ChemoCentryx’s Tavneos Approved in Japan for MPA, GPA
The Japanese Ministry of Health, Labor, and Welfare (MHLW) has approved Tavneos (avacopan) for the treatment of microscopic polyangiitis (MPA) and granulomatosis with polyangiitis (GPA).
The approval of this first-in-class therapy — for the two main forms of ANCA-associated vasculitis (AAV) — was granted to Kissei Pharmaceuticals, which owns an exclusive license from Vifor Pharma to develop and commercialize the therapy in Japan.
Tavneos’ development and commercial rights in the U.S. are owned by ChemoCentryx, the therapy’s original developer, which granted Vifor Pharma the therapy’s marketing rights in nearly all other countries.
“This marks the first ever approval by a regulatory agency of a novel medication discovered and developed by ChemoCentryx,” Thomas J. Schall, PhD, president and CEO of ChemoCentryx, said in a press release.
“We would like to thank Kissei and the MHLW for their time and tremendous efforts, which made this important milestone in the mission to bring relief to patients suffering from diseases with major unmet needs possible,” Schall added.
Similar applications requesting avacopan’s approval in the U.S. and Europe also have been filed, with decisions expected by year’s end.
After an advisory committee from the U.S. Food and Drug Administration raised some issues about the clinical benefit of avacopan, ChemoCentryx filed an amended application with additional data. A decision now is expected by Oct. 7. In Europe, a decision is expected by the end of the year.
The complement system is a part of the immune system that normally helps fight infections, but that can contribute to autoimmune disorders like AAV.
Tavneos is a new oral small molecule inhibitor of the C5a protein, one of the most potent pro-inflammatory proteins of the complement system. By blocking C5a, the therapy is thought to ease the inflammation and blood vessel damage caused by excess complement activation.
Tavneos’ approval in Japan was based on the ADVOCATE Phase 3 clinical trial (NCT02994927), which aimed to determine if the therapy could be used as an alternative to conventional steroid treatment. Such treatment often is associated with side effects.
The trial enrolled 331 patients with MPA or GPA and randomly assigned them to receive Tavneos or a standard regime of the glucocorticoid prednisone for about one year. All patients also received standard immunosuppressive medications — rituximab or cyclophosphamide, followed by azathioprine.
The study met its primary efficacy goals, demonstrating that the therapy was similar to prednisone at promoting clinical remission after six months (72.3% and 70.1%), but significantly more people in the Tavneos group were still in remission after one year (65.7% vs. 54.9%).
Additional data from the trial also demonstrated that Tavneos was better at improving kidney function than steroids, especially among patients with advanced kidney disease.
Tavneos has been granted orphan drug designation for AAV treatment in the U.S. and Europe, which helps accelerate the development and approval of medicines to treat rare diseases.