EMA accepts marketing authorisation application for avacopan

The European Medicines Agency (EMA) has accepted the conditional marketing authorisation application for avacopan in the treatment of patients with anti-neutrophil cytoplasmic antibody associated vasculitis (ANCA-associated vasculitis or ANCA vasculitis).

This news represents a significant milestone in the Kidney Health Alliance of Vifor Fresenius Medical Care Renal Pharma (VFMCRP) and ChemoCentryx.

Avacopan is an orally-administered small molecule that is a highly selective inhibitor of the terminal effector complement C5a receptor (C5aR). It is currently in late-stage clinical development for the treatment of orphan and rare renal diseases, including ANCA vasculitis.

“The EMA's validation of the conditional marketing authorisation application for avacopan represents another critical step in Europe toward realising our vision of becoming global leader in nephrology therapies. Avacopan is a much more selective, targeted means of treating ANCA than the current standard of care,” said Stefan Schulze, Vifor Pharma president of the Executive Committee and COO. “We believe this treatment has real potential for transforming the way that ANCA vasculitis is treated and that it will be of tremendous value to patients who have to live with this rare renal inflammatory disease.”

“A new treatment paradigm for ANCA vasculitis has been a goal for clinicians and patients for decades,” said Professor David Jayne, Vasculitis director, Addenbrooke's Hospital, University of Cambridge, UK. “For half a century, we have used high-dose glucocorticoids as part of the treatment of ANCA vasculitis, despite their well-documented toxicities that contribute to the long-term morbidity and incapacity associated with vasculitis. In the Phase II CLEAR study, it was demonstrated that inhibition of C5aR may make the chronic use of these toxic steroids obsolete, because the target of avacopan, the C5aR, is an important and specific driver of the destructive inflammation ANCA vasculitis. The current data are very promising and we look forward to the results from the ADVOCATE Phase III trial.”

“The value and promise of avacopan for patients with ANCA vasculitis has today been reinforced by the EMA's validation of the avacopan CMA application,” said Thomas J. Schall, PhD, president and chief executive officer of ChemoCentryx. “We at ChemoCentryx, together with our outstanding partners at Vifor Pharma, are unswerving in our devotion to creating new medicines for patients suffering from devastating kidney diseases. To ANCA patients, we say this: your cause is our cause; we are determined to succeed. Achieving this pivotal regulatory milestone is a big step toward our ultimate success.”

The EMA's Committee for Medicinal Products for Human Use (CHMP) will commence its assessment of the CMA application.

Under the terms of its kidney health alliance with VFMCRP, ChemoCentryx will receive a milestone payment triggered by this validation of the avacopan CMA application by the EMA.