相关临床试验
150
6 进行中
药物批准
33
批准总数
监管机构
2
监管机构数
成立时间
N/A
进行中（未招募）
5
3.3%
Available
1
0.7%
已完成
106
70.7%
尚未招募
1
0.7%
招募中
16
10.7%
终止
16
10.7%
Unknown
1
0.7%
撤回
4
2.7%
- Once-daily deucrictibant extended-release tablets reduced hereditary angioedema attack rates by 83% versus placebo in the Phase 3 CHAPTER-3 trial, with a p-value below 0.0001. - The global, double-blind study randomized 85 adolescents and adults across 21 countries to 40 mg deucrictibant XR or placebo for 24 weeks, with all secondary endpoints met. - Pharvaris plans to submit a prophylaxis New Drug Application to the FDA in the first half of 2027, following an accepted NDA for the immediate-release formulation. - Deucrictibant blocks the bradykinin B2 receptor, potentially making it the first oral HAE therapy spanning both on-demand treatment and long-term prophylaxis.
- Over 180 companies are advancing 250+ AAV-based gene therapy candidates globally, spanning preclinical through marketed stages, according to DelveInsight's 2026 pipeline assessment. - The FDA granted approval to Otarmeni, the first dual-AAV gene therapy for OTOF-related genetic hearing loss, and accepted Ultragenyx's BLA for UX111 in Sanfilippo syndrome Type A with a September 2026 PDUFA date. - Taysha Gene Therapies reported FDA alignment on a BLA pathway for TSHA-102 in Rett syndrome, with pivotal REVEAL trial enrollment progressing and manufacturing validation underway. - Sensorion's SENS-501 demonstrated durable efficacy and favorable safety at six months in a Phase I/II trial for otoferlin-related congenital hearing loss, while GJB2-GT advances toward regulatory filing.
- CSL broke ground on a $1.5 billion expansion of its Kankakee, Illinois manufacturing facility to enhance production of plasma-derived therapies for rare diseases. - The expansion will incorporate CSL's patented Horizon 2 manufacturing process, which significantly increases immunoglobulin production from the same amount of plasma. - The facility expansion is expected to create at least 300 new pharmaceutical jobs and will be operational by 2031. - This investment builds on CSL's $3 billion commitment to U.S. operations since 2018, bringing their total American workforce to nearly 19,000 employees.
- Hemophilia gene therapies are underperforming commercially despite initial blockbuster expectations, with Pfizer withdrawing Beqvez from the market and BioMarin seeking to divest Roctavian. - High treatment costs, including Hemgenix's $3.5 million price tag, create reimbursement challenges as payers question covering lifetime therapies for patients who may switch plans within two years. - Structural disincentives discourage adoption, with treatment centers potentially losing $500,000-$750,000 annually in revenue when patients switch to gene therapy. - Despite current market struggles, the hemophilia treatment landscape remains promising with new therapies in development, including Novo Nordisk's Mim8 and innovative approaches like Be Biopharma's re-dosable cell therapy BE-101.
- CSL plans to invest approximately $1.5 billion over the next five years to expand U.S. manufacturing capabilities for plasma-derived therapies, strengthening the domestic medicine supply chain. - The investment will generate hundreds of high-quality American jobs and reflects CSL's commitment to meet growing clinical demand for immunoglobulin therapies for rare and serious diseases. - This expansion builds on CSL's previous $3 billion U.S. investment since 2018, which created over 6,500 new jobs across 44 states and brought total U.S. workforce to nearly 19,000 employees. - The planned investments are subject to CSL Board of Directors approval and leverage the U.S. position as the world's leading source for plasma used in life-saving therapies.
- CSL Behring signed a Letter of Intent with Canada's pan-Canadian Pharmaceutical Alliance for public reimbursement of HEMGENIX, the first gene therapy authorized by Health Canada for hemophilia B treatment. - The Phase 3 HOPE-B trial demonstrated that 94% of patients discontinued routine prophylaxis and remained prophylaxis-free at 4 years post-treatment with sustained Factor IX activity levels of 37.4%. - HEMGENIX uses an AAV5 viral vector to deliver genetic instructions enabling continuous Factor IX production, reducing annualized bleeding rates from 4.16 to 0.40 at year four. - The agreement allows provinces and territories outside Quebec to proceed with formulary listing, marking a significant milestone in making this transformative one-time gene therapy accessible to Canadian patients.
- The FDA approved three novel hereditary angioedema (HAE) therapies in 2025 after four years without new approvals, increasing total marketed drugs by 38% to 11 products. - CSL Behring's garadacimab became the first FDA-approved drug targeting coagulation factor XII, offering a new biological mechanism for HAE prevention. - KalVista's sebetralstat (Ekterly) represents the first oral on-demand treatment for HAE, providing patients with rapid relief during acute episodes. - Ionis Pharma's donidalorsen sodium introduced the first antisense oligonucleotide for HAE with extended dosing intervals of every four to eight weeks.
- DelveInsight's 2025 pipeline report reveals over 20 companies are actively developing more than 30 therapeutic candidates for hereditary angioedema treatment across various clinical stages. - Recent clinical developments include CSL Behring's Phase 3b study of garadacimab (CSL312) announced in August 2025 and KalVista's pediatric trial of KVD900 for patients aged 2-11 years. - Leading pipeline therapies span multiple approaches including oral plasma kallikrein inhibitors, gene therapies, and monoclonal antibodies, with products in late-stage development showing promise for addressing unmet medical needs.
- Australian biotech giant CSL announced plans to separate its vaccine business Seqirus into a standalone company by June 2025, allowing the unit greater autonomy in the dynamic vaccines market. - The restructuring includes workforce cuts of up to 15% and is expected to generate $500-550 million in annual cost savings over three years, despite a one-time charge of $700-770 million. - CSL will launch a $750 million share buyback program as part of the strategic pivot, reflecting strong cash flow performance with operations generating $3.56 billion in fiscal 2025. - Seqirus contributed $2.2 billion to CSL's total revenue of $15.6 billion, with the vaccine unit facing competitive pressures in the U.S. influenza market despite overall 2% revenue growth.
- CSL Behring's $900 million plasma fractionation facility in Broadmeadows, Victoria, has been named 2025 Facility of the Year in the Pharma 4.0 category by the International Society for Pharmaceutical Engineering. - The facility represents the world's largest plasma fractionation facility of its kind, processing over 10 million liters of plasma annually and increasing CSL Behring's local capacity ninefold. - The award recognizes the facility's full-scale integration of robotics, automation technologies, and digital twin technology that enhances safety, reduces waste, and improves operational efficiency. - The facility produces plasma-derived therapies for treating immunodeficiencies, neurological disorders, shock, burns, and supports transplant, surgical, and cancer patients globally.
