2008

For optimal viewing of this digital publication, please enable JavaScript and then refresh the page. If you would like to try to load the digital publication without using Flash Player detection, please click here.