Drugs of Today

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Epithelial ovarian cancers are gynecological malignancies with the poorest prognosis. Intensive research over the past few years has demonstrated ovarian cancer is a type of cancer in which new molecularly targeted drugs significantly affect patients' fate and prognosis. These drugs are poly [ADP-ribose] polymerase (PARP) inhibitors, which are used for maintenance treatment. These molecules continue to be intensively studied--their combination with other targeted therapies is carefully evaluated as more of them are discovered...

In July 2020, the U.S. Food and Drug Administration (FDA) approved brexucabtagene autoleucel (BA), the first anti-CD19 chimeric antigen receptor (CAR) T-cell therapy for the treatment of relapsed/refractory mantle cell lymphoma (MCL). The pivotal ZUMA-2 trial led to the approval of BA in patients who experienced relapsed disease on prior therapies (chemotherapy and/or Bruton tyrosine kinase [BTK] inhibitors). The FDA approval of BA was based on excellent responses with this therapy in highly refractory patients with MCL, who conventionally had poor outcomes...

Psoriasis is a chronic, immune-mediated, inflammatory skin disease, affecting 1% to 3% of the population in Western countries. Due to advances in the understanding of psoriasis pathogenesis, in particular, the role of the interleukin-23 (IL-23)/T-helper 17 (Th17) immune axis, highly effective, targeted biologic therapies have been developed, shifting the psoriasis treatment paradigm. However, some patients do not respond or lose response to these novel therapies. Bimekizumab is a first-in-class humanized monoclonal immunoglobulin G1 (IgG1) antibody that potently and selectively inhibits both IL-17A and IL-17F, functioning as a dual inhibitor...

Acute lymphoblastic leukemia (ALL) is a neoplastic disease characterized by the malignant proliferation of lymphoid cells in the blood and bone marrow. It accounts for approximately 75% of childhood leukemia. Lymphoblastic lymphoma (LBL) is a type of non-Hodgkin's lymphoma characterized by rapid growth and highly aggressive characteristics that occurs most commonly in adolescents and young adults. Asparaginase is primarily used to treat patients with ALL or LBL. Because allergic reactions occur in patients treated with bacterial-derived asparaginase, it is important to develop an alternative asparaginase preparation for patients allergic to asparaginase...

The Drug Information Association (DIA) Europe held its 2022 annual meeting as a hybrid event, both as an in-person meeting in Brussels, Belgium, and as a virtual meeting. Many topics were discussed during this 3-day meeting, including the lessons learnt from the first 5 years' experience of the PRIME (PRIority MEdicine) scheme, the benefits and tools of the Clinical Trials Regulation, the roadmap of the electronic product information, the implementation of the substance, product, organization and referentials (SPOR) management services, and how real-world data, real-world evidence and digital health tools are changing the way clinical trials and research are performed...

Outpatient treatment options for mild to moderate COVID-19 are severely limited. While many therapeutic options have been proposed, very few have demonstrated the appropriate safety and efficacy to warrant approval by national or international regulatory bodies. Monoclonal antibodies have been shown to decrease hospitalization in high-risk patients, but use remains limited due to challenges associated with both production and administration, and other treatment options are urgently needed. The anti-inflammatory drug fluticasone propionate has recently emerged as a potential outpatient treatment option, especially for those with newly diagnosed disease...

Gene or advanced therapy medicinal products (ATMPs) are evolving innovative products that have recently been attracting medical and healthcare attention. ATMPs are offering advanced techniques in treating certain conditions, including rare diseases. Meanwhile, the short- and long-term safety and efficacy of these products raise concerns. In this study, a comparative analysis of gene therapy medicinal products in the U.S. and E.U. was carried out to provide an overview of their efficacy and safety. Data up until 2019 were collected from the Food and Drug Administration (FDA) and European Medicines Agency (EMA) regarding approved gene therapy products...

Cervical cancer is one of the most common gynecological malignancies. At present, cytotoxic chemotherapeutic drugs and immunotherapy are the main therapeutic options for recurrent and metastatic cervical cancer. Tisotumab vedotin is an antibody-drug conjugate (ADC) and a potential novel treatment for cervical carcinoma. Tisotumab vedotin targets tissue factor (TF), which is highly expressed on the surface of cervical cancer cells, by delivering the cytotoxic agent monomethyl auristatin E (MMAE) directly into tumor cells...

Chronic graft-versus-host disease (cGvHD) is a multisystem disease that is diagnosed in up to 70% of patients following allogeneic hematopoietic cell transplantation. In cGvHD, the donor immune cells attack the patient's cells, resulting in inflammation and fibrosis in multiple tissues. cGvHD can affect almost any organ and is the major cause of morbidity and mortality in transplant survivors. Rho-associated coiled-coil-containing protein kinase (ROCK) is a signaling pathway that modulates inflammatory response and fibrotic processes and is dysregulated in autoimmune disorders...

Tanezumab is a novel humanized IgG2 monoclonal antibody that works by selectively targeting, binding to and inhibiting nerve growth factor (NGF). NGF is upregulated in response to injury and inflammation, and preclinical data indicate it plays a role in pain signaling by inducing peripheral and central sensitization. Tanezumab potentially reduces sensitization and pain by blocking the interaction between NGF and the tropomyosin receptor kinase A (TrkA), and it has been studied extensively for the treatment of pain in patients with osteoarthritis (OA)...

Sotorasib, a direct inhibitor of the enzyme Kirsten rat sarcoma viral oncogene (KRAS) with the G12C mutation, was approved by the U.S. Food and Drug Administration (FDA), as a second-line treatment for locally advanced or metastatic non-small cell lung cancer (NSCLC) containing the KRAS G12C mutation, on the basis of results of a phase II clinical trial (Code- BreaK100). In this article, we review the mechanism of action of KRAS G12C inhibitors and the latest clinical trials with sotorasib to provide a comprehensive understanding of its efficacy and toxicity...

Atopic dermatitis (AD) is a common inflammatory skin disease that has emerging treatments targeting the underlying immunological mechanism. Interleukin-31 (IL-31) is associated with the pathobiological mechanism of AD, contributing to symptoms such as dermatitis and pruritus. Nemolizumab is an anti-IL-31 receptor α-chain (IL-31RA) monoclonal antibody agent that is efficacious in improving symptoms of AD in several phase II and phase III studies in recent years. Nemolizumab demonstrates great efficacy in reducing pruritus and to a lesser degree, dermatitis associated with AD...

Worldwide, effective management of vulvovaginal candidiasis (VVC) continues to serve as a major therapeutic goal with numerous unmet drug treatment challenges. After 3 decades of azole drug dominance, with few recent new antifungal agents and little progress in VVC management, the first-in-class oral triterpenoid glucan synthase inhibitor agent ibrexafungerp has emerged in the treatment of acute VVC. After reviewing existing treatment standards and unmet needs, the pharmacology, pharmacokinetics, antimicrobial activity and clinical efficacy of ibrexafungerp are reviewed in this article together with phase III clinical trial results and drug safety...

Detonation of an improvised nuclear weapon, or a radiological dispersal device by terrorists, or an unintended radiological/nuclear accident in populated areas would result in a mass casualty scenario with radiation exposures of different severities. Such incidences are perceived as national security threats of major consequences. Acute radiation syndrome (ARS) is triggered by an exposure to a high dose of penetrating ionizing radiation during a short time window. In humans, moderate exposure to 2 to 4 Gy of ionizing radiation results in clinically manageable hematopoietic ARS (H-ARS), characterized by severe depletion of vital blood cells and bone marrow progenitors...

The introduction of new classes of drugs for the treatment of multiple myeloma (MM) in the past 2 decades, such as proteasome inhibitors, immunomodulators and anti-CD38 monoclonal antibodies, coupled with autologous stem cell transplantation, has approximately doubled the 5-year survival rate of MM patients. However, the patients eventually relapse and/or become resistant to the drugs and treatment. The recent emergence of anti-B-cell maturation antigen (BCMA) therapies, especially chimeric antigen receptor T-cell (CAR-T) immunotherapy targeting BCMA, holds great prospect in MM treatment...

Hemophilia A and B are X-linked hereditary bleeding disorders due to factor VIII (FVIII) or factor IX (FIX) deficiency, respectively. Major advancements have been made in the care of patients with hemophilia, yet the development of inhibitors to infused FVIII or FIX continues to be a formidable challenge. The current first-line therapy for acute bleeding episodes in patients diagnosed with inhibitors are bypassing agents including activated prothrombin complex concentrates (aPCCs) and recombinant factor VIIa (rFVIIa)...

Cancer anorexia-cachexia syndrome is a multifactorial condition characterized by significant weight loss due to muscle loss. It is associated with functional impairment, changes in body composition and nutritional disorders. Ghrelin receptors are involved in the release of growth hormone (GH) in the pituitary gland and increase appetite via the hypothalamus. The secretion of GH from the pituitary gland stimulates the liver to secrete insulin-like growth factor 1 (IGF-1), which promotes muscle protein synthesis...

Ibrutinib, an oral small-molecule targeted drug, has been the first Bruton tyrosine kinase (BTK) inhibitor in the world to be approved for the market. It works by regulating cell proliferation, apoptosis and migration, and has been proven to exhibit high efficacy and good safety in the treatment of B-cell lymphomas, including chronic lymphocytic leukemia or small lymphocytic lymphoma and mantle cell lymphoma. However, some patients inevitably have drug resistance and disease recurrence, resulting in a poor prognosis...

Loteprednol etabonate is a soft corticosteroid that is rapidly deactivated after reaching the general circulation, displaying good local activity and a high therapeutic index without inducing systemic side effects. In 2021, Kala Pharmaceuticals launched Eysuvis (loteprednol etabonate ophthalmic suspension) 0.25% in the U.S. for the short-term (up to 2 weeks) treatment of the signs and symptoms of dry eye disease. Approval by the Food and Drug Administration (FDA) was based on results from one phase II trial and three phase III trials showing Eysuvis significantly improved both the signs and symptoms of dry eye disease and was well tolerated...