The FDA has granted a rare pediatric disease designation to GPS for the treatment of pediatric patients with AML.1 "GPS has already demonstrated promise in clinical settings for AML, and we believe its potential could extend to pediatric patients,” said Angelos Stergiou, MD, ScD hc, president...
Rare Pediatric Disease Designation is granted by the FDA for serious or life-threatening diseases which primarily affect individuals less than 18 years of age and fewer than 200,000 people in the United States. If a New Drug Application for WVE-N531 is approved by the FDA, Wave would be el...
FDA grants rare pediatric disease designation for Leber congenital amaurosis gene therapyYoung, AlexOcular Surgery News
The FDA granted rare pediatric disease designation for BPM31510T for the treatment of epidermolysis bullosa.Image: Adobe Stock. “We are honored to offer hope for children who suffer from debilitating wounds and pain,”Vijay Modur, MD, PhD,chief medical officer of BPGbio...
11月19日,深信生物技术及创新RNA疗法开发的临床阶段生物制药公司,宣布其在研mRNA新药IN015获美国FDA孤儿药资格认定(Orphan Drug Designation, ODD),这是继2024年11月1日获得美国FDA儿科罕见病资格认定(Rare Pediatric Disease Designation, RPDD)后又一新进展。
2024年9月4日,尧唐生物(YolTech Therapeutics)宣布,FDA已授予其在研体内基因编辑药物YOLT-203儿科罕见疾病资格认定(Rare Pediatric Disease Designation, RPDD),用于治疗原发性高草酸尿症1型(PH1)。YOLT-203采用尧唐生物自主研发的CRISP...
The FDA has granted the novel GSK-3β inhibitor elraglusib rare pediatric disease designation for the potential treatment of patients with Ewing sarcoma.1 “Receiving rare pediatric disease designation from the FDA underscores the urgent need for new treatment options for patients with Ewing sarcoma...
近日,天津尚德药缘科技股份有限公司开发的ACT001,获得美国药监局FDA批准的儿童罕见病资格,即Rare Pediatric Disease Designation,简称RPDD。RPDD是申请和获得优先评审券PRV(Priority Review Voucher)的资格证。PRV是美国FDA为了鼓励公司针对儿童罕见疾病开发新药,授予新药上市快速评审的优先券,该券可以在二级市场上转让,一次...
近日,深信生物宣布其在研mRNA新药IN022继2024年7月5日获得美国FDA儿科罕见病资格认定(Rare Pediatric Disease Designation,RPDD)后又获美国FDA孤儿药资格认定(Orphan Drug Designation,ODD)。 IN022用于治疗同型胱氨酸尿症(Homocystinuria,HCU),一种常染色体隐性遗传性代谢疾病。同时获得RPDD和ODD将极大地促进...
US FDA grants Rare Pediatric Disease Designation to Axovant’s AAV9-based gene therapy, AXO-AAV-GM1 for GM1 gangliosidosis