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
N/A
进行中（未招募）
2
28.6%
Available
1
14.3%
已完成
2
28.6%
招募中
2
28.6%
暂无批准数据
- The European Medicines Agency's Committee for Medicinal Products for Human Use has issued a positive opinion recommending approval of Aqneursa (levacetylleucine) for treating neurological manifestations of Niemann-Pick type C disease in adults and children. - The recommendation is based on Phase III trial data showing statistically significant improvement in neurological symptoms within 12 weeks, with patients demonstrating better SARA scores compared to placebo. - Aqneursa represents a potential new frontline treatment option for NPC patients in Europe, following its FDA approval in September 2024. - The drug targets underlying neurological dysfunction processes and can be used in combination with miglustat or as monotherapy when miglustat is not tolerated.
- IntraBio successfully completed recruitment for its Phase III pivotal trial of IB1001 (N-Acetyl-L-Leucine) for Niemann-Pick disease type C, enrolling 130% of target patients across 13 international sites. - The company over-enrolled by 125% due to high community interest and unmet medical need, substantially increasing the statistical power of the randomized, placebo-controlled, double-blind crossover study. - Data readout is anticipated before the end of Q2 2023, with the trial designed to support accelerated approval pathways with both FDA and EMA for chronic NPC symptom treatment. - Upon approval, IntraBio would be eligible for an FDA priority review voucher, which have sold for over $100 million each in the past two years.
