此次FDA的决定是基于一项双盲、安慰剂对照III期研究DELOS的积极结果。该研究共招募了64名来自欧洲和美国未接受皮质类固醇激素治疗的10-18岁DMD患者，研究中，患者随机分配至idebenone（900mg/天）或安慰剂治疗52周，主要终点为从基线至治疗的52周，呼气流量峰值（PEF，又名最大呼气流量）占预测值百分比的变化（PEF%）。研究结果显示，在研究的第52周，安慰剂组PEF%相对基线显著下降（-9.01%p;95% CI:-13.2,-4.8;p＜0.001），idebenone治疗组PEF%相对基线下降不显著（-3.05%p;95% CI:-7.1,0.97;p=0.134）；2组PEF%差值为5.96%p（95% CI:0.16,11.8;p=0.044），表明idebenone使患者PEF%下降幅度显著改善66%。此外，在研究的26周（p=0.007）和39周（p=0.034）及横跨所有评估时间点（p=0.018），相对安慰剂组，idebenone治疗组均表现出统计学意义的显著疗效差异。
Santhera CEO Thomas Meier十分看好idebenone，将全力打造该药物，使其成为公司长线收入的增长点。"我们将在未来几周内召开新药申请的文件准备会议，争取尽快向FDA提交。"
Santhera Pharmaceuticals Wins Fast Track Designation From FDA For Raxone/Catena Treatment
FDA's decision to award Santhera a fast track designation for Raxone/Catena treatment has come on the back of positive results from a late stage trial, DELOS
Swiss specialty pharmaceutical company Santhera Pharmaceuticals (OTCMKTS:SPHDF) has finally achieved a victory it had long been striving for. The company's Raxone/Catena regimen has won a fast track designation from the US Food and Drug Administration (FDA) for treating patients suffering from Duchenne Muscular Dystrophy (DMD).
Such a designation is bound to expedite the regulatory process for the drug, opening doors for the final approval to come soon. Fast track designation granted by FDA is meant for aiding the development and regulatory review for those drugs aimed at treating serious diseases; for which no other or very few approved treatments are available in the market.
The designation also provides aid by enabling pharmaceutical companies to communicate with the FDA more frequently, which significantly helps in the development and final approval process of the drugs. With this fast track status, there are also high chances that Santhera might win Accelerated Approval and Priority Review from the FDA, which will further speed up the regulatory process.
Raxone/Catena (chemical name idebenone), is a "synthetic short-chain benzoquinone," which works by enhancing mitochondrial electron transport to have a significant impact on increasing the energy levels of cells found in muscles. Adding to Santhera's definite success in the future, Idebenone also enjoys orphan drug designation for DMD, both in the US and in Europe. It has also been given patent protection in the US till 2027, and in Europe till 2026.
The recent FDA decision to grant fast track status to Santhera's Raxone/Catena brings good news for the large number of patients currently living with DMD across the world. DMD, considered to be a genetic, degenerative disease and the most common and serious form of muscle degeneration that leads to rapid weakness of muscles, is currently experienced by one in every 3,500 male babies around the world.
The disease, which results into high early-mortality due to high cardio-respiratory failure risk it entails, has the only available medical treatment in form of glucocorticoid steroids. Steroids, though reduce the muscles weakness, but also come with serious side effects, which significantly limits their use in DMD patients. Almost 42% of patients currently living with DMD aged 10 years or more have never taken steroids for their DMD treatment or have stopped their use due to various side effects, as per data gathered in a recent study.
The FDA's decision is due to positive results from a late stage trial, named DELOS, which was a double blind and placebo-controlled study with a total of 64 enrollments of DMD patients from both Europe and US. The results established significant efficacy for Santhera's regimen, which proved to fully meet the primary endpoint of the study by resulting into a significant 66% of reduction in loss of Peak Expiratory Flow percent from baseline to week 52, when compared to the treatment group given a placebo. In addition, Raxone/Catena also led to a "statistically significant treatment effect" when observed at week 26, 39 and across various durations that were assessed.
Thomas Meier, the CEO at Santhera, seemed very positive of the long-term growth potential that the Raxone/Catena DMD treatment holds for the company. He stated: "We are very pleased that the FDA has granted Fast Track designation for Raxone/Catena, which further underscores the unmet medical need for effective treatments for patients with DMD."
Meanwhile, he added: "On the basis of the positive data from our Phase III trial with Raxone/Catena in DMD, we have started to prepare a New Drug Application and plan to meet with the FDA in the coming weeks to discuss our NDA dossier in a pre-NDA meeting," as per a press release by the Swiss drug maker today.
Santhera stock has already gained 15.34%, trading at chf 107.9 in Six Swiss Stock Exchange (SWX) as of 12:10PM GMT+2.