Basel, 24 March 2017
FDA grants breakthrough therapy designation for MabThera/Rituxan (rituximab) in pemphigus vulgaris
- Pemphigus vulgaris (PV) is a life-threatening, autoimmune condition with limited treatment options1
- Roche is currently enrolling a Phase III clinical trial in patients with moderate to severe PV
- The FDA previously granted orphan drug designation to MabThera/Rituxan for PV
- Fifteenth breakthrough therapy designation granted to Roche medicines since 2013
Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that the US Food and Drug Administration (FDA) has granted breakthrough therapy designation status to MabThera/Rituxan® (rituximab) for pemphigus vulgaris, a rare, serious and life-threatening condition characterised by progressive painful blistering of the skin and mucous membranes.2 FDA breakthrough therapy designation is intended to expedite the development and review of medicines with early evidence of potential clinical benefit in serious diseases and to help ensure that patients receive access to medicines as soon as possible. Roche is currently enrolling a Phase III study in pemphigus vulgaris (PEMPHIX, NCT02383589), a disease for which there are limited treatment options.1
“People with pemphigus vulgaris need more options and we look forward to working with the FDA to make MabThera/Rituxan available to patients with this potentially deadly disease,” said Sandra Horning, MD, Roche’s Chief Medical Officer and Head of Global Product Development. “We are committed to developing therapies that target B cells in areas of unmet need across a range of immune and malignant diseases, including new antibody constructs that enhance efficacy, safety or both.”
This is the fifteenth breakthrough therapy designation granted to Roche medicines since 2013.
Breakthrough therapy designation was granted based on data from a Roche-supported randomised trial conducted in France which evaluated MabThera/Rituxan plus oral corticosteroid (CS) treatment compared to CS as a first-line treatment in patients with moderate to severe pemphigus.3 Results of the study, published in The Lancet, show that MabThera/Rituxan may provide substantial improvement in pemphigus vulgaris remission rates and successful tapering and/or cessation of CS therapy.3
In 2015, the FDA granted orphan drug designation to MabThera/Rituxan for the treatment of pemphigus vulgaris.
About pemphigus vulgaris
Pemphigus vulgaris is an autoimmune, intraepidermal, blistering disease affecting the skin and mucous membranes.1 It is the most common type of a group of autoimmune disorders collectively called pemphigus.2 It is estimated that around three in every 100,000 people are diagnosed with this disease.4
About the PEMPHIX study
Phase III, randomised, double-blind, double-dummy, active-comparator, parallel-arm multicentre study (PEMPHIX, NCT02383589) was designed to evaluate the efficacy and safety of MabThera/Rituxan compared with mycophenolate mofetil (MMF) in patients with moderate to severe active pemphigus vulgaris requiring 60-120 mg/day oral prednisone (or equivalent).5
MabThera/Rituxan is approved to treat rheumatoid arthritis (RA) in combination with another prescription medicine called methotrexate, to reduce the signs and symptoms of moderate to severe active RA in adults, after treatment with at least one other medicine called a tumour necrosis factor (TNF) antagonist has been used and did not work well enough.
MabThera, in combination with glucocorticoids, is indicated for the induction of remission in adult patients with severe, active granulomatosis with polyangiitis (Wegener’s, GPA) and microscopic polyangiitis (MPA).
People with serious infections should not receive MabThera/Rituxan. It is not known if MabThera/Rituxan is safe or effective in children.
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1 Medscape. Pemphigus Vulgaris. Available at: http://emedicine.medscape.com/article/1064187-overview. Accessed 7 March 2016
2 Healthline. Pemphigus Vulgaris. Available at: http://www.healthline.com/health/pemphigus-vulgaris#Overview1. Accessed 7 March 2016
3 Joly P, et al. First-Line Rituximab Combined with Short-Term Prednisone Versus Prednisone Alone for the Treatment of Pemphigus (Ritux 3): A Prospective, Multicentre, Parallel-Group, Open-Label Randomised Trial. The Lancet. March 22, 2017
4 National Health Service. Pemphigus Vulgaris. Available at: http://www.nhs.uk/Conditions/Pemphigus-vulgaris/Pages/Definition.aspx. Accessed 24 November 2015
5 ClinicalTrials.gov. A Randomized, Double-Blind Study to Evaluate the Efficacy and Safety of Rituximab Versus MMF in Patients With Pemphigus Vulgaris. Available at: https://clinicaltrials.gov/ct2/show/NCT02383589?term=pemphigus+vulgaris&recr=Open&rank=2. Accessed 7 March 2016