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New Drugs/Drug News/New Medical Devices October 2011
Xalkori and a Diagnostic Test For Lung Cancer
A new treatment, crizotinib (Xalkori, Abbott), has been approved for patients with non–small cell lung cancer (NSCLC) who express the abnormal anaplastic lymphoma kinase (ALK) gene. The FDA also approved a companion test, the Vysis ALK Break Apart FISH Probe kit, to determine whether a patient carries the ALK gene. This is the second similarly targeted therapy approved by the FDA this year.
The ALK gene abnormality is involved in cancer development and growth. From 1% to 7% of patients with NSCLC have this genetic abnormality, and these patients are typically nonsmokers. Xalkori works by blocking certain proteins (kinases), including the protein produced by the abnormal ALK gene. The tablets are taken twice a day as monotherapy.
Source: FDA, August 31, 2011
Generic Furadantin for UTIs
Prasco Laboratories and Shionogi Inc. have agreed to market and distribute Nitrofurantoin Oral Suspension, the authorized generic version of Furadantin Oral Suspension. This antibiotic is used to treat urinary tract infections. The product is AB-rated and is therapeutically equivalent to and can be substituted for Furadantin.
Firazyr Relieves Attacks Of Hereditary Angioedema
Icatibant (Firazyr, Shire), an injectable drug, is now approved to treat acute attacks of hereditary angioedema (HAE). This is the first medication available in the U.S. that patients with this rare genetic condition can administer themselves. HAE affects up to 8,000 people in the U.S.
Patients with HAE experience periodic, painful attacks of severe swelling in the hands, feet, face, abdomen, and sometimes the throat; swelling in the throat can cause airway restriction. HAE is caused by a deficiency in the C1 esterase enzyme, which regulates inflammatory and coagulation responses. Icatibant is portable and can be stored at room temperature.
Ecallantide (Kalbitor, Dyax) and Berinert (C1 esterase inhibitor, human, Behring) are also approved to treat HAE, but they must be given in a medical setting. Cinryze (C1 esterase inhibitor, human, ViroPharma/Lev) can be self-administered, but it is used prophylactically and is not approved to manage acute attacks.
Icatibant is featured in this month’s Pharmaceutical Approval Update column, page
Source: MedPage Today, August 25, 2011,
Botox Improves Bladder Control
The FDA has approved Allergan’s Botox for treating overactive bladder. The product can be injected into the bladder to treat patients who lose bladder control because of damage to the nervous system as a result of conditions such as multiple sclerosis and spinal cord injury. A single injection relaxes the bladder and increases its storage capacity. The active ingredient, a toxin, blocks nerve signals. In clinical studies, the injections decreased episodes of urinary incontinence for up to 9 months.
Sources: Reuters and Medical
Prolia for Chemotherapy-Induced Bone Loss
Amgen’s denosumab (Prolia) has now been approved (1) to increase bone mass in women with a high risk for fracture who are receiving adjuvant aromatase inhibitor therapy for breast cancer and (2) to increase bone mass in men at high risk for fracture receiving androgen-deprivation therapy for nonmetastatic prostate cancer. Denosumab is the first FDA-approved therapy for bone loss in patients undergoing hormone ablation therapy.
Aromatase inhibitors are often used to prevent breast cancer recurrence, and androgen-deprivation therapy is often used to prevent or control recurrent prostate cancer. However, these treatments reduce hormone levels, leading to bone loss and an increased risk of fracture.
The expanded indications were based on two phase 3 clinical trials. Denosumab specifically targets the RANK ligand, which regulates osteoclast production.
Denosumab is approved in the U.S. for postmenopausal women with osteoporosis who are at a high risk for fracture. It is given as a single subcutaneous injection of 60 mg once every six months.
Source: Amgen, September 19, 2011,
Soliris, an Orphan Drug For Rare Blood Disorder In Children
Eculizumab (Soliris, Alexion) has been approved for patients with atypical hemolytic uremic syndrome (aHUS). This rare, chronic blood disease can lead to renal failure; it is also associated with an increased risk of death and stroke.
Atypical HUS accounts for 5% to 10% of all cases of hemolytic uremic syndromes. The disease disproportionately affects children.
Eculizumab, a targeted therapy, inhibits proteins that play a role in aHUS. The FDA first approved this drug in March 2007 to treat paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder that can lead to disability and premature death. No other approved treatments are available for aHUS.
The drug’s safety and effectiveness were established in two single-arm trials involving 37 adults and adolescents and in one retrospective study involving 19 children and 11 adults. Treated patients experienced improvements in kidney function, platelet counts, and other blood parameters.
Common side effects included hypertension, diarrhea, headache, anemia, vomiting, nausea, upper respiratory and urinary tract infections, and leukopenia.
The new indication has been approved with an extension of the existing Risk Evaluation and Mitigation Strategy (REMS) to inform health care professionals and patients about the known risk of life-threatening meningococcal infections. Eculizumab will continue to be available only through a restricted program, and prescribers must enroll in a registration program and provide a medication guide to patients who receive the drug.
Eculizumab was reviewed under the FDA’s priority review program.
Source: FDA, September 23, 2011
Remicade for Ulcerative Colitis in Children
Infliximab (Remicade, Janssen Biotech) is now approved to treat moderately to severely active ulcerative colitis (UC) in children older than 6 years of age who have not responded to conventional therapy. This biologic agent is used to reduce signs and symptoms of UC and to induce and maintain clinical remission in these patients.
UC is a form of inflammatory bowel disease (IBD). Between 50,000 and 100,000 children in the U.S. have IBD; of these children, 40% have UC.
Infliximab, a tumor necrosis factor (TNF) inhibitor, suppresses the immune system by blocking the activity of TNF, which can cause inflammation and lead to autoimmune disease.
Infliximab is also approved for the treatment of Crohn’s disease in adults and children older than 6 years of age; rheumatoid arthritis, ankylosing spondylitis; psoriatic arthritis; and plaque psoriasis in adults.
A boxed warning mentions the risk of infections and cancer. There have been cases of unusual cancers, including hepatosplenic T-cell lymphoma, reported in adolescents and young adults who used TNF-blocking agents.
Children should have received all of their scheduled vaccinations before starting treatment with infliximab. They should not receive live vaccines while taking infliximab.
Common adverse effects of infliximab include worsening of UC, upper respiratory infections, infusion-related reactions, and headache.
FDA, September 23, 2011
Alert: Eye Infections From Repackaged Avastin
Repackaged intravitreal injections of bevacizumab (Avastin, Roche/Genentech) have caused serious eye infections in the Miami, Florida, area.
Florida’s Department of Health notified the FDA of a cluster of Streptococcus endophthalmitis infections in three clinics after patients received intravitreal injections of repackaged bevacizumab. The product was traced to a single pharmacy in Hollywood, Fla. The pharmacy repackaged the drug from sterile injectable 100-mg/4-mL, single-use, preservative-free vials into individual 1-mL single-use syringes. The pharmacy then distributed the product to eye clinics.
Although all of the patients with an infection had visual deficits before receiving bevacizumab, some of these patients lost all remaining vision in the affected eye because of endophthalmitis.
Bevacizumab is used to treat various types of cancers. It is also sometimes used off-label for patients with wet age-related macular degeneration, although it is not currently approved for this indication.
Sources: FDA, August 31, 2011;
New Guidelines Address Pediatric Pneumonia
Immunizations, including a yearly flu vaccine, are the best way to protect children from life-threatening pneumonia, according to new guidelines from the Pediatric Infectious Diseases Society and the Infectious Diseases Society of America.
These guidelines, the first to address community-acquired pneumonia in infants and children, emphasize the prevention of bacterial pneumonia as a top priority.
Every year, pneumonia kills more than 2 million children 5 years of age and younger worldwide. In 2006, 525 children 15 years of age or younger died of pneumonia and other lower respiratory tract infections in the U.S., according to the Centers for Disease Control and Prevention.
Sources: Clin Infect Dis, August 30, 2011;
Label Change: High-Dose Celexa and Arrhythmias
The FDA has informed health care professionals and patients that the antidepressant citalopram hydrobromide (Celexa, Forest) should no longer be used at dosages higher than 40 mg/day. Abnormal changes in the electrical activity of the heart have been noted with high doses. Studies have not shown a benefit in the treatment of depression at dosages higher than 40 mg/day. The label for citalopram had previously stated that some patients may require a dosage of 60 mg/day.
Prolongation of the QT interval on electrocardiograms can lead to abnormal heart rhythms, including torsades de pointes, which can be fatal. Patients at risk for experiencing a prolonged QT interval include those with underlying heart conditions and those who tend to have low serum levels of potassium and magnesium.
The Celexa label has been revised to include the new dosage and usage recommendations as well as information about the potential for QT interval prolongation and torsades de pointes.
Source: FDA, August 25, 2011
Doubling Up On Antihypertension Drugs
Treating new-onset hypertension with more than one drug is becoming a common strategy. For one thing, clinical trials have shown that most patients do not reach their blood pressure (BP) goal with one drug alone. Moreover, combining drugs allows physicians to prescribe lower doses of both. But how does combination therapy measure up in the real world of clinical practice? Pretty well, say researchers from California and Colorado. At 12 months, combination antihypertensive treatment was associated with better long-term BP control.
The team evaluated data from the Cardiovascular Research Network Hypertension Registry, which includes 161,585 patients with newly diagnosed hypertension who started treatment between 2002 and 2007.
Patients were divided into two groups: stage 1 and stage 2 (an initial systolic BP of 160 mm Hg or above or a diastolic BP of 100 mm Hg or above). In both groups, the proportion of patients who began treatment with two drugs increased between 2002 and 2007 but more so in the stage 2 group (from 21.6% to 44.5%). Between 2002 and 2007, fewer patients were treated initially for stage 2 hypertension, which might have been related to earlier recognition of hypertension. Nearly 90% of initial therapy was accounted for by a combination of two drug classes: a thiazide plus a potassium-sparing diuretic and a thiazide plus an angiotensin-converting enzyme (ACE) inhibitor. At 1 year, BP was under control for 78,095 (62.5%) of the 124,984 patients available for analysis. Treatment with two antihypertensive drugs at the start of therapy was associated with a higher rate of BP control.
The researchers also compared adherence in patients receiving single-drug and combination-drug therapy. The difference between the two groups was statistically, but not clinically, significant; however, the association between combination therapy and improved BP control remained consistent after the researchers adjusted for adherence to antihypertensive medications. Patients starting combination therapy were significantly less likely to show an increase in the number of classes of antihypertensive medications during the year of follow-up, compared with patients receiving single-agent therapy (32.1% vs. 37.4%, respectively). Dosing increases were similar (24.6% vs 24.9%, respectively).
The researchers say that their findings highlight not only the increasing role of combination antihypertensive agents in routine practice (in line with Joint National Committee guidelines) but also the potential long-term benefits of combination therapy for hypertension.
Source: Am Heart J 2011;162(2):340–346 (August)
A ‘Milkshake’ For Alzheimer’s Disease
A vanilla or strawberry milkshake (Axona, Accera, Inc.) may be able to provide energy to the brain in patients with Alzheimer’s disease (AD). A trial found short-term positive effects in cognition and memor y, but the evidence was insufficient to conclude that the drink was effective.
Axona, which is available by prescription only, is a 217-calorie packet that can be mixed with a liquid using a shaker cup or a blender. Accera, Inc., claims that the brains of patients with AD do not convert glucose into energy as efficiently as healthy brains; this can result in a decrease in cognitive function.
The shake contains caprylic triglyceride, a compound derived from coconut oil.
In a study of patients with mild-to-moderate AD, after 45 days of drinking Axona each day, patients improved by 1.9 points on a 70-point cognitive scale compared with those drinking a placebo milkshake. However, over a period of 90 days, Axona was no better than the placebo shake. After 45 and 90 days of use, Axona was effective only in patients who did not have a gene that is commonly present in AD.
The shake is sold as a medical food; thus, the FDA’s approval is not required.
Sources: Nutrition Metab 2009;6:31; The Wall Street Journal, August 30, 2011
NEW MEDICAL DEVICES
Marvin M. Goldenberg, PhD, RPh, MS
Name: LipiFlow Thermal Pulsation System
Manufacturer: TearScience, Inc., Morrisville, N.C.
Approval Date: July 12, 2011
Purpose: This pulsation system is used for the diagnosis and treatment of dry eye disease. Most patients have evaporative dry eye, which occurs when the water (aqueous) in the tears evaporates at a faster rate than normal because of an insufficient lipid layer on the surface of the tear film. This form of dry eye is caused by a blockage in the oil-producing meibomian glands, located within the eyelids. Few patients are affected exclusively by aqueous-deficient dry eye, which occurs when tear formation generation from the lacrimal glands is insufficient to keep the eyes moist.
Dry eye is a chronic disease that takes years to develop. Both the evaporative and aqueous-deficient forms have long been lumped together as “dry eye.” Only recently have researchers been able to pinpoint the differences between the two forms and their causes.
Description: The system has four or more components, including a hand-held scanner and a device resembling a desktop computer with connections that attach to the patient’s eyes. For evaporative dry eye, localized heat and pressure therapy are applied to open the blocked eyelid glands. Previous approaches, such as the use of warm compresses, are inconvenient and have limited effectiveness.
Benefit: As the population ages, an excessive amount of time spent staring at computer screens and televisions is thought to be a factor in the increase in dry eye. Thermal propulsion in the Lipi-Flow device helps to restore the ocular surface to a healthier state and offers a potential improvement over eyedrops and other therapies that are often ineffective or cumbersome for patients. The device relieves blockage of the eyelid glands during an office visit so that the production of lipids needed for the tear film can resume.
Name: Epiducer Lead Delivery System
Manufacturer: St. Jude Medical, Inc., St. Paul, Minn.
Approval Date: July 12, 2011
Purpose: The Epiducer system has been approved for limited market release for the treatment of chronic pain.
Description: Spinal cord stimulation is used for managing pain of the trunk and limbs and pain from unsuccessful back surgery. Mild electrical pulses are carried from the neurostimulator to a lead that is placed in the epidural space near the spine to interrupt or mask the transmission of pain signals to the brain. The electrodes on the lead can be programmed to meet each patient’s needs.
Benefit: According to the Institute of Medicine, chronic pain affects an estimated 116 million American adults, more than the total affected by heart disease, cancer, and diabetes combined. Pain also costs the nation up to $635 billion each year in medical treatment and lost productivity. The Epiducer system is designed to reduce the complexities of spinal cord stimulation and to enhance efficiency. Before the device was approved, the paddles could be placed only via a laminotomy, a more invasive surgical procedure that usually involves removal of part of the vertebral bone. The paddle leads can be introduced percutaneously. The new system also reduces the need for multiple incisions, which are often required to place more than one neurostimulation lead during spinal cord stimulation.
With this minimally invasive system, physicians can deliver up to three different leads through a single entry point. Complex pain patterns, such as low back pain combined with leg pain, can be managed effectively.
Name: Zenith Spiral-Z AAA Iliac Leg Graft
Manufacturer: Cook Medical, Bloomington, Ind.
Approval Date: July 12, 2011
Purpose: Cook Medical has received FDA premarket approval for the Zenith Spiral-Z AAA Iliac Leg Graft. The device provides graft support in the iliac artery in patients with abdominal aortic aneurysms (AAAs).
Description: The device is used during primary or secondary procedures in patients with adequate iliac or femoral access compatible with the required introductory systems.
Benefit: The Spiral-Z feature enhances flexibility and resistance to kinking, thanks to the continuous nitinol spiral stent. A polytetrafluoroethylene-coated lumen reduces surface friction and facilitates precise delivery of the device.