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

About MabThera/Rituxan

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.

About Roche

Roche is a global pioneer in pharmaceuticals and diagnostics focused on advancing science to improve people’s lives. The combined strengths of pharmaceuticals and diagnostics under one roof have made Roche the leader in personalised healthcare – a strategy that aims to fit the right treatment to each patient in the best way possible.

Roche is the world’s largest biotech company, with truly differentiated medicines in oncology, immunology, infectious diseases, ophthalmology and diseases of the central nervous system. Roche is also the world leader in in vitro diagnostics and tissue-based cancer diagnostics, and a frontrunner in diabetes management. 
Founded in 1896, Roche continues to search for better ways to prevent, diagnose and treat diseases and make a sustainable contribution to society. The company also aims to improve patient access to medical innovations by working with all relevant stakeholders. Twenty-nine medicines developed by Roche are included in the World Health Organization Model Lists of Essential Medicines, among them life-saving antibiotics, antimalarials and cancer medicines. Roche has been recognised as the Group Leader in sustainability within the Pharmaceuticals, Biotechnology & Life Sciences Industry eight years in a row by the Dow Jones Sustainability Indices (DJSI).

The Roche Group, headquartered in Basel, Switzerland, is active in over 100 countries and in 2016 employed more than 94,000 people worldwide. In 2016, Roche invested CHF 9.9 billion in R&D and posted sales of CHF 50.6 billion. Genentech, in the United States, is a wholly owned member of the Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, Japan. For more information, please visit www.roche.com.

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References
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