|May 2013, Volume XXIII, No. 3|
Regulatory & Legislative Highlights
by Deepen Joshi, Sepracor
The FDA has approved a new manufacturing facility used to produce influenza virus vaccines. The facility is approved for seasonal influenza vaccine production and could be used for the production of vaccine against the new 2009 H1N1 influenza strain. The facility, located in the US, is owned and operated by Sanofi Pasteur, the vaccines division of Sanofi-Aventis, which manufactures Fluzone Influenza Virus Vaccine. This new facility will greatly increase Sanofi Pasteur's production capability.
As part of its overall pandemic influenza preparedness efforts, the FDA meets with vaccine manufacturers to guide the efficient establishment of influenza vaccine facilities that comply with agency requirements. The agency promptly reviews applications and manufacturing supplements that could increase both the number of manufacturers and the overall supply of vaccine.
The FDA has interacted with the company throughout the regulatory process to help ensure compliance with applicable requirements. The bulk manufacturing facility will be used for the production of Fluzone, Sanofi Pasteur's egg-based influenza vaccine. Sanofi Pasteur is located in Swiftwater, Pa. (Source: FDA Website, 6 May, 2009)
The FDA is requesting a budget of $3.2 billion to protect and promote the public health as part of the President's fiscal year (FY) 2010 budget - a 19 percent increase over the current FDA fiscal year budget.
The FY 2010 request, which covers the period of Oct. 1, 2009, through Sept. 30, 2010, includes increases of $295.2 million in budget authority and $215.4 million in industry user fees. The FDA budget proposes two major initiatives for FY 2010: Protecting America's Food Supply and Safer Medical Products. It also includes increases for current law user fees and for infrastructure to support critical agency operations. The FDA is also proposing four new user fees to facilitate review of generic drugs, register and inspect food manufacturing and processing facilities, reinspect facilities that fail to meet Good Manufacturing Practices and other safety requirements, and issue export certifications for food and feed.
The following are the FDA's key proposed budget increases:
(Source: FDA Website7 May, 2009)
The FDA has approved Genentech's Avastin (bevacizumab) to treat patients with glioblastoma multiforme (GBM) when this form of brain cancer continues to progress following standard therapy. GBM is a rapidly progressing cancer that invades brain tissue and can impact physical activities and mental abilities. It affects about 6,700 persons in the US every year. Following initial treatment with surgery, radiation, and/or chemotherapy, the cancer nearly always returns.
Avastin is a monoclonal antibody that mimics the antibodies produced by the body's immune system to defend against harmful substances. The medication inhibits the action of vascular endothelial growth factor that helps form new blood vessels. These vessels can feed a tumor, helping it to grow and can also provide a pathway for cancer cells to circulate in the body. The drug was first approved in 2004 to treat metastatic cancer of the colon or rectum and has since been approved for treatment of non-squamous, non-small cell lung cancer and metastatic breast cancer. (Source: FDA Website, 8 May, 2009)
The FDA announced the formation of a task force to develop recommendations for enhancing the transparency of the agency's operations and decision-making process. To support the efforts of the task force, the FDA issued a Federal Register notice announcing a public meeting to solicit recommendations on how the agency can make more available, useful and understandable information on its activities and decisions.
The task force will be chaired by Principal Deputy Commissioner Joshua Sharfstein, MD, and will include center directors, the associate commissioner for regulatory affairs, chief scientist, and the chief counsel. The Transparency Task Force will amongst other things:
The establishment of the task force follows President Obama's January 21, 2009 memorandum directing executive agencies to find new ways of making information available to the public rapidly and in a form that is easily accessible and user-friendly. (Source: FDA Website, 2 June, 2009)
The FDA announced the approval of Palladia (toceranib phosphate), manufactured by Pfizer Animal Health, the first drug developed specifically for the treatment of cancer in dogs. Palladia is approved to treat canine cutaneous (skin-based) mast cell tumors, a type of cancer responsible for about 1 out of 5 cases of canine skin tumors. The drug is approved to treat the tumors with or without regional lymph node involvement.
Palladia is a tyrosine kinase inhibitor and works in two ways: by killing tumor cells and by cutting off the blood supply to the tumor. In a clinical trial, Palladia showed a statistically significant difference in tumor shrinkage when compared with an inactive substance (placebo).
All cancer drugs now used in veterinary medicine originally were developed for use in humans and are not approved for use in animals. Cancer treatments used in animals are used in an "extra-label" manner as allowed by the Animal Medicinal Drug Use Clarification Act of 1994.
While canine mast cell tumors often appear small and insignificant, they can be a very serious form of cancer in dogs. Some mast cell tumors are easily removed without the development of any further problems, while others can lead to life threatening disease. (Source: FDA Website, 3 June, 2009)
The FDA approved Caldolor, manufactured by Cumberland Pharmaceuticals of Nashville, TN, the first injectable dosage form of the common pain medication ibuprofen, to treat pain and fever. Caldolor will be available for hospital use only. It is approved to be administered in 400 mg to 800 mg doses, over 30 minutes, every 6 hours for acute pain. To treat fever, the drug is approved in a 400 mg dose administered over 30 minutes, followed by 400 mg every 4 to 6 hours, or 100-200 mg every 4 hours, as necessary.
Caldolor should be used with caution in patients with congestive heart failure, kidney impairment, at risk of blood clots and those who have a prior history of ulcers or gastrointestinal bleeding. When used in such patients, attention to using the lowest effective dose for the shortest time period is important to reduce the risk of serious adverse events. The drug has also been associated with high blood pressure, serious skin reactions, and serious allergic reactions. (Source: FDA Website, 11 June, 2009)
On May 1, 2009, the FDA warned consumers regarding products related to the 2009 H1N1 flu virus offered on the Internet. The products involved are those that are promoted and marketed to diagnose, mitigate, prevent, treat, or cure the 2009 H1N1 flu virus but are not approved, cleared, or authorized by the FDA. The agency advised operators of offending Web sites that they must take immediate action to ensure that they are not marketing products intended to diagnose, mitigate, prevent, treat, or cure the 2009 H1N1 flu virus that have not been cleared, approved, or authorized by the FDA.
Since then, the FDA has issued more than 50 warning letters to offending Web sites and as a result, more than 66 percent of these Web sites have removed the offending claims and/or products. Examples of unapproved, uncleared, or unauthorized products targeted by the FDA include a shampoo that claimed to protect against the H1N1 flu virus and a dietary supplement that claimed to protect infants and young children from contracting the H1N1 flu virus.
The FDA's warning letters are consistent with an aggressive strategy the agency put into place to protect consumers from individuals or businesses that promote fraudulent claims for products in an attempt to take advantage of the public's concerns about the 2009 H1N1 flu virus. (Source: FDA Website, 15 June, 2009)
The FDA has approved Novartis AG's Ilaris (canakinumab) for the treatment of children and adults with cryopyrin-associated periodic syndrome (CAPS), which includes a number of rare but life-long auto-inflammatory disorders with debilitating symptoms and limited treatment options. The FDA granted priority review to Ilaris based on its potential to meet an important clinical need for patients with CAPS.
CAPS is caused by a single gene mutation that leads to overproduction of interleukin-1 beta (IL-1ß), which causes sustained inflammation and tissue damage. Symptoms, such as debilitating fatigue, rash, fever, headaches, joint pain and conjunctivitis, can be present from birth or infancy, and can occur daily throughout patients' lives. Long-term consequences may be serious and potentially fatal, including deafness, bone and joint deformities, central nervous system damage leading to visual loss, and amyloidosis resulting in renal failure and early death.
Ilaris, previously known as ACZ885, is a fully human monoclonal antibody that rapidly and selectively blocks IL-1ß. "Until now, treatments for CAPS patients have been limited to traditional inflammatory-disease medications that work by suppressing the entire immune system, and newer therapies that control the disease better but require more frequent injections," said Hal Hoffman, MD, Associate Professor of Pediatrics and Medicine at University of California, San Diego.
CAPS comprises three disorders of increasing severity: FCAS, MWS and neonatal-onset multisystem inflammatory disease (NOMID). There are believed to be approximately 300 cases in the US, but many patients may remain undiagnosed due to poor disease recognition. A clinical study is ongoing to evaluate the potential of Ilaris to treat patients with NOMID. There are currently no approved therapies for the treatment of NOMID. (Source: Novartis Website, 18 June, 2009)
Chapter Manager: Amy Poole, CAMI - Tel: 1.781.647.4773 and E-mail: email@example.com