First-in-class medicine recommended for treatment of rare blood vessel inflammation

EMA has recommended granting a marketing authorisation in the European Union (EU) for Tavneos (avacopan), a first-in-class medicine to treat adult patients suffering from two forms of a rare multisystem autoimmune condition. Granulomatosis with polyangiitis and microscopic polyangiitis are forms of the autoimmune condition called anti-neutrophil cytoplasmic autoantibody (ANCA)-associated vasculitis, in which the immune system attacks small to medium-sized blood vessels in the body. Tavneos is to be used in combination with rituximab or cyclophosphamide - two medications used to treat certain autoimmune diseases and types of cancer.

ANCA-associated vasculitis can present throughout the body, but patients most frequently have symptoms in the respiratory tract and the kidneys. The recommendation for approval of Tavneos will provide patients an additional treatment for severe, active forms of a disease that has few treatment options.

EMA’s human medicines committee (CHMP) based its recommendation for approval on data from a Phase 3 clinical trial. The study investigated the safety and efficacy of Tavneos in a total of 331 patients who were treated for a year. Avacopan was used as combination treatment with cyclophosphamide, followed by combination of avacopan with azathioprine or mycophenolate, or in combination with rituximab, followed by avacopan monotherapy, and with glucocorticoids as needed.  The study showed a higher percentage of sustained remission after 52 weeks compared to the control group.

The most common side effects registered with Tavneos in clinical trials included infection of the upper airways, sore and inflamed throat and nose, headache, nausea and diarrhoea.

The opinion adopted by the CHMP is an intermediary step on Tavneos’ path to patient access. The opinion will now be sent to the European Commission for the adoption of a decision on an EU-wide marketing authorisation. Once a marketing authorisation has been granted, decisions about price and reimbursement will take place at the level of each Member State, taking into account the potential role/use of this medicine in the context of the national health system of that country.

Notes

1. The applicant for Tavneos is Vifor Fresenius Medical Care Renal Pharma France.
2. Tavneos was designated as an orphan medicinal product on 19 November 2014.
3. Following this positive CHMP opinion, the Committee for Orphan Medicinal Products (COMP) will assess whether the orphan designation should be maintained.