相关临床试验
57
26 进行中
药物批准
2
批准总数
监管机构
1
监管机构数
成立时间
1994
进行中（未招募）
26
45.6%
Approved For Marketing
1
1.8%
已完成
19
33.3%
招募中
5
8.8%
终止
5
8.8%
撤回
1
1.8%
- The EMA's CHMP issued a negative opinion on Omeros' narsoplimab (Yartemlea) for hematopoietic stem cell transplant-associated thrombotic microangiopathy, citing insufficient evidence of effectiveness. - Regulators flagged that the pivotal study lacked a placebo or active comparator arm, making it impossible to attribute observed benefits specifically to narsoplimab. - Additional concerns included mid-study design changes, unclear dose selection rationale, and insufficient pediatric dosing data. - Omeros has 15 days to request a re-examination; the drug remains FDA-approved in the U.S., where competitive pressure may ease following AstraZeneca's Ultomiris setback.
- Omeros Corporation announced successful completion of a primate study for OncotoX-AML, showing up to 99% reduction in myeloid progenitor cells that give rise to acute myeloid leukemia. - The engineered biologic demonstrated superior efficacy compared to current standard-of-care treatments, extending survival to over 100 days in xenograft models versus 8 days with venetoclax-azacitidine combination. - OncotoX-AML's unique mechanism works independently of genetic mutations found in 90% of AML patients and was well-tolerated without significant safety signals. - The company plans to initiate IND-enabling studies with a target first-in-human trial for late 2027.
- A new catalyst monitor report identifies 13 significant regulatory and clinical trial events expected in Q1 2026, based on key opinion leader interviews and predictive intelligence. - Major regulatory approval decisions are anticipated for GSK's depemokimab in asthma and sinusitis, Regenxbio's gene therapy for Hunter syndrome, and Omeros' narsoplimab for thrombotic microangiopathy. - Ongoing late-stage clinical trials from Xenon Pharmaceuticals, Cerevel, and Neumora are expected to deliver critical data across neurological and psychiatric conditions. - The pipeline momentum spans diverse therapeutic areas including respiratory diseases, rare genetic disorders, neurological conditions, and major depressive disorder.
- The FDA approved YARTEMLEA (narsoplimab-wuug) as the first and only therapy for hematopoietic stem cell transplant-associated thrombotic microangiopathy (TA-TMA) in adults and children aged two and older. - This milestone transforms Omeros Corporation from a development-stage to a commercial-stage company, with U.S. launch planned for January 2026 and an EMA decision expected mid-2026. - YARTEMLEA positions itself as a first-mover treatment for a life-threatening, previously untreated condition, potentially reshaping complement-mediated disease therapy. - The approval removes regulatory overhang and shifts focus to execution risks, including financing the commercial launch and building infrastructure for the rare disease market.
- The FDA has approved YARTEMLEA (narsoplimab-wuug) as the first and only therapy for hematopoietic stem cell transplant-associated thrombotic microangiopathy (TA-TMA), a severe complication affecting up to 56% of transplant recipients. - Clinical trials demonstrated a 61% complete response rate in the pivotal study and 73-74% 100-day survival rates, representing a three- to fourfold reduction in mortality risk compared to external controls. - The monoclonal antibody selectively inhibits MASP-2, the effector enzyme of the lectin pathway of complement, while preserving other immune functions essential for host defense. - YARTEMLEA is approved for adults and children ages two years and older, with a U.S. commercial launch planned for January 2026 and European regulatory review ongoing.
- The FDA approved narsoplimab (Yartemlea) as the first and only therapy for transplant-associated thrombotic microangiopathy (TA-TMA), marking a breakthrough for patients with this devastating complication. - Clinical trials demonstrated a 61% complete response rate in the primary study and 73-74% 100-day survival rates, representing a 3-4 fold reduction in mortality risk compared to external controls. - The approval covers both adult and pediatric patients aged 2 years and older, with the drug showing consistent efficacy across age groups in real-world expanded access programs. - Narsoplimab targets MASP-2 in the complement lectin pathway while preserving the classical complement pathway critical for immune response to infections.
- Omeros Corporation secured a $240 million upfront payment from Novo Nordisk for exclusive global rights to develop and commercialize altenobart, with an additional $100 million in near-term milestones achievable. - The company's MASP-2 antibody narsoplimab (to be marketed as Yartemlia) remains under FDA review for transplant-associated thrombotic microangiopathy with a December 26, 2025 PDUFA date. - Recent publications in Blood Advances and American Journal of Hematology demonstrate significantly improved survival outcomes for TATMA patients treated with narsoplimab compared to standard supportive care. - Omeros has established launch-ready commercial infrastructure including national diagnostic codes and expects to receive Medicare's new technology add-on payment to support hospital reimbursement.
- Novo Nordisk announced an asset purchase and license agreement to acquire exclusive global rights to zaltenibart from Omeros Corporation for up to $2.1 billion, including $340 million in upfront and near-term milestone payments. - Zaltenibart is an investigational humanized monoclonal antibody targeting MASP-3, showing positive trial data in paroxysmal nocturnal hemoglobinuria and potential applications across rare blood and kidney disorders. - Following the transaction closure expected in Q4 2025, Novo Nordisk plans to initiate a global phase 3 program for zaltenibart in PNH and explore development for other rare blood and kidney conditions. - The deal represents Novo Nordisk's strategic expansion into rare disease therapeutics, building on their previous commitment to invest $1.2 billion in new production facilities in Denmark.
- DelveInsight's 2025 pipeline analysis reveals over 20 companies developing 25+ therapies for paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder characterized by red blood cell destruction. - Recent regulatory approvals include EMA and FDA approval of PIASKY (crovalimab) from Hoffmann-La Roche for adults and adolescents with PNH, marking significant progress in treatment options. - Multiple Phase III trials are underway, including Novartis's positive APPULSE-PNH results for iptacopan and Regeneron's combination therapy study with pozelimab and cemdisiran. - Emerging therapies span various mechanisms of action including complement inhibitors, monoclonal antibodies, and siRNA therapeutics, with oral and parenteral administration routes being explored.
- Omeros Corporation completed a $22 million registered direct offering with Polar Asset Management Partners, issuing 5.37 million shares at $4.10 per share, representing a 14% premium to the July 24 closing price. - The biotech company is using the funds to support R&D activities, including its pivotal narsoplimab program currently under FDA review for hematopoietic stem cell transplant-associated thrombotic microangiopathy. - The offering results in 8.7% dilution to existing shareholders as Omeros navigates a challenging financial position with $415 million in debt and negative EBITDA of $164 million. - FDA has extended the PDUFA date for narsoplimab's biologics license application to December 26, 2025, while the European Medicines Agency validated the Marketing Authorization Application with a decision expected mid-2026.
