Tag Archives: pemphigus vulgaris

Principia Biopharma Inc. (Nasdaq: PRNB), a clinical-stage biopharmaceutical company dedicated to bringing transformative oral therapies to patients with significant unmet medical needs in immunology and oncology, today announced positive top-line data from the completed open-label Phase 2 trial of PRN1008 in patients with pemphigus (including both pemphigus vulgaris (PV) and pemphigus foliaceus (PF)) and the initiation of a Phase 3 trial of PRN1008 in pemphigus.

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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.

Syntimmune recently announced positive preliminary results from its phase 1b proof-of-concept trial of SYNT001 in pemphigus vulgaris and foliaceus patients. It’s exciting for the IPPF to share good news related to research and treatments. The full press release from Syntimmune can be found here. The following is an excerpt:

 

Syntimmune, Inc., a clinical-stage biotechnology company developing antibody therapeutics targeting FcRn, today announced positive preliminary results from its Phase 1b proof-of-concept trial of SYNT001 in patients with pemphigus vulgaris and pemphigus foliaceus. The data showed clinically meaningful benefit of SYNT001, with a favorable safety and tolerability profile similar to that observed in the Phase 1a study.

“There remains a clear unmet need for a safe and fast-acting treatment for patients with pemphigus, who face serious symptoms and complications associated with their disease,” said Donna Culton, M.D., Ph.D., an assistant professor at the University of North Carolina School of Medicine. Culton presented preliminary results of the Phase 1b study at the International Investigative Dermatology conference being held on May 16-19, 2018 in Orlando, FL. “These preliminary data demonstrate safety as well as a rapid reduction in PDAI scores and lowering of IgG levels with treatment of SYNT001, which support further studies of this drug as a potential new therapeutic option,” Culton said.

Read Syntimmune’s press release, including additional information, here. 

image of an anitgenGenentech 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).

Read Genentech’s press release, including additional information and references, here. 

In January 2013 my husband was diagnosed with prostate cancer. Thankfully, after surgery he was cancer free. But in May 2015 our whole world seemed to turn upside down when he came home with a very unusual sore in his mouth. Fearing the cancer had returned, Tony went to our family doctor and was immediately sent to an oral surgeon for a biopsy. He had the results within days, but the outbreak spread like wildfire.

Last year was shaping up to be a very important year. I had just gotten an associate degree in journalism and was all set to transfer to a four-year university in the fall to pursue my B.A. On top of that, I turned 21 in February and was looking forward to savoring all the exciting things young adulthood had to offer. It was during this very same month that everything changed.

Working for the IPPF has been something I have been interested in doing for about five years, since my pemphigus vulgaris finally got under control. I knew from my first contact with the Foundation that this is an amazing group of people. I am proud of the way our community pulls together and rallies for each other; it’s amazing how we sincerely care about one another.

As a Patient Educator for the IPPF, I have the fantastic opportunity to travel around the country to different dental schools and give lectures on my journey with pemphigus vulgaris (PV). It’s an empowering experience to have a hundred people listen to my story at once. But it’s also important that the audience relates to me. I’m a person, not just a patient.

While you are seeing a qualified dermatologist who is treating you for your Pemphigus Vulgaris, Bullous Pemphigoid, Pemphigus Foliaceus, Mucous Membrane Pemphigoid, etc. you might also be seeing your own dentist, OB/GYN, internist, ophthalmologist or ear/nose/throat specialist.

Please be sure that all of your doctors are aware of your condition and that they have access to your dermatologist.  It is important that they know the medications and dosage that you are taking for each medication.

All of your doctors need to be able to communicate with one another if necessary.  Being left in the dark will leave you at a disadvantage.  Also, if you are going to be scheduled for any major dental work, advise your dermatologist.  Depending on the procedure, your medications may be adjusted for a few days prior and a few days following to prevent any flare-ups.

Remember when you need us we are in your corner!

Pemphigus vulgaris (PV) is a paradigm of autoimmune disease affecting intercellular adhesion. The mechanisms that lead to cell–cell detachment (acantholysis) have crucial therapeutic implications and are currently undergoing major scrutiny. The first part of this review focuses on the classical view of the pathogenesis of PV, which is dominated by the cell adhesion molecules of the desmosome, namely desmogleins (Dsgs). Cloning of the DSG3 gene, generation DSG3 knock-out mice and isolation of monoclonal anti-Dsg3 IgG have aided to clarify the pathogenic mechanisms of PV, which are in part dependent on the fate of desmosomal molecules. These include perturbation of the desmosomal network at the transcriptional, translational, and interaction level, kinase activation, proteinase-mediated degradation, and hyper-adhesion. By the use of PV models, translational research has in turn helped shed light into the basic structure, function, and dynamics of assembly of desmosomal cadherins. The combined efforts of basic and applied research has resulted in tremendous advance into the understanding of epidermal adhesion and helped debunk old myths on the supposedly unique role of desmogleins in the mechanisms of cell–cell detachment in PV.

From: http://informahealthcare.com/doi/abs/10.3109/15419061.2013.763799