|May 2013, Volume XXIII, No. 3|
Regulatory & Legislative Highlights
The FDA has approved Genentech's Actemra (tocilizumab), given alone or in combination with methotrexate, for the treatment of active systemic juvenile idiopathic arthritis (SJIA) in children ages 2 years and older. SJIA, or Still's disease, is a rare, potentially life-threatening disorder in children that causes severe inflammation throughout the body. SJIA is distinguished from other forms of juvenile idiopathic arthritis (JIA) by the prominence of systemic and inflammatory features, including spiking fevers; rash; swelling and inflammation of lymph nodes, liver, and spleen; and high white blood cell and platelet counts. The prevalence of JIA is an estimated 1 to 2 per 1,000 children, and SJIA affects about 10 percent of all JIA patients.
Actemra carries a Boxed Warning for serious infections. Patients treated with Actemra who develop a serious infection should stop Actemra treatment until the infection is controlled. A Boxed Warning is a brief, concise summary of the information that is critical for a prescriber to be aware of, including any restriction on distribution or use, which is included in a black box at the beginning of the drug label. (Source: 15 April, 2011, FDA Website)
The FDA has approved the NovoTTF-100A System, a new device to treat adults with glioblastoma multiforme (GBM) that recurs or progresses after receiving chemotherapy and radiation therapy. The NovoTTF-100A System is made by Novocure of Portsmouth, New Hampshire. According to the National Cancer Institute, each year about 19,000 people in the United States are diagnosed with primary brain cancers. In 2010, there were 13,140 deaths from brain and other nervous system cancers in the US. GBM is the most common primary brain cancer. The brain tumor is highly resistant to standard treatments such as surgery, radiation and chemotherapy.
When using the NovoTTF-100A System, a health care professional places electrodes on the surface of the patient's scalp to deliver low-intensity, changing electrical fields called "tumor treatment fields" (TTFs) to the tumor site. The unique shape and electrical characteristics of dividing tumor cells make them susceptible to damage when exposed to TTF, which could stop tumor growth.
The device is portable and can be powered with batteries or plugged into an electrical outlet. Patients can use the device at home, allowing them to continue their normal daily activities. (Source: 15 April, 2011, FDA Website)
The FDA has approved Genentech's Rituxan (rituximab), in combination with glucocorticoids (steroids), to treat patients with Wegener's granulomatosis (WG) and microscopic polyangiitis (MPA), two rare disorders that cause blood vessel inflammation (vasculitis). Rituxan is an antibody that is manufactured through biotechnology methods. The drug works by greatly reducing the number of specific immune cells in the blood, known as B cells.
Vasculitis in patients with WG and MPA can lead to tissue damage. WG mostly affects the respiratory tract (sinuses, nose, trachea, and lungs) and kidneys, while MPA commonly affects the kidneys, lungs, nerves, skin, and joints. Both of these diseases affect people of all ages and ethnicities, and both genders. The causes of these disorders are unknown, and both are considered orphan diseases because they each affect less than 200,000 people in the US.
Rituxan carries a Boxed Warning for infusion reactions, which can occur during infusion or within 24 hours afterwards. Other Boxed Warnings for Rituxan include rashes and sores in the skin and mouth (severe mucocutaneous reactions); and progressive multifocal leukoencephalopathy, a brain infection that generally is fatal. Rituxan is not recommended for use in patients with severe, active infections.
Rituxan, which has been marketed since 1997, is also indicated for the treatment of patients with non-Hodgkin's lymphoma, chronic lymphocytic leukemia, and rheumatoid arthritis. (Source: 19 April, 2011, FDA Website)
The FDA has released the final version of a strategic priorities document outlining the goals that will guide the agency and its 12,000 employees through 2015. Titled "Strategic Priorities 2011 - 2015: Responding to the Public Health Challenges of the 21st Century," the 50-page document provides a vision of the FDA that includes: a modernized field of regulatory science that draws on innovations in science and technology to help ensure the safety and effectiveness of medical products throughout their life cycles; an integrated global food safety system focused on prevention and improved nutrition; and expanded efforts to meet the needs of special populations.
The document contains four sections: Introduction, Cross-Cutting Strategic Priorities, Strategic Goals and Long-Term Objectives, and Implementation. Senior staff from the FDA's seven product and research centers and two major offices contributed to the document. A Federal Register notice that invited public comment in October 2010 generated more than 200 comments and the final document includes that input.
The FDA commissioner stated that she would continue to act as an advocate for advancing the field of regulatory science and innovation, one of five cross-cutting areas that serve as strategic priorities at the agency over the next five years. (Source: 20 April, 2011, FDA Website)
The FDA has approved the use of Menactra in children as young as 9 months for the prevention of invasive meningococcal disease caused by Neisseria meningitidis serogroups A, C, Y and W-135. Menactra is manufactured by Sanofi Pasteur and is already approved for use in people ages 2 through 55 years.
Meningococcal disease is a life-threatening illness caused by bacteria that infect the bloodstream (sepsis) and the lining that surrounds the brain and spinal cord (meningitis). Neisseria meningitidis is a leading cause of meningitis in young children. Even with appropriate antibiotics and intensive care, between 10 percent and 15 percent of people who develop meningococcal disease die from the infection. Another 10 to 20 percent suffer complications such as brain damage or loss of limb or hearing.
Although the rates of meningococcal disease are low in the United States, infants and toddlers are more susceptible to getting this serious illness. Meningococcal disease is particularly dangerous because it progresses rapidly and can cause death within hours. Early symptoms are often difficult to distinguish from influenza and other common illnesses. (Source: 22 April, 2011, FDA Website)
The FDA has approved Zytiga (abiraterone acetate) in combination with prednisone (a steroid) to treat patients with late-stage (metastatic) castration-resistant prostate cancer who have received prior docetaxel (chemotherapy). Zytiga is marketed by Centocor Ortho Biotech.
In prostate cancer, the male sex hormone testosterone stimulates prostate tumors to grow. Drugs or surgery are used to reduce testosterone production or to block testosterone's effects. However, sometimes prostate cancer can continue to grow even when testosterone levels are low. Men with these cancers are said to have castration-resistant prostate cancer.
The FDA has approved Tradjenta (linagliptin) tablets, used with diet and exercise, to improve blood glucose control in adults with Type 2 diabetes. Tradjenta is marketed by Boehringer Ingelheim Pharmaceuticals and Eli Lilly Co.
People with Type 2 diabetes do not produce or respond normally to insulin, a hormone that regulates the amount of glucose in the blood. Over time, high blood glucose levels can increase the risk for serious complications, including heart disease, blindness, and nerve and kidney damage.
Tradjenta has been studied as a stand-alone therapy and in combination with other Type 2 diabetes therapies including metformin, glimepiride, and pioglitazone. Tradjenta has not been studied in combination with insulin, and should not be used to treat people with Type 1 diabetes or in those who have increased ketones in their blood or urine (diabetic ketoacidosis). (Source: 2 May, 2011, FDA Website)
The FDA has approved Afinitor (everolimus) to treat patients with progressive neuroendocrine tumors located in the pancreas (PNET) that cannot be removed by surgery or that have spread to other parts of the body (metastatic). Afinitor is marketed by East Hanover, N.J.-based Novartis.
Neuroendocrine tumors found in the pancreas are slow-growing and rare. It is estimated that there are fewer than 1,000 new cases in the United States each year.
Afinitor is also approved to treat patients with kidney cancer (advanced renal cell carcinoma) after they fail treatment with Sutent (sunitinib) or Nexavar (sorafenib); and patients with subependymal giant cell astrocytoma (a type of brain cancer) associated with tuberous sclerosis (a disease that causes tumors in various parts of the body), who cannot be treated by surgery.
Afinitor has another trade name, Zortress, and is approved to treat certain adult patients to prevent organ rejection after a kidney transplant. Zortress has a different safety profile in these patients. (Source: 6 May, 2011, FDA Website)
The FDA is requesting input from stakeholders and the public relating to the development of a user fee program for biosimilar and interchangeable biological product (351(k)) applications. The Biologics Price Competition and Innovation Act of 2009, a provision of the Affordable Care Act, creates an abbreviated approval pathway for biological products that are demonstrated to be highly similar (biosimilar) to, or interchangeable with an FDA-licensed biological product. It directs the FDA to develop recommendations for a 351(k) user fee program for fiscal years 2013 through 2017. The recommendations must be presented to Congress by January 15, 2012. (Source: 9 May, 2011, FDA Website)
The FDA has approved Victrelis (boceprevir) to treat certain adults with chronic hepatitis C. Victrelis is used for patients who still have some liver function, and who either have not been previously treated with drug therapy for their hepatitis C or who have failed such treatment. Victrelis is approved for use in combination with peginterferon alfa and ribavirin.
According to the Centers for Disease Control and Prevention, about 3.2 million people in the United States have chronic hepatitis C, a viral disease that causes inflammation of the liver that can lead to diminished liver function or liver failure. Victrelis is a pill taken three times a day with food. The therapy is part of a class of drugs referred to as protease inhibitors, which work by binding to the virus and preventing it from multiplying. (Source: 13 May, 2011, FDA Website)
The FDA approved Edurant (rilpivirine) in combination with other antiretroviral drugs for the treatment of HIV-1 infection in adults who have never taken HIV therapy (treatment-naïve). Edurant is manufactured by Tibotec Therapeutics, a division of Centocor Ortho Biotech.
Edurant is a pill taken once a day and belongs to a class of HIV drugs called non-nucleoside reverse transcriptase inhibitor (NNRTI). The drug works by blocking HIV viral replication. Edurant is to be used as part of a highly active antiretroviral therapy (HAART) regimen that is designed to suppress the amount of HIV (viral load) in the blood. Edurant does not cure HIV infection. Patients must stay on continuous HIV therapy to control HIV infection and decrease HIV-related illnesses. (Source: 20 May, 2011, FDA Website)
The FDA has approved Pfizer's Sutent (sunitinib) to treat patients with progressive neuroendocrine cancerous tumors located in the pancreas that cannot be removed by surgery or that have spread to other parts of the body (metastatic).
Neuroendocrine tumors found in the pancreas are slow-growing and rare. It is estimated that there are fewer than 1,000 new cases in the United States each year. This is the second new approval by the FDA to treat patients with this disease; the agency approved Afinitor (everolimus) earlier.
Sutent is also FDA-approved to treat patients with late-stage kidney cancer (metastatic renal cell carcinoma) and to treat patients with GIST (gastrointestinal stromal tumor), a rare cancer of the stomach, bowel, or esophagus. (Source: 20 May, 2011, FDA Website)
The FDA has approved Incivek (telaprevir) to treat certain adults with chronic hepatitis C infection. Incivek is used for patients who have either not received interferon-based drug therapy for their infection or who have not responded adequately to prior therapies. Incivek is approved for use with interferon therapy made up of peginterferon alfa and ribavirin. The current standard of care for patients with chronic hepatitis C infection is peginterferon alfa and ribavirin taken for 48 weeks. Less than 50 percent of patients respond to this therapy.
Incivek is a pill taken three times a day. It should be taken for the first 12 weeks in combination with peginterferon alfa and ribavirin. Most people with a good early response to the Incivek combination regimen can be treated for 24 weeks rather than the recommended 48 weeks of treatment with the standard of care. Incivek is part of a class of drugs referred to as protease inhibitors, which work by binding to the virus and preventing it from multiplying. (Source: 23 May, 2011, FDA Website)
The FDA has announced that it is disclosing more information about inspections and court actions, and has a web portal on its enforcement activities as part of Phase II of the agency's Transparency Initiative. These actions are being taken to make FDA's enforcement and compliance-related activities more accessible, downloadable, and searchable online.
The information includes a summary of the most common Inspectional Observations of objectionable conditions or practices made during inspections and a searchable Inspections Database that includes the names and addresses of inspected facilities, inspection dates, type of FDA-regulated products involved, and final inspectional classification.
The FDA has released draft guidance to provide regulated industries with greater certainty about the use of nanotechnology, which generally involves materials made up of particles that are one billionth of a meter in size. The guidance outlines the agency's view on whether regulated products contain nanomaterials or involve the application of nanotechnology.
Nanotechnology, the science involving manipulation of materials on an atomic or molecular scale, is an emerging technology with a broad range of potential applications, such as increasing bioavailability of a drug, improving food packaging and in cosmetics.
For products subject to premarket review, the FDA intends to apply the points contained in the draft guidance, when finalized, to better understand the properties and behavior of engineered nanomaterials. For products not subject to premarket review, the FDA will urge manufacturers to consult with the agency early in the product development process so questions related to the regulatory status, safety, effectiveness or public health impact of these products can be adequately addressed.
The FDA has a robust regulatory science agenda to develop the tools, methods and expertise necessary to evaluate products that contain nanomaterials or otherwise involve the use of nanotechnology. The FDA's regulatory science portfolio focuses on generating data needed to ensure the safety and effectiveness of products using nanomaterials, with an emphasis on products the use of which could present the greatest potential risk to public health. (Source: 9 June, 2011, FDA Website)
The FDA has approved Potiga (ezogabine) tablets for use as an add-on medication to treat seizures associated with epilepsy in adults. Potiga was developed by Valeant Pharmaceuticals North America of Durham, NC and will be distributed by GlaxoSmithKline of Research Triangle Park, NC.
Potiga was approved for the treatment of partial seizures, the most common type of seizure seen in people with epilepsy. Epilepsy is a brain disorder in which there is abnormal or excessive activity of nerve cells in the brain. Partial seizures affect only a limited or localized area of the brain, but can spread to other parts of the brain. Seizures cause a wide range of symptoms, including repetitive limb movements (spasms), unusual behavior, and generalized convulsions with loss of consciousness.
Potiga is the first neuronal potassium channel opener developed for the treatment of epilepsy. Although the mechanism of action is not firmly established, the drug may act as an anticonvulsant by reducing excitability through the stabilization of neuronal potassium channels in an "open" position. (Source: 13 June, 2011, FDA Website)
The FDA has approved a new genetic test that will help health care professionals determine if women with breast cancer are HER2-positive and, therefore, candidates for Herceptin (trastuzumab), a commonly used breast cancer treatment. The test, called Inform Dual ISH, allows for measurement of the number of copies of the HER2 gene in tumor tissue. The HER2 gene is located on chromosome 17 in human cells. An excessive amount of the protein produced by the gene is found in some types of cancer cells, including breast cancer cells. Arizona-based Ventana Medical Systems manufactures the Inform Dual ISH test. Herceptin is marketed by Genentech. (Source: 14 June, 2011, FDA Website)
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