相关临床试验
31
4 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）
4
12.9%
Available
1
3.2%
已完成
21
67.7%
No Longer Available
2
6.5%
终止
2
6.5%
撤回
1
3.2%
暂无批准数据
- Santhera Pharmaceuticals received Swissmedic approval for AGAMREE (vamorolone) to treat Duchenne muscular dystrophy in patients four years and older, marking the company's seventh global marketing approval. - Nxera Pharma secured exclusive licensing rights for vamorolone in Japan, South Korea, Australia, and New Zealand through a $40 million upfront deal with potential milestone payments up to $165 million. - The novel dissociative steroid demonstrated superior safety compared to traditional corticosteroids, with five-year data showing reduced vertebral fractures, lower cataract incidence, and maintained normal growth rates. - AGAMREE met its primary endpoint in the pivotal VISION-DMD study with significant improvement in Time to Stand velocity versus placebo (p=0.002) at 24 weeks.
- The Duchenne Muscular Dystrophy market reached approximately $2.15 billion in 2023 and is expected to grow significantly due to increased drug uptake and anticipated gene therapy launches. - Over 75 companies are actively developing pipeline therapies for DMD, with recent FDA designations including Atossa Therapeutics' (Z)-Endoxifen receiving Rare Pediatric Disease designation. - Capricor Therapeutics announced positive Phase 3 HOPE-3 trial results for Deramiocel, while the FDA accepted their BLA for review with Priority Review designation. - Current approved treatments include EMFLAZA, VYONDYS 53, EXONDYS 51, AMONDYS 45, VILTEPSO, and gene therapy ELEVIDYS in the US, with AGAMREE launched in Germany in 2024.
- Santhera Pharmaceuticals reported five-year data from up to 110 DMD patients showing AGAMREE (vamorolone) maintained motor function comparable to standard corticosteroids while demonstrating significantly fewer vertebral fractures and normal growth patterns. - The long-term analysis revealed patients treated with AGAMREE experienced a significantly lower rate of vertebral fractures (p=0.0061) and maintained normal growth without the stunting commonly seen with standard corticosteroids (p<0.0001). - AGAMREE showed a lower incidence of cataracts compared to glucocorticoids, with no cases of glaucoma observed and no new safety signals emerging during the extended follow-up period. - The data supports AGAMREE's positioning as a dissociative anti-inflammatory drug that provides durable efficacy while reducing debilitating side effects that often lead to dose reduction or treatment discontinuation.
- Health Canada has approved AGAMREE (vamorolone) as the first treatment for Duchenne muscular dystrophy in patients aged 4 years and older, marking a historic milestone for Canadian DMD care. - The approval was granted under Priority Review and is based on the pivotal VISION-DMD study, where AGAMREE met its primary endpoint with significant improvement in Time to Stand velocity versus placebo (p=0.002). - Unlike traditional corticosteroids, AGAMREE demonstrates comparable efficacy while showing no restriction of growth and no negative effects on bone metabolism, potentially offering a better-tolerated treatment option. - The Canadian Neuromuscular Disease registry estimates more than 800 boys and young men are living with DMD in Canada, representing a significant unmet medical need now addressed by this approval.
- Santhera Pharmaceuticals secured approximately CHF 20 million in additional funding from existing investors Highbridge and R-Bridge to accelerate the global rollout of AGAMREE (vamorolone) for Duchenne muscular dystrophy treatment. - Strong demand for AGAMREE has exceeded expectations across the US, Europe, and China, with over 1,000 patients treated worldwide and US sales reaching USD 49.4 million in the first half of 2025. - The funding comprises USD 13 million from a royalty monetization with R-Bridge and CHF 10 million from Highbridge through a convertible bond extension, supporting inventory expansion and launch acceleration. - AGAMREE represents a novel dissociative anti-inflammatory drug that demonstrated efficacy in the pivotal VISION-DMD study while potentially avoiding growth restrictions and bone metabolism issues associated with traditional corticosteroids.
- KALA BIO has appointed Todd Bazemore as permanent President and CEO, transitioning from his interim role since February 2025. - The company is approaching a critical Phase 2b CHASE trial readout for KPI-012 in persistent corneal epithelial defect, a rare orphan disease with no FDA-approved treatments. - Bazemore brings over 30 years of biopharmaceutical experience and will lead KALA's potential transition from clinical-stage to commercial operations. - KPI-012 utilizes KALA's proprietary mesenchymal stem cell secretome platform and has received Orphan Drug and Fast Track designations from the FDA.
- Taiho Pharmaceutical's pizuglanstat (TAS-205) failed to demonstrate significant improvement in time to rise from floor compared to placebo in the Phase III REACH-DMD study. - The randomized, double-blind trial enrolled 82 male DMD patients aged 5 years and older across 26 Japanese sites over three years. - This setback adds to recent DMD therapeutic failures, including Sarepta/Roche's Elevidys and Pfizer's withdrawn gene therapy program. - Pizuglanstat works by inhibiting hematopoietic prostaglandin D synthase to reduce muscle necrosis and inflammatory response in DMD patients.
- Satellos Bioscience's investigational treatment for Duchenne muscular dystrophy (DMD) has demonstrated encouraging efficacy signals in adult patients, offering potential new options in a challenging therapeutic area. - The development comes amid setbacks in the DMD field, including Pfizer's withdrawal of its gene therapy fordadistrogene movaparvovec following a Phase III failure and a Phase II patient fatality. - The global DMD treatment market is projected to grow substantially from $2.3 billion in 2023 to $5.2 billion by 2033 across major markets, primarily driven by Elevidys and Santhera Pharmaceuticals' Agamree.
- The Duchenne muscular dystrophy market across seven major markets is projected to grow from $2.3 billion in 2023 to $5.2 billion by 2033, fueled by recent approvals of Elevidys and Agamree. - Exon-skipping therapies currently dominate the market with $1.0 billion in sales, expected to reach $1.8 billion by 2033, while gene therapies are forecast to contribute $821 million. - The United States maintains market leadership with 84.8% share, though significant unmet needs persist for non-ambulatory patients and those requiring treatment for late-stage complications.
- The FDA has requested Santhera Pharmaceuticals to conduct a second Phase 3 trial for Raxone (idebenone) in Duchenne muscular dystrophy treatment, potentially delaying approval until 2020. - Despite successful DELOS Phase 3 study results, the FDA wants to await findings from the SIDEROS trial before considering approval, causing Santhera's shares to drop significantly. - The setback adds to recent challenges in DMD drug development, following BioMarin's Kyndrisa withdrawal and Sarepta's ongoing regulatory discussions for eteplirsen.
