相关临床试验
7
2 进行中
药物批准
0
批准总数
监管机构
0
监管机构数
成立时间
2019
进行中（未招募）
1
14.3%
已完成
1
14.3%
尚未招募
1
14.3%
招募中
4
57.1%
暂无批准数据
- Vaderis Therapeutics' VAD044, an allosteric AKT-inhibitor, has been granted Fast Track designation by the FDA for treating Hereditary Hemorrhagic Telangiectasia (HHT). - The FDA's Fast Track program aims to accelerate the development and review of drugs addressing serious conditions with unmet medical needs, potentially expediting VAD044's availability. - VAD044 is an oral, once-daily therapy and the first novel treatment specifically designed for HHT, a genetic bleeding disorder lacking approved treatments. - HHT is a rare, inherited bleeding disorder that causes significant disease burden, reduced life expectancy, and impaired quality of life, highlighting the need for effective therapies.
- Vaderis Therapeutics' VAD044 demonstrated favorable safety and tolerability in a Phase 2 proof-of-concept trial for Hereditary Hemorrhagic Telangiectasia (HHT). - The trial showed clinically meaningful improvements in epistaxis frequency, duration, and epistaxis-free days in HHT patients treated with VAD044. - Interim data from an open-label extension study indicate continued improvements in bleeding parameters with sustained VAD044 treatment over six months. - Vaderis is engaging with health authorities to advance VAD044 into pivotal phase development for HHT, a disease with no approved treatments.
