FDA Works to Get New Drugs to Patients Faster with Multiple Approvals This Week


2020-12-03 01:33:19 BioSpace


JHVEPhoto/Shutterstock Availability of safe drugs to treat serious, dangerous diseases is a top priority of all in healthcare. The FDA developed these four distinct approaches to make drugs for rare, pediatric and otherwise unsuccessfully treated diseases are available as rapidly as possible: Priority Review, Breakthrough Therapy, Accelerated Approval and Fast Track. Here are three drugs that received one of these distinctions this week.  Zymeworks’ Zanidatamab  Zanidatamab received FDA Breakthrough Therapy designation for patients with previously-treated HER2 gene-amplified biliary tract cancer (BTC). Breakthrough Therapy designation is granted to new medicines for serious conditions when clinical evidence shows a substantial improvement over currently available therapies on a specific endpoint. This designation was granted based on an ongoing Phase I study of the drug. BTC is a rare and aggressive cancer, affecting the bile tract. Comprising approximately 3% of all adult cancers, prognosis for those diagnosed is usually poor.  Zanidatamab has the potential to be the first HER2-targeting therapy approved for metastatic BTC patients.   The drug takes a new approach to cancer treatment. As a bispecific antibody, it can simultaneously bind two non-overlapping epitopes of HER2. The unique design facilitates dual HER2 signal blockade, increased binding, and removal of HER2 protein from the cell surface. This leads to encouraging antitumor activity in patients.   In addition to the Breakthrough Therapy designation, the FDA has also granted two Fast Track designations to zanidatamab – one for refractory BTC as a single agent and one in combination with chemotherapy for first-line gastroesophageal adenocarcinoma. The drug also received Orphan Drug designations for the treatment of biliary tract, gastric and ovarian cancers from the U.S. Moleculin Biotech’s WP1066  With unexpected and encouraging results, Moleculin announced approval for a Rare Pediatric Disease designation for its child brain tumor drug candidate WP1066.   The designation also comes with a transferrable Priority Review Voucher (PRV) once the company submits an NDA for each of the three indications – diffuse intrinsic pontine glioma (DIPG), medulloblastoma and atypical teratoid rhabdoid tumor. PRV’s have proven to be quite valuable to companies who sell for a tidy profit, up to $100 million or more.  Moleculin’s focus is on groundbreaking therapies for highly resistant cancers and viruses. The blood-brain barrier keeps many cancer drugs from reaching brain tumors, which severely limits treatment options. The past 12 years of clinical trials for brain tumor drugs have yet to show a statistically significant improvement in overall survival.  Inspired by active ingredients found in bee pollen, molecule WP1066 modulates both the immune response to tumors and the aberrant activity of oncogenic transcription factors that enable tumor survival and progression. The patient’s own immune system will then fight tumors while reducing cell signaling that supports tumor activity.   "The approval of these three Rare Pediatric Disease designations is a reminder of just how important our efforts are to potentially help children with brain tumors,” said Walter Klemp, Chairman and CEO of Moleculin.  Y-mAbs Therapeutics’ Danyelza  Y-mAbs' monoclonal antibody has been granted accelerated approval by the FDA. Danyelza, in combination with granulocyte-microphage colony-stimulating factor, is turning up to be a much-needed treatment for patients 1 year and older with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow.   Neuroblastoma is by far the most common cancer in infants and accounts for about 6% of all cancers in children. The average age for diagnosis is 1-2 years old, with 90% diagnosed by age 5. For high-risk neuroblastoma, the target of Y-mAbs drug, the 5-year survival rate is around 40-50%.  The accelerated approval is based on overall response rate and duration of response in two pivotal studies. A confirmatory, postmarketing clinical trial will enroll a minimum of 80 patients with a primary endpoint of overall response rate.  “Today is an important day for children living with refractory/relapsed high-risk neuroblastoma,” said Thomas Gad, founder, Chairman and President. “It’s very exciting to see this treatment go from being an experimental therapy used at my daughter’s bedside to now being FDA approved. On behalf of Y-mAbs, I want to thank all the patients and physicians who took part in our clinical trials and our scientific partner, Memorial Sloan Kettering, for helping us achieve this goal.”  Danyelza should be available in the U.S. in the coming weeks.   Most read today on BioSpace:
JHVePhoto/Shutterstock 提供安全的药物来治疗严重的,危险的疾病是所有医疗保健的首要任务。FDA开发了四种不同的方法,以使用于罕见,儿科和其他未成功治疗的疾病的药物尽可能快地获得:优先审查,突破性治疗,加速批准和快速通道。这里有三种药物本周获得了其中的一个区别。 Zymeworks公司Zanidatamab Zanidatamab获得FDA突破性治疗方案,用于先前治疗过的HER2基因扩增胆道癌(BTC)患者。当临床证据显示在某一特定终点上比现有疗法有显著改善时,突破性疗法的名称被授予用于治疗严重疾病的新药。这一指定是根据正在进行的药物I期研究而授予的。 BTC是一种罕见的侵袭性癌症,影响胆道。约占所有成人癌症的3%,被诊断者的预后通常很差。扎尼达他单抗有潜力成为首个被批准用于转移性BTC患者的HER2靶向疗法。 这种药采用了一种治疗癌症的新方法。作为一种双特异性抗体,它能同时结合HER2的两个不重叠的表位。独特的设计促进了双重HER2信号阻断,增加了结合,并从细胞表面去除HER2蛋白。这导致鼓励患者的抗肿瘤活性。 除了突破性疗法的命名外,FDA还授予扎尼达他单抗两个快速通道命名--一个作为单一药物用于难治性BTC,另一个联合化疗用于一线胃食管腺癌。该药物还获得了美国的孤儿药称号,用于治疗胆道,胃癌和卵巢癌。 分子生物技术公司的WP1066 取得了意想不到和令人鼓舞的结果,Moleculin宣布批准其儿童脑肿瘤候选药物WP1066的罕见儿科疾病命名。 一旦公司针对三种适应症--弥漫性固有脑桥胶质瘤(DIPG),髓母细胞瘤和不典型畸胎样横纹肌样瘤--提交NDA,该指定还附带可转移优先审查凭证(PRV)。事实证明,PRV对那些利润丰厚,高达1亿美元或更多的公司来说是相当有价值的。 Moleculin的重点是对高抗药性癌症和病毒的开创性疗法。血脑屏障使许多抗癌药物无法到达脑肿瘤,这严重限制了治疗选择。过去12年的脑肿瘤药物临床试验尚未显示总生存率有统计学意义上的显著改善。 受到蜂花粉中活性成分的启发,分子WP1066既调节对肿瘤的免疫反应,又调节使肿瘤存活和进展的致癌转录因子的异常活性。患者自身的免疫系统随后将对抗肿瘤,同时减少支持肿瘤活动的细胞信号。 Moleculin公司董事长兼首席执行官沃尔特·克勒姆普说:“这三种罕见儿科疾病的批准提醒我们,我们的努力在潜在地帮助患有脑瘤的儿童方面是多么重要。 Y-mAbs Therapeutics公司的Danyelza Y-Mabs的单克隆抗体已经获得FDA的加速批准。Danyelza与粒细胞-微噬细胞集落刺激因子联合治疗1岁及以上复发或难治性高危骨或骨髓神经母细胞瘤是一种急需的治疗方法。 神经母细胞瘤是迄今为止婴儿最常见的癌症,约占儿童所有癌症的6%。确诊的平均年龄为1-2岁,5岁确诊的占90%。对于高危神经母细胞瘤,Y-mAbs药物的靶点,5年生存率在40-50%左右。 加速批准的依据是两项关键研究的总体答复率和答复持续时间。一项验证性的上市后临床试验将纳入至少80名患者,主要终点为总体应答率。 创始人,董事长兼总裁托马斯·盖德说:“今天对于患有难治/复发高危神经母细胞瘤的儿童来说是一个重要的日子。“看到这种疗法从在我女儿床边使用的试验性疗法到现在获得FDA的批准,这是非常令人兴奋的。我要代表Y-mAbs感谢所有参加我们临床试验的患者和内科医生,感谢我们的科学合作伙伴,纪念斯隆·凯特林,感谢他们帮助我们实现了这一目标。“ Danyelza应该会在未来几周在美国上市。 《生物空间》杂志今天的读者最多: