FAQ about BENLYSTA
Updated: April 4, 2011
The Lupus Foundation of America (LFA), Philadelphia Tri-State Chapter applauds the U.S. Food and Drug Administration (FDA) decision to approve BENLYSTA® for the treatment of lupus, a potentially life-threatening and unpredictable autoimmune disease. BENLYSTA is the first treatment approved for lupus in more than 52 years, and is the first drug ever approved that was specifically developed to treat the disease. It signals a new era of improved treatment for the disease, and offers hope for a better quality of life for the over 40,000 people in the Philadelphia Tri-State Region and an estimated 1.5 million Americans living with lupus.
“This is a historic day for the millions of people with lupus and their families around the world who have waited more than 50 years for a treatment breakthrough for lupus,” said Sandra C. Raymond, President and CEO of the national Lupus Foundation of America. “BENLYSTA is the first drug ever to be specifically developed to treat lupus, and is a significant first step toward reaching our goal of developing an arsenal of safe, effective, and tolerable treatments.” Click below to read the LFA’s complete statement on the approval of BENLYSTA.
“We know that people with lupus and their families will have many questions about what this historic decision means for them or their loved ones,” said Annette Myarick, CEO of the Lupus Foundation of America’s Philadelphia Tri-State Chapter. “Staff and volunteers from our Chapter, along with LFA’s health educators, are ongoing resources for people with lupus in our community, are here to answer questions, and can provide the information and resources they need to live with lupus.”
1. What is BENLYSTA?
BENLYSTA is a human monoclonal antibody that was approved for the treatment of lupus by the U.S. Food and Drug Administration (FDA) on March 9, 2011. A monoclonal antibody is a type of protein made in the laboratory that is developed to find and attach to only one type of substance in the body.
2. How does BENLYSTA work?
BENLYSTA is a human monoclonal antibody that specifically recognizes and blocks the biological activity of B-lymphocyte stimulator, or BLyS® (pronounced bliss), a naturally occurring protein which was discovered by scientists at Human Genome Sciences (HGS). Elevated levels of BLyS prolong the survival of B cells which can contribute to the production of autoantibodies – antibodies that target the body’s own tissues. Studies have shown that BENLYSTA can reduce autoantibody levels and help control autoimmune disease activity.
3. Who developed BENLYSTA?
BENLYSTA was co-developed by Human Genome Sciences (HGS) and GlaxoSmithKline (GSK).
4. What clinical research has been conducted on BENLYSTA?
HGS conducted two large scale Phase III clinical trials, BLISS-52 and BLISS 76. Both trials had positive top-line results and met their primary endpoints. The design of the two trials was similar, but the durations of therapy in the two trials were different, 52 weeks for BLISS-52, and 76 weeks for BLISS-76. The BLISS-52 study was conducted primarily in Asia, South America, and Eastern Europe, and BLISS-76 was conducted mostly in North America and Europe.
5. What does an FDA approval of BENLYSTA mean for people with lupus?
BENLYSTA represents a breakthrough in the treatment of lupus. BENLYSTA is the first drug approved to treat lupus in more than 50 years AND is the first drug developed specifically for lupus since the disease was discovered more than a century ago! Successful treatment of lupus will require an arsenal of safe, effective, and tolerable treatments. The approval of BENLYSTA is a significant first step toward reaching that goal.
6. What makes BENLYSTA different from other lupus treatments?
BENLYSTA is the FIRST FDA-approved medication specifically designed for the treatment of lupus. BENLYSTA targets specific immune cells, rather than the blanket approach of other therapies which suppress the entire immune system. Currently approved medications for lupus are borrowed from other diseases and conditions; other treatments are used off-label, which means they were never approved by the FDA for lupus. Many of these treatments have serious and devastating side effects.
These drugs include high doses of steroids, antimalarial medications, immunosupressive drugs, and organ-rejection drugs.
7. When will BENLYSTA be available for patients?
According to Human Genome Sciences, BENLYSTA will be available to physicians and patients before the end of March 2011. It is best to talk to your doctor about the availability of BENLYSTA and if it is right for you.
8. Where can I find out more information about BENLYSTA?
BENLYSTA Gateway is a centralized resource for patients and health care providers for one-on-one services and support. For more information call 1-877-4-BENLYSTA, Monday through Friday, 8AM to 8PM (EST). You can also visit www.benlysta.com for more information, or www.benlystahcp.com.
9.Who should take BENLYSTA? Will it work for everyone?
Each person with lupus is unique, and BENLYSTA will not be an option for everyone. You will need to discuss with your doctor if BENLYSTA may be an appropriate treatment option for you.
BENLYSTA is approved for the treatment of adult patients with active, autoantibody-positive systemic lupus erythematosus (SLE) who are receiving standard therapy.
The label for BENLYSTA includes the following limitations of use: The efficacy of BENLYSTA has not been evaluated in patients with severe active lupus nephritis or severe active central nervous system lupus, and has not been studied in combination with other biologics or intravenous cyclophosphamide. Use of BENLYSTA is therefore not recommended in these situations.
9. Is BENLSYTA approved for use in children with lupus?
No. Additional studies are required before BENLYSTA can be approved for use in children.
10. Is BENLYSTA effective?
Lupus is a complex disease, and BENLYSTA is not right for everyone. Individuals with lupus are on strong background medications which impact the margin of effectiveness between the placebo and treatment group. In two major clinical trials, BENLYSTA achieved statistical significance over these medications. There are also the first lupus trials to meet is primarty endpoints.
11. Is BENLYSTA effective for African-Americans?
The population size of African Americans in the study was not large enough to determine the effectiveness of BENLYSTA in that population. Additional research is needed. Human Genome Sciences and GlaxoSmithKline have already committed to doing a follow-up study that will evaluate the effectiveness of BENLYSTA in African Americans. Weencourage you to visit the LFA's Center for Clinical Trials Education (lupus.org/clinicaltrials) to learn more about future trials in your community through the Lupus Research Registry.
12. Is BENLYSTA approved for use in children with lupus?
No. Additional studies are required before BENLYSTA can be approved for use in children.
13. Will there be any further clinical trials on BENLYSTA?
Yes, additional trials are being planned. To learn more about clinical trials, visit lupus.org/clinicaltrials. On the Website you can also join our Lupus Research Registry to stay informed about lupus trials in your community.
14. Is BENLYSTA effective for individuals with lupus who have organ involvement?
BENLYSTA was not studied for use in individuals with severe lupus nephritis, or active central nervous system involvement.
15. What side effects have been found with BENLYSTA?
The most commonly reported adverse reactions with BENLYSTA were nausea, diarrhea, pyrexia, nasopharyngitis, bronchitis, insomnia, pain in extremity, depression, migraine, and pharyngitis.
16. How is BENLYSTA administered?
BENLYSTA is administered through an IV (intravenous) infusion directly into the vein.
17. Should I receive vaccinations if I am taking BENLYSTA?
Overall, vaccinations are considered to be safe and effective for people with lupus. However, it is not recommended for people with lupus to receive vaccines with a form of attenuated (weakened, but still life) virus and should not be exposed to recent recipients of live-vaccines such as oral polio virus (OPV). It is recommended that individuals always speak with their physician prior to receiving a vaccine.
18. How much does BENLYSTA cost?
The cost of BENLYSTA is in line with similar therapies. It will be important for insurance companied to cover BENLYSTA since it is the first and only treatment developed specifically for lupus. The LFA will be working to ensure that insurance companies cover BENLYSTA on their formularies. We also encourage you to inquire about financial assistance programs at your local hospital. Human Genome Sciences and GlaxoSmithKline will also be offering a Patient Assistance Program. For additional information and questions about eligibility call BENLYSTA Gateway at 1-877-4-BENLYSTA, or visit www.benlysta.com.
19. Will there be any patient assistance programs available for BENLYSTA?
Yes, Human Genome Sciences and GlaxoSmithKline will be offering a Patient Assistance Program (PAP). For individuals who are uninsured and meet eligibility requirements the PAP will provide the medication free of charge. For commercially insured and underinsured patients who are unable to afford the cost-share with BENLYSTA, and do not qualify for the PAP, BENLYSTA Gateway can help determine eligibility for assistance. For more additional information and questions about eligibility call BENLYSTA Gateway at 1-877-4-BENLYSTA, or visit www.benlysta.com.
20. Are there other treatments being researched for lupus?
There are a number of pioneering biotechnology and pharmaceutical companies involved in the research and development of new therapies for lupus, and there are several promising treatments in the near-term pipeline. The historic decision by the FDA will likely stimulate further investment in additional clinical trials in lupus. We can’t make new treatments a reality without your support. You can help by learning more about clinical trials and volunteering to participate in a clinical trial. To learn more, visit the LFA’s Center for Clinical Trial Education at www.lupus.org/clinicaltrials.
21. Why has it taken so long to find a treatment for lupus?
Lupus is a complex disease. It can affect multiple organ systems and symptoms can range in severity from one day to the next. Also, lupus affects each person differently, with varying responses to treatment. The complexity and heterogeneity of the disease presents challenges in evaluating potential new therapies. With each research study, regardless of the outcome, there are new discoveries that help pave the way for new therapies.
22. What are the research barriers to developing new treatments for lupus?
The unique biology of this disease has made it difficult to assemble uniform patient groups to test new treatments, and the various background medications taken by patients have affected clinical trial results. The pharmaceutical and biotechnology industries have lacked a clearly defined pathway for measuring outcomes in lupus clinical trials as required by the FDA. In addition, the instruments used by investigators to assess disease activity are also extremely complex.
23. What is the LFA doing to further the development of new treatments for lupus?
As part of the LFA’s National Research Program: Bringing Down the Barriers™, the LFA has recently launched new initiatives that will standardize and improve clinical trial design, allowing future studies to be completed successfully. These initiatives include a new worldwide Lupus Research Registry through the LFA’s Center for Clinical Trials Education, and a Web-based service to standardize research training on instruments used to assess disease activity in clinical studies and practice. The LFA also is partnering with key stakeholders from industry, government, and the scientific community to evaluate data from previous lupus clinical trials with the goal to improve the design of future studies. These initiatives, along with the approval of BENLYSTA, will help provide a pathway toward additional treatments specifically developed for lupus.