Several new drugs used to treat different types of cancer are to be included in the national health insurance (NHI) program for the first time later this year, a government agency said on Tuesday, September 8.
In a press release, the National Health Insurance Administration (NHIA) announced that following an Aug. 20 meeting, it was decided that new medicines for treating triple-negative breast cancer, ovarian cancer, follicular lymphoma and medullary carcinoma will be covered by the program in November.
In addition, rituximab, a biologic agent that can treat pemphigus vulgaris, will also be added to the program, benefiting about 600 patients suffering from the blistering autoimmune disease that affects the skin and mucous membrane. Currently, severe pemphigus vulgaris patients can only be treated with steroids which often have noticeable side effects, Huang said.
Genentech, a member of the Roche Group, announced data this week from the Phase III PEMPHIX study evaluating the efficacy and safety of Rituxan® (rituximab) compared to mycophenolate mofetil (MMF) in adults with moderate to severe pemphigus vulgaris (PV). The study met the primary endpoint at week 52 and demonstrated that Rituxan is superior to MMF, with 40.3% of patients treated with Rituxan achieving sustained complete remission (CR) without the use of steroids for 16 consecutive weeks or more, compared to 9.5% in the MMF arm (p<0.0001). All secondary endpoints were statistically significant in favor of Rituxan: lower cumulative oral corticosteroid dose (mean difference: 1595 mg; p=0.0005), fewer flares (6 vs. 44, p<0.0001), a greater likelihood of sustained CR (hazard ratio [HR]=4.83; p=0.0003), a lesser likelihood of flare (HR=0.15; p<0.0001) and a greater improvement in the Dermatology Life Quality Index (DLQI) at week 52 (estimated mean change from baseline -8.87 vs. -6.00, p=0.0012) compared to the MMF arm. Adverse events were generally consistent with those seen in previous Rituxan clinical studies in PV and other approved autoimmune indications.
“The approval of Rituxan for the treatment of pemphigus vulgaris was the first major advancement in the treatment of this rare, serious disease in more than 60 years,” said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development. “The PEMPHIX study showed that 40% of people in the study could achieve complete remission from painful blistering without the need for corticosteroids for 16 weeks or more and that Rituxan may be a superior treatment option to mycophenolate mofetil.”
The study is ongoing, with patients participating in a 48-week safety follow-up period after treatment completion or discontinuation.
Our sixth story in the Patient Journey Series comes from Toby:
When I was diagnosed with pemphigus vulgaris (PV) in late 2010, like most patients I’d never heard of the disease. Also, like most, I’d endured about six months of worsening symptoms before finding a doctor who was able to help me. After starting me on medication, the next thing my dermatologist did was to tell me about the IPPF. He encouraged me to reach out to them if I needed support, but I shelved the idea for a long time because I was sure I could manage by myself. I had the mistaken idea that if I followed my doctor’s instructions, I would “get better,”and soon.
Having PV was a wake-up call for me, however, as the disease proved to be tenacious, unpredictable, and unmanageable in my case on prednisone and CellCept alone. I embarked on a series of rituximab infusions ⎯ four over the past eight years ⎯ which have helped to control my symptoms. But the most important part of that wake-up call was the realization that I couldn’t go it alone. I called the IPPF nearly a year after diagnosis and talked to a Peer Health Coach, and the cloud lifted right away. For a while I attended support group meetings in the New York area. It was good to meet others who were facing the same challenges I was.
When I moved to New Hampshire three years later, I researched dermatologists to find one who was familiar with PV. Lo and behold, one turned up at UVM Medical Center in Burlington, VT, who had worked with IPPF Medical Advisory Board Member Dr. Victoria Werth at the University of Pennsylvania and co-authored an article on blistering diseases. I emailed Dr. Werth and asked her if she recommended this doctor, and she sent a thoughtful, encouraging response. I remained under the care of that doctor for five years until this year, when I moved to North Carolina.
This was a lucky move in many ways. Last October I attended the IPPF annual conference in Raleigh, NC, co-hosted by Dr. Donna Culton at UNC’s School of Medicine, and she was incredibly knowledgeable and inspiring. Because the IPPF set up the conference to make it easy for patients, staff, and physicians to mix, I was able to have one-on-one conversations with anyone I liked. There were cocktail hours, meals, and time between presentations, and everyone was approachable. I sought out Dr. Culton to say hello and chat with her. Afterwards, I told my daughter Kate, who lives in the area and had attended the conference with me, that I could do worse than moving down to NC and having Dr. Culton as my dermatologist. Then I made it happen. Two weeks before I packed up my car and drove the 800 miles with my cat to my new home, I had an appointment with Dr. Culton on the calendar!
Having excellent care and support (and even an infusion center) right here in my neighborhood gives me a sense of security and well-being, and I can trace that support directly back to the IPPF, who helped me find the way. Thank you, IPPF!
Every day, our patient services team hears stories from our community about what it’s like to live with pemphigus and pemphigoid. From getting diagnosed to finding the right doctor to thriving post-treatment, many patients express similar frustrations. And yet, there’s a common hope that runs through many of the stories we hear at the IPPF.
Each week through August and September, we’re featuring a story that highlights a specific part of the patient journey. OUR HOPE is that by sharing stories from our community, more patients and caregivers will realize they are not alone.
Genentech announced this week that positive top line results from the Roche-sponsored Phase III PEMPHIX study evaluating the efficacy and safety of Rituxan® (rituximab) compared to mycophenolate mofetil (MMF) in adults with moderate to severe pemphigus vulgaris (PV). The study met the primary endpoint, and demonstrated that Rituxan is superior to MMF in achieving sustained complete remission.
“The PEMPHIX study provides additional clinical evidence for the use of Rituxan for the treatment of pemphigus vulgaris,” said Sandra Horning, M.D., chief medical officer and head of Global Product Development. “These data also demonstrated that Rituxan may provide complete remission rates and successful tapering of corticosteroid therapy that is superior to MMF in adults with pemphigus vulgaris.”
On March 15, 2019, Roche announced that the European Commission has approved MabThera® (rituximab) for the treatment of adults with moderate to severe pemphigus vulgaris (PV), a rare condition characterised by progressive painful blistering of the skin and/or mucous membranes. Extensive blistering can lead to serious, life-threatening fluid loss, infection and/or death.
MabThera is the first biologic therapy approved by the European Commission for PV and the first major advancement in the treatment of the disease in more than 60 years. Following approval by the US Food and Drug Administration (FDA) in June 2018 and today’s decision, MabThera is now approved to treat four autoimmune diseases in the US and Europe.
“We’re pleased to bring the first biologic medicine to the more than 50,000 people in Europe suffering from pemphigus vulgaris,” said Sandra Horning, MD, Roche’s Chief Medical Officer and Head of Global Product Development. “This MabThera approval provides a much needed new treatment that has been shown to provide higher remission rates than corticosteroids alone, which can cause debilitating side effects.”
The European approval is based on data from the phase III Ritux 3 trial, a Roche-supported randomised controlled study, conducted in France, which evaluated MabThera plus a tapering regimen of oral corticosteroids (CS) compared to a standard dose of CS alone, as a first-line treatment in patients with newly diagnosed moderate to severe pemphigus. The primary endpoint of the study was complete remission at month 24 without the use of CS for two or more months. The study demonstrated that 89.5% of people with PV treated with MabThera, in combination with short-term oral CS treatment, achieved complete remission without the use of CS for two or more months, compared to 27.8% of people with PV receiving CS alone, the current standard of care. The results of the Ritux 3 trial were published in The Lancet in March 2017.
On Thursday, June 7th, the FDA approved Rituxan for the treatment of adults with moderate to severe pemphigus vulgaris (PV).
Rituxan is the first biologic therapy approved by the FDA for PV and the first major advancement in the treatment of PV in more than 60 years. The FDA previously granted Priority Review, Breakthrough Therapy Designation and Orphan Drug Designation to Rituxan for the treatment of PV. With this decision, Rituxan is now approved to treat four autoimmune diseases.
“It is our hope that this announcement will open the door to approval for other indications in our diseases and usher in a renewed focus on available treatments,” said Marc Yale, Executive Director of the International Pemphigus & Pemphigoid Foundation.
The FDA approval is based on data from the Ritux 3 trial, a Roche-supported, randomized, controlled trial conducted in France that used Roche-manufactured, European Union (EU)-approved rituximab product as the clinical trial material. The study compared the Ritux 3 regimen (EU-approved rituximab product plus short-term corticosteroids [CS]) to CS alone as a first-line treatment in patients with newly diagnosed, moderate to severe pemphigus. The primary endpoint of the study was complete remission at month 24 without the use of steroids for two or more months. (Complete remission defined as complete epithelialization and absence of new and/or established lesions.)
Results of the study showed that 90 percent of PV patients treated with the Ritux 3 regimen met the endpoint, compared to 28 percent of PV patients treated with CS alone. These results supported the efficacy of Rituxan in treating patients with moderate to severe PV, while tapering off of CS therapy. These results were published in The Lancet in March 2017.
An international panel of experts called the International Bullous Disease Consensus Group recently provided new recommendations on the diagnosis and management of pemphigus in the Journal of the American Academy of Dermatology. Based on existing European treatment guidelines, a Delphi survey process was used to help achieve international expert consensus. The consensus includes the recommendation to use an anti-CD20 monoclonal antibody (Rituxan) and corticosteroids as first line therapy options for moderate to severe pemphigus.
The Role of the IPPF
The IPPF aims to serve as a primary source of information for you regarding this approved treatment and is available to help answer your questions in the upcoming months. If you are considering Rituxan as a potential therapy, please consult your healthcare provider. Inform them of your medical history, and ask about the potential side effects.
The IPPF’s Peer Health Coaches (PHC) are pemphigus and pemphigoid patients who help more than 1,200 patients and caregivers each year. These specially trained PHCs reduce patient anxiety and uncertainty while providing unbiased disease and treatment knowledge. You can find our PHCs engaging the community through social media, emails, phone calls, and in-person support. The goal of our PHC program is to ensure we help every person who needs assistance in the shortest amount of time possible.
Genentech Access Solutions
Genentech is the drug company that produces Rituxan (rituximab). Genentech Access Solutions is a resource for people considering Rituxan as a treatment option. It may be worth contacting Access Solutions directly regardless of whether or not you have health insurance.
Genentech recently announced an important FDA decision that could potentially impact future treatment options for pemphigus. Here at the IPPF, it’s especially exciting when we get to share good news related to research and treatments. The full press release from Genentech can be found here. The following is an excerpt:
The U.S. Food and Drug Administration (FDA) has accepted Genentech’s Supplemental Biologics License Application (sBLA) and granted Priority Review for the use of Rituxan® (rituximab) for the treatment of pemphigus vulgaris (PV). Last year, the FDA granted Breakthrough Therapy Designation and Orphan Drug Designation to Rituxan for the treatment of PV.
“We are committed to developing medicines for rare diseases with limited treatment options, such as pemphigus vulgaris,” said Sandra Horning, M.D., chief medical officer and head of Global Product Development. “We look forward to continued work with the FDA to hopefully provide patients with a new treatment for this serious and potentially life-threatening disease.”
The sBLA submission is based on data from a Roche-supported randomized trial conducted in France which evaluated Rituxan plus a tapering regimen of low dose oral corticosteroid (CS) treatment compared to a standard dose of CS alone as a first-line treatment in patients with newly diagnosed moderate to severe pemphigus. Results of the study show that Rituxan provides substantial improvement in pemphigus vulgaris remission rates and successful tapering and/or cessation of CS therapy. These results were published in The Lancet in March 2017. Genentech is currently conducting another Phase III study in PV which is evaluating Rituxan plus a tapering regimen of CS compared to Cellcept (PEMPHIX, NCT02383589).
My name is Marlis Lippow, and I reside in Northern California. I participated in a randomized, double-blind, double dummy study evaluating rituximab infusions vs. 2,000 mg of mycophenolate mofetil (Cellcept®). I had previously received rituximab infusions so I had a pretty good idea of what to expect if I was to again receive it. I had three previous rounds and my doctor said the effects should last about six months, after which I would probably need another round. I was lesion-free for about seven months before the lesions started to return. I also was on CellCept and prednisone, so I know how those affect me.
Learning About the Trial
My doctor mentioned a clinical trial, answered my many questions, and asked me to think about it. I returned a month later for a followup appointment and there was another doctor present. She was talking about the trial and it seemed she expected me to be a part of it. I was still unsure and had more questions. She did explain that before I could be accepted, I’d undergo a screening (ECG, chest x-ray, and blood work). That was great, I would find out how I am doing. If I passed the tests, I could decide if I wanted to participate. The trial included a stipend, $50 for each session for gas and parking. That sounded good since I live about 45 miles away.
Making the Decision
My doctor and I discussed the pros and cons of the assorted medications and what I’d need to take if I did not participate in the trial but still needed rituximab. Either way, the side effects are not pleasant. Basically, we talked about the lesser of the two evils.
I learned my doctor is referred to as the Principal Investigator (PI) and the other doctor is the Sub-Investigator (SI). The SI would be seeing me every month. Since the SI is not my primary doctor, it is very important she have a complete grasp of my medical history. During my visits I would have blood work and urinalysis done. The PI would get the test results and be aware of my progress and any possible problems. If he felt there was a concern, he would end my participation in the study.
I also learned I could opt out of the study at any time if I became uncomfortable.
In the end, I chose to be in the trial.
My Trial Experience
Throughout the study, I felt my doctor was most concerned about my well-being, as he should be. He even called me in between visits and that gave me a good, positive feeling.
I received two initial infusions two weeks apart. After about five months, I received two more, also two weeks apart. By the third round I no longer had any sores. I was told that I was “controlled” and after the last infusion, I’d be in remission! My doctor told me this remission should last anywhere from six months to three years. I am hoping it will be longer!
Don’t be worried about the infusions. The infusion nurses are angels and take wonderful care of you! They are kind, let you know what to expect, and give you an idea of how you will feel. If you have any questions, you will have the phone numbers for the PI and SI and are encouraged to use them.
Clinical trials are not for everyone. In fact, there are many qualifying and disqualifying criteria set by the drug manufacturer. I encourage everyone to consider participating in a trial to help advance research on new and emerging pemphigus and pemphigoid treatments. While the short-term benefits help us now, the long-term benefits may change the lives of patients for years to come.
