PubMed Journals Articles About "Silencing Technology Gets First Drug Approval After Year" RSS

05:32 EST 21st January 2019 | BioPortfolio

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Showing "silencing technology gets first drug approval after year" PubMed Articles 1–25 of 22,000+

First granted example of novel FDA trial design under Expedited Access Pathway for premarket approval: BeAT-HF.

The Food and Drug Administration (FDA) initiated the Expedited Access Pathway (EAP) to accelerate approval of novel therapies targeting unmet needs for life-threatening conditions. EAP allows for the possibility of initial FDA approval using intermediate end points with postapproval demonstration of improved outcomes.

Proposed study designs for approval based on a surrogate endpoint and a post-marketing confirmatory study under FDA's accelerated approval regulations for disease modifying osteoarthritis drugs.

In 1992, the Food and Drug Administration (FDA) instituted the accelerated approval regulations that allow drugs or biologics for serious conditions that fill an unmet medical need to be approved on the basis of a surrogate endpoint or an intermediate clinical endpoint. The current definition of a serious condition includes chronic disabling conditions, such as osteoarthritis (OA), and thereby provides expanded opportunities for the use of biomarkers for regulatory approval of drugs for OA. The use of surro...

Identification of Factors Associated with First-Cycle Drug Approval Rates and Regulatory Outcomes for New Drug Applications.

The aims of this study were to identify types of deficiencies resulting in delay of approvals for drugs eventually approved by the US FDA and to search for factors associated with higher first-cycle approval rates. Review documents of New Drug Applications approved between 2008 and 2017 were retrieved from the Drugs@FDA database. Basic characteristics of the applications, regulatory actions, and reasons for non-approvals and/or major amendments after first review cycle were investigated. Of 825 applications...

Engineering artificial microRNAs for multiplex gene silencing and simplified transgenic screen.

Artificial microRNA (amiRNA) technology offers reversible and flexible gene inactivation and complements genome editing technologies. However, obtaining transgenic plants with maximal gene silencing remains a major technical challenge in current amiRNA applications. Here, we incorporated an empirically determined feature of effective amiRNAs to the amiRNA design and in silico generated a database containing 533,429 gene-specific amiRNAs for silencing 27,136 genes in Arabidopsis thaliana, with a genome cover...

Impact of Food and Drug Administration Approval of Vismodegib on Prevalence of Orbital Exenteration as a Necessary Surgical Treatment for Locally Advanced Periocular Basal Cell Carcinoma.

To test the hypothesis that the US Food and Drug Administration approval of vismodegib in early 2012 has reduced the prevalence of orbital exenteration for locally advanced periocular basal cell carcinoma (BCC).

A 61-Year-Old Caribbean Man With Thrush and Acute Respiratory Failure.

A 61-year-old Caribbean man presented to the ED with dyspnea that had progressed over the previous week with associated cough and high fevers. Four days prior to admission, his primary care physician noted oral thrush and obtained a chest radiograph that revealed a right middle lobe infiltrate. He was prescribed levofloxacin and clotrimazole. Despite therapy, his symptoms progressed. He had an 11 pack-year smoking history and hypertension but had been in good health. He denied recent travel, alcohol or illi...

Perspective: Regulation of Pest and Disease Control Strategies and Why (Many) Economists are Concerned.

Pests and diseases are a continuous challenge in agriculture production. A wide range of control strategies have been and will continue to be developed. New control strategies are in almost all countries around the world assessed prior to approval for use in farmers' fields. This is rightly so to avoid and even reduce negative effects for human health and the environment. Over the past decades the approval processes have become increasingly politicized resulting in an increase in the direct approval costs a...

Pyrotinib: First Global Approval.

Pyrotinib is an irreversible dual pan-ErbB receptor tyrosine kinase inhibitor developed for the treatment of HER2-positive advanced solid tumours. Based on positive results in a phase II trial, the drug recently received conditional approval in China for use in combination with capecitabine for the treatment of HER2-positive, advanced or metastatic breast cancer in patients previously treated with anthracycline or taxane chemotherapy. This article summarizes the milestones in the development of pyrotinib l...

Information Transparency in the Drug Approval Process.

First Approval of a Drug Derived from Marijuana.

Using real world data to extrapolate evidence from randomized controlled trials.

Randomized controlled trials provide evidence for regulatory agencies, shape clinical practice, influence formulary decisions and have important implications for patients. However, many patient groups that are major consumers of drugs are underrepresented in randomized trials. We review 3 methods to extrapolate evidence from trial participants to different target populations following market approval and discuss how these could be implemented in practice to support regulatory and health technology assessmen...

Key Elements in Adverse Drug Reactions Safety Signals: Application of Legal Strategies.

Adverse drug reactions, or unintended and harmful outcomes related to the administration of a pharmaceutical product, are a major public health concern, particularly for cancer patients. If counted as a separate cause of death, adverse drug reactions would represent the fourth leading cause of death in the United States. Several legal strategies are available to help mitigate their occurrences and to compensate victims for the harm that results from adverse events. Prior to FDA approval of a drug, the limit...

Streamlining nonclinical drug development using the FDA 505(b)(2) new drug application regulatory pathway.

In the USA, drugs are approved by the FDA by three main regulatory pathways: (i) 505(b)(1) new drug applications (NDAs); (ii) 505(b)(2) NDAs; and (iii) 505(j) abbreviated NDAs (ANDAs). The appropriate pathway depends on the active ingredient, already approved drug products, drug formulation, clinical indication, route of exposure, among other factors. The 505(b)(2) NDA pathway is a regulatory approval pathway that allows sponsors to use existing public data in lieu of conducting studies; thus, potentially o...

Chinese approval for Ascletis' HCV drug is first homegrown success.

Information Transparency in the Drug Approval Process-Reply.

Management of complex tuberculosis cases: a focus on drug-resistant tuberculous meningitis.

Drug-resistant tuberculous meningitis has been reported worldwide. Isoniazid mono-resistance is the most frequent cause of drug-resistant tuberculous meningitis, a life-threatening disease. Extensive drug-resistant tuberculous meningitis has also been reported in some isolated case reports. Areas covered: We reviewed the current literature on drug-resistant tuberculous meningitis, as well as drug-resistant tuberculosis. Expert commentary: Drug-resistant tuberculous meningitis is a life-threatening disease a...

Changing standards for drug approval: A longitudinal analysis of conditional marketing authorisation in the European Union.

Drug regulatory agencies around the world increasingly implement expedited regulatory pathways allowing for approval of medicines that intend to address unmet medical needs based on lower evidentiary standards than would be conventionally required. Few studies have investigated how companies and regulators utilise these pathways. We therefore conducted a longitudinal analysis of the emergence and implementation of the conditional marketing authorisation (CMA) instrument in the European Union. Drawing on arc...

Postmarketing Safety-Related Modifications of Drugs Approved by the US Food and Drug Administration Between 1999 and 2014 Without Randomized Controlled Trials.

To investigate whether US Food and Drug Administration approval of new drugs without randomization or an active drug comparator is associated with more postmarketing safety-related label modifications.

Duration of Pediatric Clinical Trials Submitted to the US Food and Drug Administration.

The increasing prevalence of pediatric chronic disease has resulted in increased exposure to long-term drug therapy in children. The duration of recently completed drug trials that support approval for drug therapy in children with chronic diseases has not been systematically evaluated. Such information is a vital first step in forming safety pharmacovigilance strategies for drugs used for long-term therapy in children.

Ethical approval for multicenter cohort studies on drug exposure during pregnancy: a survey among members of the European Network of Teratology Information Services (ENTIS).

The European Network of Teratology Information Services (ENTIS) is in a privileged position to perform independent post-marketing surveillance of drugs in pregnancy. The aim of this survey was to describe the legal requirements and procedures involved in obtaining ethical approval for collaborative cohort studies. We sent a survey questionnaire to all 28 Teratology Information Services (TIS), of which 25 (89%) in 18 countries completed our questionnaire. For 15 TIS, specific research ethical approval was ma...


Despite FDA approval of growth hormone(GH) for idiopathic short stature(ISS), many providers face challenges obtaining insurance coverage. We reviewed the insurance coverage experience for ISS at our hospital to identify factors predictive of approval or denial.

Fexinidazole: First Global Approval.

Fexinidazole Winthrop (hereafter referred to as fexinidazole) is a DNA synthesis inhibitor developed by the Drugs for Neglected Diseases initiative (DNDi), in collaboration with Sanofi, for the oral treatment of human African trypanosomiasis (HAT) [commonly known as 'sleeping sickness'] and Chagas' disease. The drug is a 5-nitroimidazole derivative first discovered by Hoechst AG (now part of Sanofi) and was identified by the DNDi in 2005 as having activity against Trypanosoma brucei gambiense and T. b. rhod...

Studying cellular heterogeneity and drug sensitivity in colorectal cancer using organoid technology.

Intra-tumor heterogeneity (genotypic and functional diversity among cancer cells within the same tumor) represents one of the key challenges in cancer medicine. As heterogeneity of cancer cells constitutes an important parameter in the development of therapy resistance, an accurate assessment of intra-tumor heterogeneity is essential for the prediction of drug resistance and development of effective treatment. In this review, we evaluate primary patient derived-tumor organoid technology as a new tool for co...

The A-Z of Zika drug discovery.

Despite the recent outbreak of Zika virus (ZIKV), there are still no approved treatments, and early-stage compounds are probably many years away from approval. A comprehensive A-Z review of the recent advances in ZIKV drug discovery efforts is presented, highlighting drug repositioning and computationally guided compounds, including discovered viral and host cell inhibitors. Promising ZIKV molecular targets are also described and discussed, as well as targets belonging to the host cell, as new opportunities...

Sarecycline: First Global Approval.

Sarecycline (Seysara™) is an oral, once-daily, tetracycline-class drug for which a tablet formulation is approved in the USA for the treatment of inflammatory lesions of non-nodular moderate to severe acne vulgaris in patients aged ≥ 9 years. The drug was developed by Paratek and Allergen and later acquired by Almirall S.A. (a Barcelona-based pharmaceutical company focused on medical dermatology). Sarceycline tablets were approved in early October 2018 and are planned to be available for patients i...

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