英国MHRA暂停Amgen罕见病药Avacopan Vifor对新患者使用并拟撤销授权

The U.K. drug regulator, the Medicines and Healthcare Products Regulatory Agency, has suspended the use of Amgen’s ( AMGN ) rare-disorder therapy Avacopan Vifor (formerly known as Tavneos), citing concerns over data supporting its approval. Amgen ( AMGN ), the U.S. rights holder for Avacopan Vifor, has partnered with CSL Vifor of CSL Limited ( CSLLY ) ( CMXHF ) to market the drug outside its home country, including Britain. The decision came after a review determined that data from a pivotal clinical study supporting Avacopan Vifor’s marketing authorization could no longer be relied upon, the MHRA said in a statement on Tuesday. The suspension took effect on Sept. 1, affecting how the drug is used, supplied, and sold; Avacopan Vifor was approved in the U.K. in 2022 for two rare inflammatory diseases that affect blood vessels. Following a six-month managed withdrawal period that allows those who are already using the drug to switch to alternative treatments, the MHRA plans to impose a complete ban on Tavneos on March 1, 2027. "We disagree with the MHRA's decision to withdraw Tavneos from the UK market and are deeply concerned about the impact this decision may have on rare-disease patients," Reuters reported, quoting a statement from an Amgen ( ​( AMGN ) spokesperson. The company added that ​the MHRA ⁠ did not adequately review data from clinical trials and over 20 published real-world studies supporting Tavneos. In August, the European Commission revoked the drug ’ s EU marketing authorization due to data integrity issues. In April, the U.S. FDA proposed withdrawing Tavneos, citing safety concerns and alleged manipulation of data supporting its marketing application, a decision that Amgen ( AMGN ) has challenged.