European Approval for Roche’s Treatment for Rare Disease


Roche today 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.

MabThera is the first biologic therapy approved by the European Commission for PV and the first major advancement in the treatment of the disease in more than 60 years. Following approval by the US Food and Drug Administration (FDA) in June 2018 and today’s decision, MabThera is now approved to treat four autoimmune diseases in the US and Europe.

We’re pleased to bring the first biologic medicine to the more than 50,000 people in Europe suffering from pemphigus vulgaris. This MabThera approval provides a much needed new treatment that has been shown to provide higher remission rates than corticosteroids alone, which can cause debilitating side effects,

said Sandra Horning, MD,
Roche’s Chief Medical Officer
and Head of Global Product Development.

The European approval is based on data from the phase III Ritux 3 trial, a Roche-supported randomised controlled study, conducted in France, which evaluated MabThera plus a tapering regimen of oral corticosteroids (CS) 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 CS for two or more months. The study demonstrated that 89.5% of people with PV treated with MabThera, in combination with short-term oral CS treatment, achieved complete remission without the use of CS for two or more months, compared to 27.8% of people with PV receiving CS alone, the current standard of care. The results of the Ritux 3 trial were published in The Lancet in March 2017 [1].

The Roche-sponsored phase III multicentre, randomised, double-blind PEMPHIX study, evaluating the efficacy and safety of MabThera compared with mycophenolate mofetil (MMF), an immunosuppressant, in patients with moderate to severe PV, is ongoing.

Recently, an international panel of experts, the International Bullous Disease Group, published new recommendations on the diagnosis and management of pemphigus in the Journal of the American Academy of Dermatology [2], and recommended the use of an anti-CD20 monoclonal antibody, such as MabThera combined with a tapering regimen of oral CS, as a first-line therapy option for moderate to severe pemphigus.

1. 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.
2. Murrell DF, et al. Diagnosis and Management of Pemphigus: recommendations by an International Panel of Experts. Journal of the American Academy of Dermatology. February 10, 2018.