European Commission approves Roche’s MabThera for rare autoimmune disease

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

“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.”

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.^[3] The results of the Ritux 3 trial were published in The Lancet in March 2017.^[4]

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.^[5]

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, 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.

Pemphigus vulgaris is an autoimmune, blistering disease, occurring within the epidermis, 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 globally.^[7]

Ritux 3 is a Roche-supported phase III, prospective, multicentre, parallel-group, open-label randomised trial (NCT00784589), conducted in France by the French Study Group on Autoimmune Bullous Diseases. It was designed to evaluate MabThera plus a tapering regimen of oral corticosteroid (CS) treatment compared to a standard dose of CS monotherapy 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 corticosteroids for two or more months.

A phase III, randomised, double-blind, double-dummy, active-comparator, parallel-arm multicentre study (PEMPHIX, NCT02383589), designed to evaluate the efficacy and safety of MabThera compared with mycophenolate mofetil (MMF), an immunosuppressant, in patients with moderate to severe active pemphigus vulgaris requiring 60-120 mg/day oral prednisone (or equivalent).^[5]

MabThera (Rituxan in the US) in combination with methotrexate is indicated for the treatment of adults with severe active rheumatoid arthritis (RA) who have had an inadequate response or intolerance to other disease-modifying anti-rheumatic drugs (DMARD) including one or more tumour necrosis factor (TNF) inhibitor therapies.

MabThera/Rituxan, in combination with glucocorticoids, is indicated for the treatment of adults 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.

Source: Company Press Release