2. FDA:2017 New Drug Therapy Approvals 3. The Year in New Drugs:FDA approvals hit a 20-year high in 2017,with cancer and rare-disease drugs dominating the list of new medicines
2. FDA:2017 New Drug Therapy Approvals 3. The Year in New Drugs:FDA approvals hit a 20-year high in 2017,with cancer and rare-disease drugs dominating the list of new medicines
总体而言,美国孤儿药政策促进了药物的研发,我国在制定相关政策时可借鉴美国的研发管理经验,要从罕见病患者角度出发,确保药物研发满足真正的临床需求。 罕见病(rare disease)是发病率极低的一类疾病统称。目前还没有统一的界定罕见病的标准,一些国家和地区以发病率或患病人数作为界定标准[1]。20世纪70年代,大约只有10种...
FDA approves rare liver disease drugErin Bastick
“This designation from FDA underscores that significant unmet needs remain in DMD, and it also supports Wave’s innovative and purposeful approach to drug development in the rare disease space,” said Anne-Marie Li-Kwai-Cheung, MChem, MTOPRA, RAPS, Chief Development Officer at Wave Life Science...
上周FDA共颁发19项孤儿药资格,Kezar Life Sciences的“first-in-class”选择性免疫蛋白酶体抑制剂KZR-616获得2项孤儿药资格,诺华(Novartis)治疗亨廷顿病(Huntington's disease,HD)的小分子RNA剪接调节剂、Antisense Therapeutics在研杜氏肌营养不良(Duchenne muscular dystrophy,DMD )RNA疗法纷纷入围,此外还有多款基因疗法...
FDA Grants Rare Pediatric Designation to Elraglusib for Ewing Sarcoma Ryan Scott November 16th 2024 The FDA granted elraglusib a rare pediatric disease designation for Ewing sarcoma based on findings from the 1/2 Actuate-1902 trial. BLA Filed for Dato-DXd for EGFR-Mutant Lung Cancer Chris Ry...
For the upcoming gene therapy candidates getting ready for BLA submission in the US, the first on the list is Krystal Biotech’s beremagene geperpavec, a topical COL7A1-expressing gene therapy for the treatment of dystrophic epidermolysis bullosa (DEB), a rare and severe disease that affects ...
For the upcoming gene therapy candidates getting ready for BLA submission in the US, the first on the list is Krystal Biotech’s beremagene geperpavec, a topicalCOL7A1-expressing gene therapy for the treatment of dystrop...
The FDA has licensed Baxter Healthcare's (Deerfield, IL, www.baxter.com) Ceprotin, the first biologic treatment for patients with a rare genetic defect that can cause a potentially life-threatening clotting disorder. The FDA has licensed Baxter Healthcare’s (Deerfield, IL, www.baxter.com) ...