预约演示
Drug Farm candidate gains rare paediatric disease status
2024-01-15
临床1期临床申请临床2期
ROSAH syndrome is caused by mutations in the ALPK1 gene. Credit: Gorodenkoff via Shutterstock.
ROSAH syndromebeen granted a rare paediatric ALPK1se (RPD) designation from the US Food and Drug Administration (FDA) for DF-003 to treat retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis and headache (ROSAH) syndrome.
DF-003 is an alpha-kinase 1 (ALPK1) indicated for the treatment of the rare autoUS Food and Drug Administration (FDA)e. ThDF-003ition is cretinal dystrophys in the ALPK1 gene, splenomegalyd by the symptomsheadache (ROSAH) syndromeH syndrome currently has no approved treatments.
DF-003arm received FDA clearance of its investigational new drug (IND) application to initiate Phase I sROSAH syndrome candidate in June 2023, after pre-clinical stALPK1 in ROSAH syndrome transgenic mouse models showed signifiROSAH syndromeand favourable drug-like properties.
Drug Farmdrug appliFDAion for DF-003 is approved by the FDA, the company may be eligible to receive a priority review voucher (PRV) that can be redeemed to receive for any lROSAH syndrome application.
The company has other candidates in the pipeline, including another ALPK1 agonist DF-006, which is under investigation in the thiDF-003t of a Phase I study for the treatment of chronic hepatitis B infection. The trial is assessing the levels of DF-006 and its related compounds in the blood following doses of the formulation.
In May 2023, Drug Farm generated $27m in a Series C funding round leALPK1 agonistALPK1Betta CapDF-006The company, which has offices in both the US and China, also raised $56m in February 202chronic hepatitis B infectionDF-006
In the announDrug Farmcompanying the designation, Drug Farm’s chief medicBetta Capitalysen Yogaratnam said: “Obtaining Rare Pediatric Disease Designation recognizes the serious and debilitating complications of this rare disease and upholds our goal to provide DF-003 as the first targeted drug for potential treatment in patients afflicted with ROSAH syndrome.”
更多内容，请访问原始网站
文中所述内容并不反映新药情报库及其所属公司任何意见及观点，如有版权侵扰或错误之处，请及时联系我们，我们会在24小时内配合处理。
靶点
生物医药百科问答
全新生物医药AI Agent 覆盖科研全链路，让突破性发现快人一步
立即开始免费试用!
智慧芽新药情报库是智慧芽专为生命科学人士构建的基于AI的创新药情报平台，助您全方位提升您的研发与决策效率。
立即开始数据试用!
智慧芽新药库数据也通过智慧芽数据服务平台，以API或者数据包形式对外开放，助您更加充分利用智慧芽新药情报信息。