FDA approves Roche’s MabThera/Rituxan (rituximab) for pemphigus vulgaris

• MabThera/Rituxan is the first FDA-approved treatment for moderate to severe pemphigus vulgaris (PV) in more than 60 years
• FDA previously granted Priority Review, Breakthrough Therapy Designation and Orphan Drug Designation to MabThera/Rituxan for the treatment of PV
• MabThera/Rituxan is now FDA-approved to treat four autoimmune diseases

Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that the US Food and Drug Administration (FDA) has approved MabThera/Rituxan® (rituximab) for the treatment of adults with moderate to severe pemphigus vulgaris (PV), a rare, serious, potentially life-threatening condition characterised by progressive painful blistering of the skin and mucous membranes.¹

MabThera/Rituxan is the first biologic therapy approved by the FDA for PV and the first major advancement in the treatment of the disease in more than 60 years. The FDA previously granted Priority Review, Breakthrough Therapy Designation and Orphan Drug Designation to MabThera/Rituxan for the treatment of PV. With today’s FDA decision, MabThera/Rituxan is now approved to treat four autoimmune diseases.

“Today’s decision by the FDA provides the first approved treatment option in more than 60 years for patients with pemphigus vulgaris, a potentially life-threatening disease,” said Sandra Horning, MD, Chief Medical Officer and Head of Global Product Development. “We are pleased to offer Rituxan as a new and effective therapy to patients with this serious condition.”

The FDA approval is based on data from the Ritux 3 trial, a Roche-supported, randomised, controlled trial conducted in France which evaluated European Union-approved rituximab product (MabThera) plus a tapering regimen of 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. The primary endpoint of the study was complete remission at month 24 without the use of steroids for two or more months. Results of the study showed that 90 percent of PV patients treated with MabThera met the endpoint, compared to 28 percent of PV patients treated with CS alone. These results supported the efficacy of MabThera/Rituxan in treating adults with moderate to severe PV, while tapering off of CS therapy. These results were published in The Lancet in March 2017. ²

Recently an international panel of experts, the International Bullous Disease Consensus Group, provided new recommendations on the diagnosis and management of pemphigus in the Journal of the American Academy of Dermatology.³ Based on existing European treatment guidelines, a Delphi survey process was used to help achieve international expert consensus. The consensus includes the recommendation to use an anti-CD20 monoclonal antibody (MabThera/Rituxan) and corticosteroids as first line therapy options for moderate to severe pemphigus.

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.⁴ It is estimated that around three in every 100,000 people are diagnosed with this disease.⁵

About the PEMPHIX study

Phase III, randomised, double-blind, double-dummy, active-comparator, parallel-arm multicentre study (PEMPHIX, NCT02383589) 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).³

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. Thirty 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 nine 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 2017 employed about 94,000 people worldwide. In 2017, Roche invested CHF 10.4 billion in R&D and posted sales of CHF 53.3 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 15 May 2018
2. 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
3. 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 15 May 2018
4. International Pemphigus & Pemphigoid Foundation. Pemphigus. Available at: http://www.pemphigus.org/research/clinically-speaking/pemphigus/. Accessed 15 May 2018
5. Skin Support, British Association of Dermatologists. Pemphigus Vulgaris. Available at: http://skinsupport.org.uk/conditions-details/pemphigus-vulgaris. Accessed 15 May 2018