相关临床试验
582
289 进行中
药物批准
13
批准总数
监管机构
2
监管机构数
成立时间
1992
进行中（未招募）
279
47.9%
Approved For Marketing
2
0.3%
Available
1
0.2%
已完成
169
29.0%
Enrolling By Invitation
1
0.2%
No Longer Available
5
0.9%
尚未招募
9
1.6%
招募中
56
9.6%
终止
45
7.7%
Unknown
1
0.2%
撤回
14
2.4%
- The EMA's CHMP has recommended approval of Alexion's Klygefa (gefurulimab) as an add-on therapy for anti-AChR antibody-positive adults with generalised myasthenia gravis. - The positive opinion rests on the pivotal PREVAIL Phase III trial, where gefurulimab improved MG-ADL total score at week 26 versus placebo by -1.6 (95% CI: -2.4, -0.8; p<0.0001). - If approved, Klygefa would be the first and only dual-binding nanobody C5 inhibitor for this population, given once weekly by subcutaneous self-administration via autoinjector. - Klygefa is already approved in Japan and other countries, while submissions based on PREVAIL remain under review in the US, China and additional markets.
- The FDA accepted and granted Priority Review to Alexion's Biologics License Application for efzimfotase alfa in hypophosphatasia patients aged two years and older. - The PDUFA action date is anticipated in the first half of 2027, with regulatory submissions also under review in Japan and other markets. - The filing rests on the three-trial Phase III programme HICKORY, MULBERRY and CHESTNUT, which enrolled 196 patients across 22 countries. - MULBERRY met its primary radiographic endpoint with a median RGI-C difference of 1.67 versus placebo, while HICKORY missed statistical significance on the six-minute walk test.
- Xencor reported second quarter 2026 financial results, including $51.2 million in revenue and a net loss of $21.7 million, while advancing its wholly owned clinical pipeline. - Phase 1 results for XmAb819 in advanced clear cell renal cell carcinoma were accepted for a proffered paper oral presentation at ESMO Congress 2026 in Madrid. - XmAb541 monotherapy showed an approximate 14% overall response rate in ovarian cancer and 28% in germ cell tumors, supporting prioritization of its combination with XmAb808. - The U.S. FDA granted Fast Track designation to XmAb541 in July 2026 for germ cell tumors relapsed or refractory after two or more lines of platinum therapy.
- Ultomiris (ravulizumab) failed to achieve statistical significance for event-free survival at 26 weeks versus placebo in the ALXN1210-TMA-313 Phase III trial in adults and adolescents with HSCT-TMA. - In the paediatric ALXN1210-TMA-314 open-label trial, Ultomiris demonstrated clinically meaningful overall survival of 87.2% at 26 weeks and 73.4% at 52 weeks. - Alexion is advancing regulatory filings for Ultomiris in paediatric HSCT-TMA, supported by real-world evidence from the ALX-TMA-502 external control study. - HSCT-TMA is a rare, life-threatening post-transplant complication with one-year survival rates historically ranging from 17% to 58% in adults and 17% to 44% in paediatric patients.
- AstraZeneca's CARES Phase III program failed to meet its primary endpoint in the overall AL amyloidosis population, but demonstrated significant benefits in a prespecified kappa light chain subgroup. - Anselamimab showed a 62% improvement in survival and 71% reduction in cardiovascular hospitalizations in patients with kappa light chain amyloidosis compared to placebo. - The potential first-in-class anti-fibril therapy represents the first treatment to show clinically meaningful survival benefits by targeting existing amyloid fibril deposits in this rare disease affecting approximately 74,000 patients worldwide.
- Efzimfotase alfa met its primary endpoint in pediatric patients with hypophosphatasia, demonstrating statistically significant bone health improvements in the MULBERRY trial. - The investigational therapy failed to achieve statistical significance in its primary endpoint for adolescents and adults in the HICKORY trial, though it showed benefits in specific subgroups. - The drug offers potential advantages over current treatment with less frequent dosing every two weeks and maintained efficacy when patients switched from existing therapy.
- Health Canada has approved Koselugo (selumetinib) for adult patients with neurofibromatosis type 1 who have symptomatic, inoperable plexiform neurofibromas, addressing a significant unmet medical need. - The approval was based on the KOMET Phase III trial, which demonstrated a statistically significant 20% objective response rate compared to 5% with placebo. - This marks the first treatment option for Canadian adults with this rare genetic condition, as Koselugo was previously only available for pediatric patients. - The drug has been recently listed on the common drug list under Canada's National Strategy for Drugs for Rare Disease, reflecting its clinical impact.
- Arvinas has appointed Randy Teel, Ph.D., as President and CEO, succeeding John Houston who is retiring after nine years of leadership. - The leadership transition comes after Arvinas achieved its first-ever successful pivotal trial of a PROTAC degrader, marking a significant milestone for the protein degradation platform. - The company is advancing multiple clinical programs including ARV-102 for neurodegenerative disorders, ARV-393 for non-Hodgkin lymphoma, and ARV-806 for KRAS G12D mutated cancers. - Under Houston's leadership, Arvinas raised over $2 billion in funding and established itself as the industry leader in targeted protein degradation therapeutics.
- DelveInsight's 2025 pipeline analysis reveals over five companies are actively developing Wilson Disease treatments, with ALXN1840 completing Phase III enrollment as a potential first-in-class targeted copper reduction therapy. - ALXN1840 has received Orphan Drug Designation in both the United States and European Union, positioning it as a promising once-daily oral treatment specifically designed to bind and eliminate excess copper from tissues and bloodstream. - The Wilson Disease pipeline includes diverse therapeutic approaches spanning gene therapies, small molecules, and targeted treatments across various development stages from preclinical to late-stage Phase III trials. - Market growth is driven by increasing prevalence and demand for better treatments, though high drug costs and lack of reimbursement policies present significant barriers to market expansion.
- CalciMedica terminated its Phase II KOURAGE trial evaluating Auxora in acute kidney injury patients after the independent data monitoring committee identified safety concerns requiring study design reevaluation. - The company's stock dropped over 75% following the announcement, with shares falling from $5.12 to $1.25 at market close, reflecting investor concerns about the program's future. - Despite the trial halt, CalciMedica reported no treatment-related deaths and no serious adverse events requiring expedited FDA reporting, with plans to review unblinded data to guide future development. - The company remains committed to advancing Auxora in acute pancreatitis, with plans to finalize a pivotal trial design in the first half of 2026 pending FDA feedback.
