Shanghai-based Convalife Pharmaceuticals has announced that it has received orphan drug designation (ODD) status from the US FDA for its second-generation PARP inhibitor, mefuparib (CVL218). The designation is in recognition of the drug’s potential as a treatment for cholangiocarcinoma, a rare and aggressive form of cancer.
Advantages of CVL218 Over First-Generation PARP Inhibitors
Co-developed by the Shanghai Institute of Materia Medica, Chinese Academy of Sciences, CVL218 addresses several limitations associated with first-generation PARP inhibitors. These include poor water solubility, low bioavailability, limited tissue distribution, inability to penetrate the blood-brain barrier, unstable formulations, and safety risks. A Phase Ib/II clinical study suggests that CVL218 may outperform first-generation PARP inhibitors in terms of efficacy, showing effectiveness against BRCA-mutant cholangiocarcinoma, prostate cancer, pancreatic cancer, and breast cancer.
Promising Clinical Study Results
Data from an ongoing multicenter Phase Ib/II clinical study indicates that patients with advanced and multiple metastatic cholangiocarcinoma experienced a significant reduction of target lesions by more than 30% after receiving CVL218 as a monotherapy. These patients achieved a duration of up to 15 months, demonstrating the potential effectiveness of CVL218 in treating this difficult-to-treat cancer.-Fineline Info & Tech
