AstraZeneca's Voydeya Approved for PNH, Merck Advances KRAS Inhibitor
核心洞察
The FDA has approved AstraZeneca's Voydeya (danicopan) as an add-on therapy for extravascular hemolysis in adults with paroxysmal nocturnal hemoglobinuria (搜索) (PNH (搜索)).
Merck (搜索)'s oral KRAS G12C (搜索) inhibitor candidate, MK-1084, has entered phase III development for first-line treatment of metastatic non-small cell lung cancer (搜索) (NSCLC (搜索)).
Jazz Pharmaceuticals has completed filing a regulatory application for zanidatamab, targeting HER2 (搜索)-positive metastatic biliary tract cancer (搜索) (BTC (搜索)).
AstraZeneca's Voydeya (danicopan) has received FDA approval as an add-on therapy for extravascular hemolysis in adult patients with paroxysmal nocturnal hemoglobinuria (搜索) (PNH (搜索)) who are already being treated with C5 inhibitors like Ultomiris or Soliris. This approval addresses the 10-20% of PNH patients who continue to experience hemolysis, or the destruction of red blood cells, despite C5 inhibitor treatment. The approval was based on the pivotal ALPHA phase III study.
Voydeya's Clinical Impact
Voydeya's approval marks a significant advancement for PNH (搜索) patients experiencing persistent hemolysis. The drug, already approved in Japan, is under regulatory review in the European Union and other countries. The ALPHA phase III study demonstrated Voydeya's efficacy in reducing hemolysis in patients inadequately controlled by existing C5 inhibitors.
Merck's KRAS Inhibitor Enters Phase III
Merck (搜索) has initiated a phase III study of its oral KRAS G12C (搜索) inhibitor candidate, MK-1084, in combination with Keytruda, for the first-line treatment of metastatic non-small cell lung cancer (搜索) (NSCLC (搜索)) in patients with KRAS G12C mutations. The global study aims to enroll approximately 600 patients, with progression-free survival and overall survival as primary endpoints. MK-1084 is also being evaluated in a phase I study as a monotherapy and in combination with other therapies for KRAS G12C mutant advanced solid tumors.
Advancing Ovarian Cancer Treatment
Merck (搜索) also announced the dosing of the first patient in the phase II/III REJOICE-Ovarian01 study of its CDH6 (搜索)-directed DXd ADC, raludotatug deruxtecan (R-DXd), in patients with platinum-resistant ovarian cancer (搜索). This study is based on promising phase I data showing R-DXd's activity in advanced ovarian cancer. Merck is co-developing R-DXd with Daiichi Sankyo, along with two other ADCs, under a global partnership deal.
Jazz Pharmaceuticals Seeks Approval for Zanidatamab
Jazz Pharmaceuticals has completed the submission of a rolling BLA seeking approval of zanidatamab, a HER2 (搜索)-targeted bispecific antibody, for the treatment of previously treated metastatic HER2-positive biliary tract cancer (搜索) (BTC (搜索)). The BLA is supported by data from the phase IIb HERIZON-BTC-01 study, which met its primary endpoint in patients receiving zanidatamab for previously treated HER2-positive BTC. Zanidatamab is also being evaluated in phase III studies for first-line BTC, first-line gastroesophageal adenocarcinoma (GEA), and previously treated breast cancer.