The European Medicines Agency (EMA) has accepted for review ChemoCentryx’s application to approve Avacopan (CCX168) as a treatment for anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis.
The company, with its partner Vifor Fresenius Medical Care Renal Pharma, applied for a Conditional Marketing Authorization (CMA) of the medicine. A CMA may be granted for therapies that have not yet completed clinical testing if there is an unmet need for treating a seriously debilitating or life-threatening disease.
If ChemoCentryx’s CMA application is approved, the company still has to complete clinical trials normally required for approval.
“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,” Stefan Schulze, Vifor Pharma president of the executive committee and COO, said in a press release.
Avacopan, an oral drug, blocks an immune receptor called C5aR. This has very specific effects on the immune system — fighting disease processes while leaving normal immune functions unaffected.
“Avacopan is a much more selective, targeted means of treating ANCA than the current standard of care,” said Schulze.
Current treatment options for ANCA vasculitis include non-specific immunosuppressants and high-dose corticosteroids.
Avacopan is being studied in a Phase 3 clinical trial, called ADVOCATE (NCT02994927). The global study is still recruiting participants, but data from the earlier Phase 2 CLEAR trial (NCT01363388) showed that treated patients could reduce their use of corticosteroids significantly.
Long-term treatment with steroids and immunosuppressants is linked to severe side effects, including potentially life-threatening infections.
“A new treatment paradigm for ANCA vasculitis has been a goal for clinicians and patients for decades,” said David Jayne, MD, a professor and vasculitis director, Addenbrooke’s Hospital at the 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,” he said.
Researchers now hope that Avacopan may change that practice, as the Phase 2 study showed that C5aR appears to be specifically driving the inflammation in ANCA vasculitis.
“The value and promise of Avacopan for patients with ANCA vasculitis has today been reinforced by the EMA validation of the Avacopan CMA application,” said Thomas J. Schall, PhD, president and CEO 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,” Schall said.
Avacopan also has been granted the EMA’s Priority Medicines (PRIME) status, under which the regulatory review is accelerated.