• Rituxan Shown as Superior in Achieving Sustained PV Remission

  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

• NORD and Trio Health Publish New Book to Provide Critical Patient Insights Into the Burden of Living With and Treating Rare Diseases

  As a NORD IAMRARE™ Registry Partner, the IPPF is proud to share that a new book, “The Power of Patients: Informing Our Understanding of Rare Diseases” was published today by the National Organization for Rare Disorders, Inc. (NORD) and Trio Health.

• Akari Therapeutics Announces Positive Initial Phase II Clinical Data in Orphan Skin Disease Bullous Pemphigoid

  On April 23, 2019, Akari Therapeutics, Plc (Nasdaq: AKTX), a biopharmaceutical company focused on innovative therapeutics to treat orphan autoimmune and inflammatory diseases where the complement and/or leukotriene systems are implicated, announced positive initial Phase II clinical data from the first three of bullous pemphigoid (BP) patients in an ongoing clinical trial.

• European Commission Approves Roche’s MabThera (rituximab) for 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.

• Principia Biopharma Announces Positive Data From Phase 2 Pemphigus Vulgaris Trial at 2019 American Academy of Dermatology Annual Meeting in Late-Breaking Presentation

  On March 2, 2019, Principia Biopharma Inc. (Nasdaq: PRNB), a late-stage biopharmaceutical company dedicated to bringing transformative oral therapies to patients with significant unmet medical needs in immunology and oncology, today announced Phase 2 clinical data from the Believe-PV study for PRN1008 as part of the Late-breaking Research: Clinical Trials program at the American Academy of Dermatology (AAD) annual ...

• Principia Biopharma Announces the Acceptance of Data from Phase 2 Pemphigus Vulgaris Trial as a Late-Breaking Presentation at 2019 American Academy of Dermatology Meeting

  On February 22, 2019,  Principia Biopharma Inc. (Nasdaq: PRNB), a late-stage biopharmaceutical company dedicated to bringing transformative oral therapies to patients with significant unmet medical needs in immunology and oncology, announced that an abstract has been accepted for oral presentation at the Late-Breaking Research during the 2019 American Academy of Dermatology annual meeting in Washington, ...

• Breaking Bad: IgG4 in Autoimmunity

  Our laboratory researches a rare and debilitating autoantibody-mediated disease called pemphigus vulgaris, in which the immune system generates antibodies against desmogleins, which are proteins responsible for holding skin cells together.

• Syntimmune Announces FDA Orphan Drug Designation for SYNT001 in Treatment of Pemphigus

  On September 12, 2018, the U.S. Food and Drug Administration (FDA) granted an orphan drug designation to Syntimmune Inc.’s SYNT001 for the treatment of pemphigus.

• 2018 Highlight: Patient Education Conference

  The 2018 IPPF Patient Education Conference was an amazing opportunity for patients and their support systems to not only have direct access to IPPF staff, but also some of the leading minds who treat pemphigus and pemphigoid.  

• Principia Biopharma Reports Positive PRN1008 Phase 2 Top-Line Results and Initiates Phase 3 Pemphigus Program

  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 ...

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