Category Archives: PemPress

Immune Pharmaceuticals Logo

The U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to bertilimumab for the treatment of bullous pemphigoid. The FDA’s Fast Track program is designed to facilitate the development and expedite the review of drugs to treat serious conditions that are unmet medical needs.

Pemphigus and pemphigoid (P/P) patients may agree that in the early months of their condition it is a very painful and difficult time. However, the following suggestions may be helpful like learning how to care for our skin in terms of taking and applying topical medications, cleansers, moisturizers, dressings, and staying comfortable.

FDA logo

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. 

It can be difficult to accept the news of a lifelong chronic disease. It may be easier to understand the diagnosis of appendicitis when a procedure is needed, or medication to treat a cold, or the need to use crutches for a couple of months because of a broken bone. But accepting a disease like asthma, diabetes, lupus, pemphigus, or pemphigoid may be more difficult. Managing your life first begins with a strategy to live with your medical condition.

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.