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Beleodaq approved for rare lymphomas.

On July 3, the FDA granted accelerated approval to Beleodaq, a histone deacetylase inhibitor, for the treatment of patients with relapsed or refractory peripheral T-cell lymphoma.

Icosapent ethyl for the treatment of hypertriglyceridemia.

INTRODUCTION: Icosapent ethyl (IPE; Vascepa) is a high-purity prescription form of eicosapentaenoic acid (EPA) ethyl ester recently approved in 2012 to reduce triglyceride (TG) levels in adult patients with severe (≥ 500 mg/dL) hypertriglyceridemia. Elevated TG levels are associated with increased risk for coronary heart disease. Currently available TG-lowering agents (fibrates, niacins, omega-3 fatty acid products containing both EPA and docosahexaenoic acid [DHA]) may be associated with adverse effects such as flushing, hepatotoxicity, myopathy, elevated glucose levels, and/or increases in l

Saxagliptin. No more effective than other gliptins, but a high potential for drug interactions.

Sitagliptin and vildagliptin are dipeptidyl peptidase 4 (DPP-4) inhibitors, also known as "gliptins". They are approved for use as oral glucose-lowering agents, although they have no proven impact on morbidity or mortality. Their glucose-lowering effect is limited and their long-term risks are poorly documented. A third gliptin, saxagliptin, is now authorised in the European Union for second-line treatment of type 2 diabetes, in combination with metformin, a glucose-lowering sulphonylurea, or a glitazone. Clinical evaluation of saxagliptin is mainly based on 8 double-blind randomised trials in

Ramelteon: new drug. Insomnia: no role for risky placebos.

(1) Patients complaining of insomnia should first be treated with non-drug measures (information, advice). Short-course benzodiazepine therapy can be tried if non-drug measures and established herbal remedies fail; (2) Ramelteon, a drug that antagonises receptors for melatonin, a hormone involved in circadian rhythms, is being considered for European marketing authorization in the treatment of insomnia; (3) Ramelteon has only been compared with placebo in clinical trials. Only one of three trials in which the patients were studied in their normal environment showed that ramelteon reduced the t

Olanzapine: new indication. Prevention of bipolar disorder: unconvincing trials.

(1) Lithium, the standard preventive treatment for patients with bipolar disorder, reduces the number of relapses and suicide attempts. (2) Olanzapine is the first neuroleptic to be approved in France for prevention of relapse in patients with bipolar disorder. Many neuroleptics are already used for this indication but their efficacy has not been established in comparative clinical trials. (3) One placebo-controlled double-blind trial involved 361 patients who were treated just after recovering from a manic episode. The trial was supposed to last 48 weeks, but only 146 patients were treated fo

Small molecules in targeted cancer therapy: advances, challenges, and future perspectives.

Due to the advantages in efficacy and safety compared with traditional chemotherapy drugs, targeted therapeutic drugs have become mainstream cancer treatments. Since the first tyrosine kinase inhibitor imatinib was approved to enter the market by the US Food and Drug Administration (FDA) in 2001, an increasing number of small-molecule targeted drugs have been developed for the treatment of malignancies. By December 2020, 89 small-molecule targeted antitumor drugs have been approved by the US FDA and the National Medical Products Administration (NMPA) of China. Despite great progress, small-mol

Orphan drug development in alpha-1 antitypsin deficiency.

Alpha-1 antitrypsin deficiency (AATD, OMIM #613490) is a rare metabolic disorder affecting lungs and liver. The purpose of this study is to assess the impact of the US orphan drug act on AATD by providing a quantitative clinical-regulatory insight into the status of FDA orphan drug approvals and designations for compounds intended to treat AATD. This is across-sectional analysis of the FDA database for orphan drug designations. Primary endpoint: orphan drug approvals. Secondary endpoint: orphan drug designations by the FDA. Close of database was 16 July 2021. STROBE criteria were respected. Pr…

The impact of level of documentation on the accessibility and affordability of new drugs in Norway.

Introduction: Over the preceding decade, an increasing number of drugs have been approved by the European Medicines Agency (EMA) with limited knowledge of their relative efficacy. This is due to the utilization of non-randomized, single-arm studies, surrogate endpoints, and shorter follow-up time. The impact of this trend on the accessibility and affordability of newly approved drugs in Europe remains uncertain. The primary objective of this study is to provide insights into the issues of accessibility and affordability of new drugs in the Norwegian healthcare system. Method: The presented stu

Совпадение в полном тексте источника
… Five years of cancer drug approvals: innovation, efficacy, and costs. JAMA Oncol. 1 (4), 539–540. 10.1001/jamaoncol.2015.0373 Malinowski K. P., Kawalec P., Trabka W., Sowada C., Pilc A. (2018). … Magnitude of clinical benefit of cancer drugs approved by the US food and drug administration. J. Natl. Cancer Inst. 110 (5), 486–492. 10.1093/jnci/djx232 Tichy E. M., Hoffman J. M., Suda K. J., Rim M. H., Tadrous M., Cuellar S., et al. (2022). …

Adverse Reactions Identified after Approval of Novel Medicines: Analysis of 339 New Molecular Entities and Therapeutic Biologics.

Postmarketing safety surveillance data identify safety issues not identified in preapproval clinical trials. We describe the frequency of new safety issues overall and by labeling section and the relationship of the most commonly added safety issues with the cumulative number of prescriptions dispensed via retail pharmacies. Using publicly available sources, we determined the frequency of the most common safety issues added after approval overall and by labeling section (Boxed Warning, Warnings and Precautions, and Adverse Reactions) for new molecular entities (NMEs) and new therapeutic biolog

An introduction to biosimilar cancer therapeutics: definitions, rationale for development and regulatory requirements.

Monoclonal antibodies and other biologic drugs play important roles in the treatment of various hematological malignancies and solid tumors. However, such drugs are intrinsically more expensive to develop than small molecules and their clinical benefits are often accompanied by challenges relating to affordability and access. Patent expiry for 'originator' biologics is providing opportunities for a new generation of biosimilar drugs, potentially capable of relieving pressure on healthcare budgets. This article discusses key characteristics of biosimilars, distinguishes them from generics and n

Treatment of animal toxicoses: a regulatory perspective.

This article focuses on the regulatory issues to consider when veterinarians are called upon to treat animal toxicoses, in particular those involving food-producing animals. The lack of Food and Drug Administration-approved drugs to treat animal toxicoses has been a long-standing problem. This article reviews extralabel drug use regulations, and the responsibilities of the treating veterinarian. It discusses the legal implications of compounding and the use of unapproved drugs to treat animal toxicoses. Efforts should be made to increase the availability of life-saving antidotal therapies.

Canadian drug regulatory framework.

The role of regulatory drug submission evaluators in Canada is to critically assess both the data submitted and the sponsor's interpretation of the data in order to reach an evidence-, and context-based recommendation as to the potential benefits and potential harms (i.e., risks) associated with taking the drug under the proposed conditions of use. The purpose of this document is to outline the regulatory framework in which this assessment occurs, including: defining what "authorization to market a drug in Canada" means, in terms of the role of the sponsor, the responsibility of Health Canada

Teicoplanin inhibits Ebola pseudovirus infection in cell culture.

There is currently no approved antiviral therapy for treatment of Ebola virus disease. To discover readily available approved drugs that can be rapidly repurposed for treatment of Ebola virus infections, we screened 1280 FDA-approved drugs and identified glycopeptide antibiotic teicoplanin inhibiting Ebola pseudovirus infection by blocking virus entry in the low micromolar range. Teicoplanin could be evaluated further and incorporated into ongoing clinical studies.

Delayed Methotrexate Clearance Despite Carboxypeptidase-G2 (Glucarpidase) Administration in 2 Patients With Toxic Methotrexate Levels.

High-dose methotrexate has been a treatment for osteosarcoma; however, its nephrotoxic effects are considerable. Carboxypeptidase-G2 (glucarpidase) was approved by the US Food and Drug Administration in 2012 for treatment of toxic methotrexate levels. We report our experience using glucarpidase under compassionate use before Food and Drug Administration approval in 2 patients who had delayed methotrexate clearance and prolonged kidney injury despite glucarpidase administration. Our results show that patients with methotrexate toxicity may require repeated doses of glucarpidase in addition to s

Biopharmaceuticals for Cancer Treatment: An Update.

Cancer is one of the most debilitating diseases, causing profound physical and psychological impacts on patients. The global number of cancer patients has been steadily rising over the last few decades. The development of effective cancer therapies remains the primary focus in life science research, requiring a precise understanding of specific pathways and the regulation of tumor markers. The approved cancer therapies demonstrate effectiveness by acting on specific regulatory enzymes, components required for cell division, hormones, and cell-specific receptors, etc. While small-molecule drugs