FDA approves Remicade to treat ulcerative colitis in children older than 6 years

The U.S. Food and Drug Administration today approved Remicade (infliximab) to treat moderately to severely active ulcerative colitis (UC) in children older than 6 years who have had inadequate response to conventional therapy.

Remicade reduces signs and symptoms of UC and induces and maintains clinical remission in these patients.

UC is a type of inflammatory bowel disease (IBD) that affects the lining of the large intestine (colon) and rectum. Symptoms of UC include abdominal pain, diarrhea, rectal bleeding, weight loss and fever. Between 50,000 and 100,000 children in the United States have IBD; of these, 40 percent have UC.

Prolia (denosumab) approved by the FDA for the treatment of osteoporosis

The U.S. Food and Drug Administration today approved Prolia (denosumab), an injectable treatment for postmenopausal women with osteoporosis who are at high risk for fractures.  Prolia is a fully-human monoclonal antibody and is the first biologic agent approved for the treatment of osteoporosis. 

Prolia was approved with a risk evaluation and mitigation strategy (REMS) that includes a Medication Guide for patients and communications to health care providers that explains the risks and benefits of the drug.

Prolia is manufactured by Amgen Manufacturing Limited, a subsidiary of Thousand Oaks, Calif.-based Amgen Inc.

Nanotechnology to Fight Cancer

This is a guest post by Teresa Jackson. 

Nanotechnology to Fight Cancer

It’s arguably the most horrible disease known to man, and even though it’s been around for ages, we’re yet to find a cure for this deadly illness we know as cancer. It’s a poison that kills you slowly, and with a great deal of physical and emotional pain and suffering. But that’s not the worst part – what’s really unbearable is that you know you’re definitely going to die, and suffer through the process. But if there’s one spark of hope for those who’re being affected by the disease, it’s the one being generated by the field of nanotechnology.

The problem with current methods of treatment of cancer is that they destroy healthy cells as well when they destroy the cancerous ones. And this causes you to suffer side effects and other adverse consequences. Nanotechnology comes to the rescue in such situations by targeting only the cancerous cells using tiny particles that are used to inject the cancer-killing medicine exactly where they’re needed and nowhere else. This form of targeted therapy is now being used not only to deliver medication but also to turn off the proteins that are associated with and contribute to the growth of cancerous cells.

Genomics and Personalized Medicine

The topics of genomics and personalized medicine are very hot topics these days. Let's start by seeing how Wikipedia defines these terms:

Genomics:

Genomics is the study of the genomes of organisms. The field includes intensive efforts to determine the entire DNA sequence of organisms and fine-scale genetic mapping efforts. The field also includes studies of intragenomic phenomena such as heterosis, epistasis, pleiotropy and other interactions between loci and alleles within the genome. In contrast, the investigation of the roles and functions of single genes is a primary focus of molecular biology or genetics and is a common topic of modern medical and biological research. Research of single genes does not fall into the definition of genomics unless the aim of this genetic, pathway, and functional information analysis is to elucidate its effect on, place in, and response to the entire genome's networks.

Personalized Medicine

The United States Congress defines personalized medicine as "the application of genomic and molecular data to better target the delivery of health care, facilitate the discovery and clinical testing of new products, and help determine a person's predisposition to a particular disease or condition."

The application of personalized medicine is being studied heavily in the area of oncology. Tailored therapies are being developed for patients who have specific genetic mutations. Targeted biologic agents are being used to treat diseases at the molecular level. The definition of the term "bioengineering" or "biological engineering" will continue to evolve as we see tremendous advances in medical technology.

2009 ASCO EHR Symposium

The 2009 ASCO (American Society of Clinical Oncology) Electronic Health Record (EHR) symposium is this October in San Francisco, CA. Because of the HITECH provisions found in the ARRA, we know that EHRs will become very prevalent across all medical specialties, including oncology.

In the field of oncology, so many medications cause significant adverse reactions. Plus, combination therapy is often used to treat cancer. EHRs can help clinicians keep track of all the treatment infusions and they may also alert providers about the possible risks associated with combining certain chemotherapy agents with targeted biologics.

Interested in knowing who will be speaking? Here are just a few examples:

• Peter Yu, M.D. - Chair ASCO EHR Workgroup, Oncologist, Palo Alto Medical Foundation (PAMF)
• Barbara McAneny, M.D. - CEO, New Mexico Oncology Hematology Consultants
• Charles McKay, M.D. - CEO, Tennessee Oncology
• John Cox, D.O., M.B.A. - Editor-in-Chief, Journal of Oncology Practice
• Robert Miller, M.D. - Oncologist, Johns Hopkins Kimmel Cancer Center
• Kenneth Buetow, Ph.D. - Director, NCI's Center for Biomedical Informatics and Information Technology (CBIIT)
• Lawrence Shulman, M.D. - Chief Medical Officer, Dana- Farber Cancer Center
• William Hersh, M.D. - Chair, Department of Medical Information and Clinical Epidemiology, Oregon Health and Sciences University

If you're an oncologist (and you're not retiring anytime soon), you won't want to miss this! To get more information about the ASCO EHR symposium, visit: http://www.asco.org/ehrsymposium

FDA: "Expanded Access to Investigational Drugs for Treatment Use"

FDA Issues Final Rules to Help Patients Gain Access to Investigational Drugs

The U.S. Food and Drug Administration published two rules today that seek to clarify the methods available to seriously ill patients interested in gaining access to investigational drugs and biologics when they are not eligible to participate in a clinical trial and don’t have other satisfactory treatment options.

To support the effort to help these patients, the agency also is launching a new Web site where patients and their health care professionals can learn about options for investigational drugs. In general, these options include being treated with a drug that has been approved by FDA, being given an investigational drug as part of a clinical trial, or obtaining access to an investigational drug outside of a clinical trial.

The new rule, “Expanded Access to Investigational Drugs for Treatment Use,” makes investigational drugs more widely available to patients by clarifying procedures and standards. The other rule, “Charging for Investigational Drugs Under an Investigational New Drug Application,” clarifies the specific circumstances and the types of costs for which a manufacturer can charge patients for an investigational drug when used as part of a clinical trial or when used outside the scope of a clinical trial.

“With these initiatives, patients will have the information they need to help them decide whether to seek investigational products,” said Margaret A. Hamburg, M.D., Commissioner of Food and Drugs. “For patients seeking expanded access to investigational drugs and biologics, the new rules make the process easier to understand.”

Clinical trials are studies of drugs and biologics that are still in development and have not yet been approved by the FDA. Many patients enroll in clinical trials to gain access to investigational therapies and contribute to finding out how well an investigational therapy works, and how safe it is for patients. Obtaining a drug or biologic under an expanded access program may be an option for some patients who are not able to enroll in clinical trials.

The FDA has allowed expanded access to experimental drugs and biologics since the 1970s. That access has allowed tens of thousands of patients with HIV/AIDS, cancer, and other conditions to receive promising therapies when no approved alternative is available.

“The final rules balance access to promising new therapies against the need to protect patient safety and seek to ensure that expanded access does not discourage participation in clinical trials or otherwise interfere with the drug development process,” said Janet Woodcock, M.D., director of the FDA’s Center for Drug Evaluation and Research. “Clinical trials are the most important part of the drug development process in determining whether new drugs are safe and effective, and how to best use them.”

Web site that explains the options for investigational drugs

Final Rules for Expanded Access to Investigational Drugs for Treatment Use and Charging for Investigational Drugs

So, how many of your patients should be on an investigational drug? If you're an oncologist or an HIV specialist, then you may be referring your patients to all sorts of clinical studies. There are so many new biologic agents and novel antiviral combinations that are emerging for cancer and HIV/AIDS patients.

Co-pay support program for patients taking Enbrel (Etanercept)

Amgen and Wyeth Pharmaceuticals have announced the ENBREL Support™ Co-pay Card Program for all eligible patients who take Enbrel (Etanercept). Who's eligible? Well, maybe we should start with those who are not eligible:

This program is not open to uninsured patients or patients receiving prescription reimbursement from federal, state, or government-funded insurance programs (for example, Medicare, Medicaid, etc.) or patients who live in Massachusetts (or where prohibited by law). Restrictions, including monthly maximums, may apply. Offer subject to change or discontinuation without notice.

This co-pay support program may offer:

• 6 months at no co-pay cost, and
• A co-pay of $10 or less per month thereafter (Patients will receive 50% off their co-pay or pay no more than $10 per month—whichever helps more)

The ENBREL Support™ Co-pay Card Program provides patients with up to $750 of assistance per month for months 1-12. For patients with moderate to severe plaque psoriasis who are first starting ENBREL, the program provides up to $1,500 per patient per month for months 1-3. Patient is responsible for costs above these amounts. Participation in the program can be renewed every year.

More than 45,000 people have already used a co-pay card for their ENBREL treatment.
The program is simple for you to share with patients. Direct patients to call 1-888-4ENBREL to enroll today.

Patients who lose their jobs but are covered by private insurance, including COBRA, may receive up to an additional 6 months of ENBREL at no co-pay cost. Potential help for patients who are uninsured or in need of other financial help is also available.

Here are the FDA-approved indications for ENBREL:

• ENBREL is indicated for reducing signs and symptoms, inducing major clinical response, inhibiting the progression of structural damage, and improving physical function in patients with moderately to severely active rheumatoid arthritis. ENBREL can be initiated in combination with methotrexate (MTX) or used alone.
• ENBREL is indicated for reducing signs and symptoms of moderately to severely active polyarticular juvenile idiopathic arthritis in patients ages 2 and older.
• ENBREL is indicated for reducing signs and symptoms, inhibiting the progression of structural damage of active arthritis, and improving physical function in patients with psoriatic arthritis. ENBREL can be used in combination with methotrexate in patients who do not respond adequately to methotrexate alone.
• ENBREL is indicated for reducing signs and symptoms in patients with active ankylosing spondylitis.
• ENBREL is indicated for the treatment of adult patients (18 years or older) with chronic moderate to severe plaque psoriasis who are candidates for systemic therapy or phototherapy.

Click here for additional details of the co-pay support program.

Since Enbrel is a TNF blocker, make sure you read this:

• FDA: Cancer Warnings Required for TNF Blockers

FDA: Cancer Warnings Required for TNF Blockers

Here is the latest news from the FDA regarding TNF blockers and the risk of cancer:

FDA: Cancer Warnings Required for TNF Blockers

The U.S. Food and Drug Administration is requiring stronger warnings in the prescribing information for a class of drugs known as TNF blockers. The warnings, which include an updated boxed warning, highlight the increased risk of cancer in children and adolescents who receive these drugs to treat juvenile rheumatoid arthritis, the inflammatory bowel disorder, Crohn’s disease, and other inflammatory diseases.

In addition, the FDA is working with manufacturers to explore new ways to further define the risk of cancer in children and adolescents who use these drugs.

TNF blockers target and neutralize tumor necrosis factor-alpha (TNF-α), a protein that, when overproduced in the body due to chronic inflammatory diseases, can cause inflammation and damage to bones, cartilage and tissue. The drugs in this class include

• Remicade (infliximab),
  
• Enbrel (etancercept),
  
• Humira (adalimumab),
  
• Cimzia (certolizumab pegol) and
  
• Simponi (golimumab).

Today’s action is based on the completion of an investigation first announced by the FDA in June 2008. An analysis of U.S. reports of cancer in children and adolescents treated with TNF-blockers showed an increased risk of cancer, occurring after 30 months of treatment on average. About half of the cancers were lymphomas, a type of cancer involving cells of the immune system. Some of the reported cancers were fatal.

Additional required updates to the prescribing information include incorporation of reports of psoriasis associated with the use of TNF blockers.

Can you list who manufacturers the medications listed above? Eventually, I think we're going to realize that there's an increased risk of cancer with many of the biologic agents that are out there. The irony is that many biologic agents (different classes of drugs with varying mechanisms of actions) are also used to treat cancer. As we learn more about biologic agents, we may eventually complete the cancer circle. The more we understand about what causes cancer, the better we may be able to treat cancer.

The FDA is reviewing safety information on Xolair

The FDA has released an Early Communication about an ongoing safety review of Xolair (omalizumab). This is a biologic agent used for the treatment of asthma. Please note that the FDA is not recommending any changes to the prescribing information for Xolair and is not advising patients to stop taking Xolair at this time.

Here's the early communication from the FDA:

Early Communication about an Ongoing Safety Review of Omalizumab (marketed as Xolair)

FDA is evaluating interim safety findings from an ongoing study of Xolair (omalizumab) that suggests an increased number of cardiovascular and cerebrovascular adverse events in a group of patients using Xolair compared to a group of patients not given the drug (control group).

Xolair is approved for use by adults and adolescents (12 years of age and above) with moderate to severe persistent asthma who test positive for reactivity to a perennial airborne allergen, and whose symptoms are inadequately controlled with inhaled corticosteroids.

The ongoing study, titled Evaluating the Clinical Effectiveness and Long-Term Safety in Patients with Moderate to Severe Asthma (EXCELS), is an observational study of approximately 5000 Xolair treated patients and a control group of approximately 2500 non-Xolair treated patients. The primary objective of the EXCELS study is to assess the long-term safety profile of Xolair in patients followed for 5 years. Study patients are 12 years of age and older with moderate to severe persistent asthma and who have a positive skin test or blood test for an aeroallergen.

The interim data, submitted by the manufacturer of Xolair (Genentech), suggests a disproportionate increase in ischemic heart disease, arrhythmias, cardiomyopathy and cardiac failure, pulmonary hypertension, cerebrovascular disorders, and embolic, thrombotic and thrombophlebitic events in patients treated with Xolair compared to the control group of patients not given the drug.

FDA is not recommending any changes to the prescribing information for Xolair and is not advising patients to stop taking Xolair at this time. Until the evaluation of the EXCELS study is completed, healthcare providers and patients should be aware of the risks and benefits described in the prescribing information, as well as the new information from the ongoing EXCELS study that may suggest a risk of cardiovascular and cerebrovascular adverse events.

This early communication is in keeping with FDA’s commitment to inform the public about ongoing safety reviews of drugs. FDA has not made any conclusions regarding these data. The Agency is working with Genentech to obtain further information and will continue to review the strengths and limitations of these interim results. For example, since EXCELS is an observational study, there could be differences in underlying risk factors for cardiovascular and cerebrovascular events between the two study groups. The Agency will communicate any new findings when its analysis of the interim safety data is complete. The EXCELS study is ongoing and final results are not expected until 2012.

The FDA urges both healthcare professionals and patients to report side effects from the use of omalizumab to the FDA's MedWatch Adverse Event Reporting program either online, by regular mail or by fax, using the contact information at the bottom of this page.

This information reflects FDA’s current analysis of available data concerning these drugs. Posting this information does not mean that FDA has concluded there is a causal relationship between the drug products and the emerging safety issue. Nor does it mean that FDA is advising health care professionals to discontinue prescribing these products. FDA is considering, but has not reached a conclusion about whether this information warrants any regulatory action. FDA intends to update this document when additional information or analyses become available.

Let's get the word out to healthcare professionals so that we can see what type of data comes in through MedWatch. I believe that we can improve public health by using social media outlets, including blogs, Twitter, RSS feeds, and forums. Click here to view the FDA early communication about Xolair.

Arzerra (ofatumumab) appears promising for CLL

Arzerra (ofatumumab) appears promising for chronic lymphocytic leukemia (CLL). Ofatumumab (formerly known as HuMax-CD20) is a targeted therapy. It is actually a human monoclonal antibody that targets a distinct antibody binding site (the small loop epitope) of the CD20 molecule on the cell membrane of B cells. It is currently under development for the treatment of CLL and has also shown potential in treating follicular non-Hodgkin’s lymphoma, diffuse large B cell lymphoma, rheumatoid arthritis and relapsing remitting multiple sclerosis. Is it available yet? No. The FDA is currently still reviewing this biologic agent. Here's a snippet from the June GSK press release:

GlaxoSmithKline and Genmab A/S today announced that the United States Food and Drug Administration (FDA) informed the companies that the agency has extended the action date for the ofatumumab BLA application by three months.

The FDA has been slow recently, don't you think? Better safe than sorry? Or, is the FDA being overly-cautious? We don't want to see another Raptiva incident again, do we? Raptiva was voluntarily withdrawn in April by Genentech because of an increased risk of progressive multifocal leukoencephalopathy (PML), a rare and usually fatal disease of the central nervous system. It's difficult to find such rare adverse effects until the drug gets used by the public, so I don't think anyone is faulting either the FDA or Genentech.

Well, let's see what happens with ofatumumab.

FDA Approves First Maintenance Drug Therapy for Advanced Lung Cancer

This isn't a new drug, but it's a new indication: "maintenance therapy of advanced or metastatic lung cancer." Alimta was originally approved in 2004 for mesothelioma. Here's the summary straight from the FDA:

The U.S. Food and Drug Administration has approved Alimta (pemetrexed), the first drug available for maintenance therapy of advanced or metastatic lung cancer.

Patients with cancer often receive maintenance therapy to prevent the disease from progressing after their tumor has shrunk or the disease has stabilized in response to chemotherapy. Alimta disrupts metabolic processes that are dependent on the B-vitamin folate, a necessary ingredient for cell replication.

“This drug represents a new approach in the treatment of advanced non-small cell lung cancer,” said Richard Pazdur, M.D., director, Office of Oncology Drug Products in the FDA’s Center for Drug Evaluation and Research. “Typically, patients whose tumors respond to chemotherapy do not receive further treatment after four-to-six chemotherapy cycles. This study demonstrates an advantage in overall survival in certain patients who received Alimta for maintenance therapy.”

Non-small cell lung cancer has several subtypes, including squamous cell, large cell, adenocarcinoma and mixed histology cancers. In a 600-patient clinical trial, people with predominantly squamous cell cancer did not benefit from Alimta. But those with other subtypes of non-small lung cancer survived an average 15.5 months following treatment compared with 10.3 months for patients who received an inactive substance (placebo). All patients in the study received standard medical care.

Reported adverse events included damage to blood cells, fatigue, nausea, loss of appetite, tingling or numbness in the hands and feet, and skin rash.

Alimta initially was approved in 2004 for the treatment of patients with mesothelioma, a cancer frequently related to asbestos exposure. The drug was later approved for the treatment of patients with non-small cell lung cancer whose disease worsened on prior chemotherapy drugs and also as an initial therapy for advanced non-small cell lung cancer.

Alimta is manufactured by Eli Lilly & Co. of Indianapolis.

It's exciting to see advances in cancer treatment that focus on new drugs, biologic agents, and combination approaches. The world of oncology is buzzing with new science and the application of technology such as nanotechnology and targeted molecular therapies. Will we ever discover a cure? I doubt that we'll ever see a magic pill that completely eliminates every type of cancer, but lung cancer is a a highly preventable cancer so hopefully we'll see improved efforts at prevention. The greatest public health impact will be made if we improve our preventive health efforts.

US Oncology Launches Oncology-Specific EHR

US Oncology Launches Oncology-Specific EHR to the Open Market. iKnowMed is now available to community-based oncologists. Will this become the dominant EHR among oncologists? US Oncology acquired iKnowMed in 2004.

So what's unique about iKnowMed? One feature is that it leverages technology to help oncologists focus cost effectiveness in community cancer care. After all, biologic agents are very expensive and many patients have trouble paying for their treatment when it involves a combination of chemotherapy plus biologic targeted therapies. iKnowMed also provides oncologists with easy access to US Oncology's Innovent Oncology program so they can gain benefits associated with pay-for-performance (P4P) programs.

Personalized Cancer Treatment

Each person who develops cancer has a unique condition. The world of oncology isn't driven by a cookbook approach (although sometimes it may seem like it). What does it mean to receive personalized cancer treatment? Are we talking about personalized medicine? Genomics? The use of biologic targeted therapies that are designed to combat cancer on the molecular level? The application of nanotechnology to improve drug delivery?

Have you heard of Champions Biotechnology? They've created a new website dedicated to the topic of Personalized Cancer Treatment.

The URL is very simple: PersonalizedCancerTreatment.com
There, you'll find information about:

• Personalized Tumorgraft™
• Personalized Vaccine™
• Personalized Oncology Panel™

Image source: GSBTM

Stelara (ustekinumab) Delayed by the FDA

The psoriasis Stelera (ustekinumab) is a biologic agent that is being reviewed by the FDA for the treatment of psoriasis. The FDA wants more time to review the safety of this biologic (I wonder if this is related to the fact that Raptiva (efalizumab) was voluntarily removed from the market because of safety concerns). Raptiva was approved in 2003 and was used to treat over 46,000 patients. Ustekinumab is a monoclonal antibody given by injections and works on interleukin-12 (IL-12) and interleukin-23 (IL-23). Stelera is currently an investigational agent manufactured by Johnson & Johnson.

Do you know why all these biologic agents sound somewhat familiar? They all seem to end in ab or mab, don't they? Take a look at this: Anticytokine Therapies - confusing names?

CIMZIA and OXO GOOD GRIPS

I love kitchenware made by OXO GOOD GRIPS®. Who would have thought that they would team up with a pharmaceutical company to develop an innovative syringe filled with a biologic agent?

UCB, the biopharmaceutical company that makes CIMZIA (certolizumab pegol), partnered with OXO GOOD GRIPS® to design a syringe that would be easy to use for many people. Sounds like a great idea to me. A soft, non-slip grip should make this easier to use. Should we expect to see more of these "easy to use" syringes for other injectable drugs?

Cimzia is currently the only PEGylated anti-TNF (Tumor Necrosis Factor) approved by the FDA for reducing signs and symptoms of Crohn's disease and maintaining clinical response in adult patients with moderate to severe active disease who have had an inadequate response to usual treatments. Cimzia is also FDA approved for the treatment of adults who have moderately to severely active rheumatoid arthritis (RA).

FDA Approves ATryn

Have you heard of ATryn? No, that's not a typo. It's an anti-coagulant used for patients with hereditary antithrombin (AT) deficiency. ATryn is made by GTC Biotherapeutics, Inc.

ATryn is unique in that it is the first FDA approved drug made by genetically engineered (GE) animals. So if we're genetically engineering animals, do you think anyone's genetically engineering any humans? Both the Center for Biologics Evaluation and Research (CBER) approved this agent based on its safety and efficacy and the Center for Veterinary Medicine (CVM) approved the rDNA construct in the goats that produce ATryn.

ATryn has an orphan drug designation by the FDA since hereditary antithrombin (AT) deficiency is so uncommon.

Anticytokine Therapies - confusing names?

Ever get confused with medical terminology? Happens to me all the time. Anticytokine therapies and other biologics are revolutionizing the treatment of some serious diseases. Here's an example of a patient with psoriasis who responded to alefacept.

When you're talking about some of the newer biologic agents, you may find that they're easy to mix up. Here's a helpful guide regarding nomenclature.

Abbreviations placed at the ends of the names convey specific information relating to their structure:

* "-cept" refers to fusion of a receptor to the Fc part of human IgG1
* "-mab" indicates a monoclonal antibody (mAb)
* "-ximab" indicates a chimeric mAb
* "-zumab" indicates a humanized mAb

Etanercept is a classic example of a TNF receptor fusion protein. Infliximab is a chimeric mAb directed against TNF. Certolizumab and tocilizumab are humanized mAbs.