Cemiplimab for the treatment of advanced cutaneous squamous cell carcinoma.
Authors: Hernández-Guerrero T, Doger B, Moreno V Abstract Cutaneous squamous cell carcinoma (cSCC) is the second most frequent type of malignancy in Caucasians worldwide. Several factors have been correlated with aggressiveness and likelihood of recurrence and distant metastases, which are challenging to control. Metastatic disease has a dismal prognosis, and standard chemotherapy has failed to significantly improve outcomes. Recently, it has been recognized that cSCCs are highly mutated tumors with a denoting potential likelihood of response to immune checkpoint blockade. Cemiplimab is an anti-programmed ce...
Source: Drugs of Today - August 29, 2019 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research

Erdafitinib to treat urothelial carcinoma.
Authors: Hanna KS Abstract Erdafitinib is the first Food and Drug Administration (FDA)-approved oral pan-fibroblast growth factor receptor (FGFR) kinase inhibitor that binds to four FGFRs (FGFR-1 to -4), leading to decreased cell signaling and cellular apoptosis. Erdafitinib also binds to RET, colony-stimulating factor 1 receptor (CSF-1R), platelet-derived growth factor receptor α and β (PDGFR-α and PDGFR-β), Fms-related tyrosine kinase 4 (FLT4), KIT and vascular endothelial growth factor receptor 2 (VEGFR-2), exhibiting additional antitumor mechanisms resulting in cell kill. In this article, ...
Source: Drugs of Today - August 29, 2019 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research

Relugolix for the treatment of uterine fibroids.
Authors: Barra F, Seca M, Della Corte L, Giampaolino P, Ferrero S Abstract Uterine leiomyomas represent the most common form of benign gynecological tumors affecting 20-40% of women during their life. Several therapeutic options are available for treating these patients. The use of medical treatment for myomas has largely grown in the last years, in particular for women who would refuse, postpone or are not candidates for surgery. In the last years, the clinical investigation of gonadotropin-releasing hormone (GnRH) antagonists (GnRH-ants) has emerged. This class of drugs exerts pure competitive antagonistic activi...
Source: Drugs of Today - August 29, 2019 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research

Combating colorectal cancer with immunotherapy: Where are we?
Authors: Gupta R, Bhatt LK, Prabhavalkar KS Abstract Despite incessant advancement in the therapeutic regimens against colorectal cancer (CRC), treatment failure, metastasis and serious side effects associated with the available therapeutic options demand specific targeted therapies that could help in improving the survival rate with significantly less toxicity, fewer untoward effects and improved efficacy. In-depth studies highlighted the potential of CRC to be immune-responsive, thereby opening a new door in the development of strategies to combat CRC. Immunotherapy has attracted a myriad of researchers with a ho...
Source: Drugs of Today - August 29, 2019 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research

Gepirone hydrochloride: a novel antidepressant with 5-HT1A agonistic properties.
Authors: Kaur Gill A, Bansal Y, Bhandari R, Kaur S, Kaur J, Singh R, Kuhad A, Kuhad A Abstract Depression is a neuropsychiatric disorder that affects more than 350 million people all over the world. There are psychological and pharmacological treatments for depression which mainly focus on monoaminergic neurotransmission theory. The main concern regarding available antidepressants is the lag period and other side effects, such as sexual dysfunction. Gepirone is a drug of the azapirone group which is a 5-HT1A receptor agonist belonging to the buspirone family. Gepirone is under clinical development and has been show...
Source: Drugs of Today - July 29, 2019 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research

Lanadelumab to treat hereditary angioedema.
Authors: Wedi B Abstract Lanadelumab is a human monoclonal antibody against plasma kallikrein indicated for prevention of attacks of hereditary angioedema (HAE). HAE is caused by SERPING1 gene mutations resulting in decreased or dysfunctional plasma protease C1 inhibitor (C1-INH) leading to a loss of inhibition of plasma kallikrein activity with subsequent cleavage of high-molecular weight kininogen and release of bradykinin. There is a clear need for a non-plasma-derived, safe, effective and convenient prophylaxis of HAE attacks to reduce patients' daily burden of disease and disability. The percentage of patients...
Source: Drugs of Today - July 29, 2019 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research

Contraceptive vaginal ring containing segesterone acetate and ethinyl estradiol: long-acting, patient-controlled, procedure-free, reversible prescription birth control.
Authors: Paton DM Abstract The Food and Drug Administration (FDA) approved on August 10, 2018, a soft, reusable, flexible silicone ring (56 mm diameter) containing segesterone acetate and ethinyl estradiol as the first contraceptive vaginal ring (CVR) that can be used for a year and that is totally under the control of the woman using it. The vaginal ring releases segesterone and ethinyl estradiol at estimated rates of 150 mcg/day and 13 mcg/day, respectively. The CVR is inserted into the upper two-thirds of the vagina and left in place for 21 days, then removed for 7 days. The same ring can be used for 13 cycles f...
Source: Drugs of Today - July 29, 2019 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research

Talazoparib to treat BRCA-positive breast cancer.
Authors: Guney Eskiler G Abstract Talazoparib tosylate (BMN-673, Talzenna; Pfizer) is an oral poly [ADP-ribose] polymerase (PARP) inhibitor (PARPi) that has been approved by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of germline BRCA-mutated locally advanced or metastatic breast cancer (BC). In preclinical and clinical studies, talazoparib exerted superior efficacy and offered a significant clinical benefit in advanced or metastatic BC patients harboring germline BRCA mutations compared with other PARPi and standard chemotherapy regimens through the concept...
Source: Drugs of Today - July 29, 2019 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research

The neurobiology under the placebo effect.
Authors: Girach A, Aamir A, Zis P Abstract The placebo effect is a phenomenon of great scientific interest that affects the response in both inactive and active treatments. It is broadly understood as the product of a central integration of positive expectations, reward learning and continuous conditioning inducing physiological changes in the brain. The placebo effect is accepted as a phenomenon which can be harnessed in clinical practice. It has emerged that there is not a single neurobiological mechanism involved in placebo responses, but many depending on the underlying disease. Molecular neuroimaging technique...
Source: Drugs of Today - July 29, 2019 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research

Baloxavir marboxil: a novel cap-dependent endonuclease (CEN) inhibitor for the treatment of acute uncomplicated influenza.
Authors: Locke SC, Splawn LM, Cho JC Abstract Baloxavir marboxil is a newly approved antiviral agent with activity against influenza via a novel mechanism of action of inhibition of cap-dependent endonuclease (CEN). The novel agent was approved in October of 2018 in the United States for the treatment of acute uncomplicated influenza A and B in patients aged 12 years or older. Baloxavir is given as a single weight-based dose of 40 mg orally once for patients weighing less than 80 kg and 80 mg orally once for those weighing 80 kg or more within 48 hours of symptom onset. In comparison with current therapy, baloxavir...
Source: Drugs of Today - June 30, 2019 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research

Caplacizumab to treat immune-mediated thrombotic thrombocytopenic purpura.
Authors: Poullin P, Bornet C, Veyradier A, Coppo P Abstract Immune-mediated thrombotic thrombocytopenic purpura (iTTP) is a rare and life-threatening disease characterized by microangiopathic hemolytic anemia, thrombocytopenia and multiorgan failure, resulting from autoantibody-mediated severe A disintegrin and metalloproteinase with thrombospondin motifs 13 (ADAMTS13) deficiency. In spite of treatment with plasma exchange and immunosuppression, patients remain at risk of exacerbations, refractoriness and death. Caplacizumab (Cablivi; Ablynx, a Sanofi company), a nanobody targeting von Willebrand factor (vWF), has ...
Source: Drugs of Today - June 30, 2019 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research

Omidenepag isopropyl for the treatment of glaucoma and ocular hypertension.
Authors: Ferro Desideri L, Cutolo CA, Barra F, Ferrero S, Traverso CE Abstract Glaucoma is a main cause of irreversible vision impairment and its prevalence is expected to rise significantly in the near future. Among the current medications, prostaglandin analogues (PGAs) are widely used and considered as a first-line strategy in the management of glaucoma and ocular hypertension (OHT). However, given the non-negligible incidence of adverse ocular effects (conjunctival hyperemia, increase of iris pigmentation and eyelash changes) due to the use of this class of drugs, novel PGAs are being investigated. Omidenepag i...
Source: Drugs of Today - June 30, 2019 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research

A review of current evidence allied to step-up and top-down medication therapy in inflammatory bowel disease.
Authors: Salahudeen MS Abstract Inflammatory bowel disease (IBD), classified as a global disease in the 21st century, comprises Crohn's disease (CD) and ulcerative colitis (UC), and is manifested by chronic inflammation of the gastrointestinal tract of unknown etiology. The step-up approach is effective for inducing remission and controlling inflammation, whereas the top-down therapy appears to be a better solution for the maintenance of remission with an improved bowel function. A systematic search was performed in Ovid MEDLINE and Embase (1946-December 2018) to identify relevant trials that support medication the...
Source: Drugs of Today - June 30, 2019 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research

American Society of Clinical Oncology (ASCO) - 55th Annual Meeting (May 31-June 4, 2019 - Chicago, Illinois, USA).
This report covers some of the later-stage developments and results presented at the meeting. PMID: 31250844 [PubMed - in process] (Source: Drugs of Today)
Source: Drugs of Today - June 30, 2019 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research

Dolutegravir plus lamivudine dual therapy - a new option for initial antiretroviral therapy.
Authors: Fida M, Rizza SA, Temesgen Z Abstract The current standard of care for treating HIV infection is the use of three antiretroviral drugs: a combination of two nucleoside/nucleotide reverse transcriptase inhibitors (NRTIs) and a third agent from either the integrase strand transfer inhibitor (INSTI), boosted protease inhibitor (PI) or non-nucleoside reverse transcriptase inhibitor (NNRTI) classes. In an effort to minimize the long-term adverse effects and cost of antiretroviral therapy, the use of regimens with fewer drugs in the combination has been under active investigation. To this end, the combination of...
Source: Drugs of Today - May 28, 2019 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research

Lenvatinib mesylate to treat hepatocellular carcinoma.
Authors: Ielasi L, Tovoli F, Piscaglia F Abstract Hepatocellular carcinoma (HCC) is a worldwide healthcare problem, with a rising incidence. In its advanced stage, the prognosis of untreated HCC is very poor. Only in 2007, after a long series of failed trials, the multi-tyrosine kinase inhibitor sorafenib demonstrated its superiority over placebo, becoming the first approved frontline therapy for advanced HCC. For a decade, all of the frontline trials using sorafenib as a comparator systematically failed, leaving this drug as the only available treatment in this setting. In 2018, lenvatinib mesylate (another multit...
Source: Drugs of Today - May 28, 2019 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research

Patisiran for the treatment of patients with familial amyloid polyneuropathy.
Authors: Rizk M, Tüzmen S Abstract Onpattro, also commonly known as patisiran, is a small interfering RNA (siRNA) molecule packaged within a lipid nanoparticle and is transported into the cell to target transthyretin gene (TTR) messenger mRNA (mRNA) by attaching to its complementary sequence. The target mRNA is degraded and both mutant and wild-type amyloid transthyretin (ATTR) protein production becomes suppressed. This drug was developed by Alnylam Pharmaceuticals to treat a rare disease called hereditary ATTR (hATTR) amyloidosis. This disease develops as a result of the deposition of toxic aggregates of mis...
Source: Drugs of Today - May 28, 2019 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research

LDL cholesterol-lowering therapies: emphasis on proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors.
Authors: Sabatel-Pérez F, García-Ropero A, Santos-Gallego CG, Urooj Zafar M, Badimón JJ Abstract Atherosclerotic cardiovascular disease is the leading cause of death all over the world. Its etiopathogenesis involves many correlated processes, with hypercholesterolemia being one of the main risk factors. Several large clinical trials have established the association between low-density lipoprotein cholesterol (LDL-C) levels and cardiovascular events. With the aim to take control over high LDL-C levels, several drugs with different targets in the cholesterol pathway have been developed. Statins a...
Source: Drugs of Today - May 28, 2019 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research

BIO-Europe Spring 2019 - 13th Annual International Partnering Conference (March 25-27, 2019 - Vienna, Austria).
This report covers highlights from the meeting. PMID: 31131844 [PubMed - in process] (Source: Drugs of Today)
Source: Drugs of Today - May 28, 2019 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research

Dacomitinib, a second-generation irreversible epidermal growth factor receptor tyrosine kinase inhibitor (EGFR-TKI) to treat non-small cell lung cancer.
Authors: Nagano T, Tachihara M, Nishimura Y Abstract Dacomitinib (PF-00299804, Vizimpro) was developed as a second-generation, oral, irreversible inhibitor of human epidermal growth factor receptor (EGFR)- 1, -2 and -4 tyrosine kinase. On September 27, 2018, the United States Food and Drug Administration (FDA) approved dacomitinib for the first-line treatment of patients with metastatic non-small cell lung cancer (NSCLC) with EGFR exon 19 deletion or exon 21 L858R substitution mutations. On January 8, 2019, the Ministry of Health, Labour and Welfare of Japan approved this second-generation EGFR tyrosine kinase inhi...
Source: Drugs of Today - May 7, 2019 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research

Elagolix sodium for the treatment of women with moderate to severe endometriosis-associated pain.
Authors: Barra F, Scala C, Ferrero S Abstract First-line medical therapies for treating pain associated with endometriosis mainly consist in combined oral contraceptives and progestins. However, some women, having persistence of symptoms, may require further therapeutic options. Among these, gonadotropin-releasing hormone (GnRH) agonists (GnRH-as) have been widely employed in the last 30 years, despite being characterized by an unfavorable safety profile. Currently, new alternative investigational options are being investigated to treat this benign chronic disease. GnRH antagonists (GnRH-ants) are innovative hormon...
Source: Drugs of Today - May 7, 2019 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research

Encorafenib and binimetinib for the treatment of BRAF V600E/K-mutated melanoma.
Authors: Rose AAN Abstract BRAF is a constituent of the mitogen-activated protein kinase (MAPK) signaling pathway, which serves to activate downstream MEK, and is one of the most commonly mutated oncogenes in human tumors. Indeed, BRAF V600 mutations are present in approximately 40% of metastatic melanoma tumors. Encorafenib (LGX-818, Braftovi) and binimetinib (MEK-162, Mektovi) are small-molecule inhibitors of BRAF and MEK, respectively. BRAF and MEK inhibitors have been shown to improve overall and progression-free survival among patients with metastatic melanoma. Of these inhibitors, encorafenib and binimetinib ...
Source: Drugs of Today - May 7, 2019 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research

Fremanezumab for the prevention of chronic and episodic migraine.
Authors: Lionetto L, Cipolla F, Guglielmetti M, Martelletti P Abstract On September 15, 2018, the U.S. Food and Drug Administration (FDA) approved subcutaneous fremanezumab, a calcitonin gene-related peptide (CGRP) monoclonal antibody, for the treatment of episodic and chronic migraine in adults, with two recommended dosages: 225 mg monthly or 675 mg every 3 months. On March 28, 2019, the European Commission granted fremanezumab Marketing Authorization in the E.U. for the same indication. In this monograph we review data on the pharmacokinetics, metabolism and safety of fremanezumab as reported in the scientific li...
Source: Drugs of Today - May 7, 2019 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research

Unintended consequences of the new ACC/AHA blood pressure treatment guidelines.
Authors: Chrysant SG, Chrysant GS Abstract Hypertension is a major risk factor for cardiovascular disease, heart failure, chronic kidney disease and stroke. Therefore, its early detection and treatment are very important according to blood pressure (BP) treatment guidelines issued by the various scientific societies. Over the years, BP treatment guidelines have changed from strict control of BP to more relaxed control, and lately to a strict BP control influenced by the results of the SPRINT trial. The recently published BP treatment guidelines by the American College of Cardiology/American Heart Association (ACC/A...
Source: Drugs of Today - May 7, 2019 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research

World Pharma Pricing and Market Access Congress - 11th Terrapinn Congress (March 19-20, 2019 - Amsterdam, the Netherlands).
Authors: Barkan J Abstract The 11th World Pharma Pricing and Market Access Congress 2019, organized by Terrapinn and held in Amsterdam, the Netherlands, brought together speakers and delegates from across the Globe, and enabled discussions surrounding market access, pricing, value and evidence generation. There was a focus on developments in rare diseases and advanced therapies, with emphasis on the growing importance of digital health and big data to generate results and manage real-world evidence (RWE). The conference was divided into both keynote plenaries and six parallel-running tracks, with deliberations conc...
Source: Drugs of Today - May 7, 2019 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research

Ertugliflozin for the treatment of type 2 diabetes.
Authors: Sharma R, Razdan K, Kuhad A, Kuhad A Abstract Sodium/glucose cotransporter 2 (SGLT2) is exclusively expressed in the S1 and S2 segments of proximal convoluted tubules and accounts for roughly 90% of glucose reabsorption. Ertugliflozin, a highly selective and reversible SGLT2 inhibitor, is the latest addition to the gliflozin class of SGLT2 inhibitors for the treatment of type 2 diabetes mellitus (T2DM). It was granted approval by the U.S. Food and Drug Administration (FDA) in December 2017 for treatment of T2DM as a monotherapy, and as part of two separate fixed-dose combination therapies with sitagliptin ...
Source: Drugs of Today - April 4, 2019 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research

Cannabidiol as adjunctive treatment of seizures associated with Lennox-Gastaut syndrome and Dravet syndrome.
This article provides a critical review of the pharmacology of CBD and the most recent clinical studies that evaluated its efficacy and safety as adjunctive treatment of seizures associated with LGS and DS. PMID: 30938373 [PubMed - in process] (Source: Drugs of Today)
Source: Drugs of Today - April 4, 2019 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research

Dual versus triple antithrombotic therapy: is there a role for direct oral anticoagulants in arterial thrombosis?
Authors: Garcia-Ropero A, Santos-Gallego CG, Zafar MU, Badimon JJ Abstract The number of patients receiving dual antiplatelet therapy with an additional indication for long-term oral anticoagulation has substantially increased over time. This population is facing an unacceptable risk of bleeding events, particularly among elderly individuals, who are especially vulnerable to complications. Further strategies to minimize this bleeding risk, including various drug combinations, different dosage regimens and even numerous attempts to find the appropriate duration of the treatment, have been evaluated in a multitude of...
Source: Drugs of Today - April 4, 2019 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research

Management of multidrug-resistant tuberculosis in the 21st century.
Authors: Kaul G, Kapoor E, Dasgupta A, Chopra S Abstract The spread of drug-resistant tuberculosis (TB) continues to be a global health crisis with increasing cases of multidrug-resistant TB (MDR-TB) being diagnosed worldwide. The identification and clinical management of MDR-TB is associated with additional problems related to resistance, diagnosis and treatment as compared to drug-susceptible TB. Treatment of MDR-TB usually involves therapy with long regimens of second-line drugs up to 24 months resulting in higher risks of serious adverse effects, lack of compliance and heightened economic burden on the patients...
Source: Drugs of Today - April 4, 2019 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research

Darvadstrocel for the treatment of patients with perianal fistulas in Crohn's disease.
Authors: Kotze PG, Spinelli A, Warusavitarne J, Di Candido F, Sahnan K, Adegbola SO, Danese S Abstract Despite significant advances in medical and surgical therapy for perianal fistulas in Crohn's disease (CD), treatment results are still modest, and a specific need for more effective therapies is a reality. Darvadstrocel is composed of expanded human allogeneic mesenchymal adult stem cells extracted from adipose tissue and constitutes the first stem cell therapy for perianal fistulizing CD to receive approval from the European Medicines Agency (EMA). This therapy is injected in both internal and external openings,...
Source: Drugs of Today - March 1, 2019 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research

Lorlatinib for the treatment of patients with non-small cell lung cancer.
Authors: Akamine T, Toyokawa G, Tagawa T, Yamazaki K, Seto T, Takeo S, Mori M Abstract Lorlatinib is a novel third-generation tyrosine kinase inhibitor (TKI) which targets anaplastic lymphoma kinase (ALK) as well as receptor tyrosine kinase c-ros oncogene 1 (ROS1). A critical limitation of conventional ALK/ROS TKIs is their association with acquired resistance mutations (particularly ALK G1202R and ROS1 G2032R) in the ALK or ROS1 gene, although these are not the only resistance mechanisms. Another limitation of this class of drugs is their inadequate efficacy against central nervous system metastasis, likely attrib...
Source: Drugs of Today - March 1, 2019 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research

Abscopal effects observed in cancer radiation therapy and oncolytic virotherapy: an overview.
Authors: Romano G, Marino IR Abstract Immunoglobulin-mediated suppression of immune checkpoint pathways may lead to a considerable activation of host immune responses against malignancies. Substantial therapeutic benefits were reported among patients who participated in cancer immunotherapy clinical trials which utilized monoclonal antibodies against cytotoxic T-lymphocyte-associated antigen 4 (CTLA-4), programmed cell death protein 1 (PD-1) and programmed cell death 1 ligand 1 (PD-L1). In a subsequent stage, immune checkpoint inhibitors were used in various clinical trials in combination with other therapeutic age...
Source: Drugs of Today - March 1, 2019 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research

The year's new drugs and biologics 2018: Part II - News that shaped the industry in 2018.
Authors: Graul AI, Dulsat C, Pina P, Cruces E, Tracy M Abstract This eagle's-eye overview of the drug industry in 2018 provides insight into some of last year's top stories, including a large and still growing outbreak of Ebola in the Democratic Republic of the Congo, as well as the potential uses (and abuses) of CRISPR technology. As in previous years, we also review orphan drug development, new regulatory agency-supported programs such as Priority Medicines Scheme (PRIME) and Sakigake, pipeline attrition, and pharma/biotech mergers and acquisitions of note. Finally, we take a glimpse into the crystal ball to anti...
Source: Drugs of Today - March 1, 2019 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research

Abiraterone acetate to treat metastatic castration-resistant prostate cancer in combination with prednisone.
Authors: Duarte C, Jimeno A, Kessler ER Abstract Prostate cancer is one of the most common cancers in the United States, with an estimated incidence of 164,690 cases, accounting for 9.5% of all new cancer diagnoses. The mainstay of therapy for metastatic prostate cancer involves suppressing testosterone production through androgen deprivation therapy. However, nearly all patients on androgen deprivation therapy will develop resistance to hormone therapy. An improved understanding of the biology of castration resistance has allowed for the development of novel inhibitors of the androgen axis. Agents such as abirater...
Source: Drugs of Today - February 12, 2019 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research

Condoliase for treatment of lumbar disc herniation.
Authors: Matsuyama Y, Chiba K Abstract Lumbar disc herniation (LDH) is generally treated with a conservative therapy, and surgery is the only therapeutic option currently available for patients unresponsive to the conservative therapy. In the 1980s, chemonucleolysis with chymopapain, a protease, was widely used as the intermediate treatment between conservative therapy and surgical therapy in the Western countries. However, since chymopapain was withdrawn from the market in 2002 for non-scientific commercial reasons, chemonucleolysis has not been a therapeutic option for LDH. Condoliase (chondroitin sulfate ABC end...
Source: Drugs of Today - February 12, 2019 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research

Ibalizumab for the treatment of multidrug-resistant HIV-1 infection.
Authors: Rizza SA, Bhatia R, Zeuli J, Temesgen Z Abstract Ibalizumab, a humanized monoclonal antibody to CD4, was recently approved by the United States Food and Drug Administration (FDA) for the treatment of HIV-1 infection in heavily treatment-experienced adults with multidrug-resistant HIV-1 infection failing their current antiretroviral regimen. Ibalizumab is the first in a new class of antiretroviral drugs designated as post-attachment inhibitors. It exerts its antiviral effect by noncompetitive binding of CD4, thereby blocking conformational changes in the CD4-gp120 complex that are essential for viral entry....
Source: Drugs of Today - February 12, 2019 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research

The year's new drugs and biologics 2018: Part I.
Authors: Graul AI, Pina P, Cruces E, Stringer M Abstract Sixty-four new drugs and biologics reached their first global markets in 2018, the greatest number in at least a decade. In addition, 19 important new line extensions (new indications, new combinations or new formulations of previously marketed drugs) were introduced over the course of 2018. Twenty-three other new products and new line extensions were approved but not launched before December 31. As has been the trend in recent years, oncology was the most active therapeutic area in terms of new launches, with cancer drugs accounting for 25% of all novel drug...
Source: Drugs of Today - February 12, 2019 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research

Lumateperone: a new treatment approach for neuropsychiatric disorders.
Authors: Kumar B, Kuhad A, Kuhad A Abstract Lumateperone tosylate (ITI-007 tosylate, ITI-722) is a first-in-class investigational drug which acts syn-ergistically through multiple systems (serotonergic, dopaminergic and glutamatergic), thus representing a unique approach for the therapeutic management of a range of neuropsychiatric disorders. It possesses a potent antagonistic activity at 5-hydroxytryptamine (serotonin) 2A (5-HT2A) receptors and also binds to dopamine (D1, D2) receptors with partial agonism at presynaptic D2 receptors and postsynaptic antagonism. Further, preclinical data demonstrated that lumatepe...
Source: Drugs of Today - January 2, 2019 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research

Volanesorsen for treatment of patients with familial chylomicronemia syndrome.
Authors: Warden BA, Duell PB Abstract Familial chylomicronemia syndrome (FCS) is a rare autosomal recessive disorder typically caused by mutations in genes for lipoprotein lipase (LPL), apolipoprotein C-II (Apo-CII), apolipoprotein A-V (Apo-AV), lipase maturation factor 1 (LMF1) and glycosylphosphatidylinositol-anchored high-density lipoprotein-binding protein 1 (GPI-HBP1). FCS is associated with severe morbidity that includes recurrent pancreatitis and other problems. Effective treatment to reliably prevent complications has been unavailable, so there is a quest to identify novel interventions to achieve sustained...
Source: Drugs of Today - January 2, 2019 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research

β-Lactamase inhibitor peptides as the new strategies to overcome bacterial resistance.
β-Lactamase inhibitor peptides as the new strategies to overcome bacterial resistance. Drugs Today (Barc). 2018 Dec;54(12):737-746 Authors: Silva ON, Franco OL, Porto WF Abstract Bacterial resistance has become a problem of great concern all over the world. Gram-negative bacteria, including the Enterobacteriaceae family and Pseudomonas and Acinetobacter species, are among the leading causes of healthcare-associated infections. The rate of antibiotic resistance among these pathogens has increased dramatically in recent years, reaching a pandemic scale. The most common mechanism of resistance descri...
Source: Drugs of Today - January 2, 2019 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research

The Financial Times Global Pharmaceutical and Biotechnology Conference. Embracing Disruption for a New Era in Health (November 5-6, 2018 - London, UK.
Authors: Kibble A Abstract The Financial Times (FT) Global Healthcare conference brought together top-level executives and leaders in global healthcare and technology around a fascinating and thought-provoking agenda. Consisting of a wide range of expert panel discussions chaired by the FT and Deloitte's specialist editors, as well as a series of keynote CEO interviews, the conference took an in-depth and nuanced view of the forces of disruption colliding in healthcare at the moment. Gains in R+D are implicit, but beyond this, technology and data disruption can potentially transform all aspects of healthcare throug...
Source: Drugs of Today - January 2, 2019 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research

Avatrombopag maleate for the treatment of periprocedural thrombocytopenia in patients with chronic liver disease.
Authors: Al-Samkari H Abstract Avatrombopag maleate (Doptelet; Dova Pharmaceuticals) is an oral, small-molecule second-generation thrombopoietin (TPO) receptor agonist under development for the treatment of thrombocytopenia. Recently approved by the U.S. Food and Drug Administration (FDA) for the treatment of periprocedural thrombocytopenia in patients with chronic liver disease on the basis of the results of two phase III trials, it is currently under investigation for treatment of immune thrombocytopenia (two randomized trials demonstrating efficacy have already been completed), chemotherapy-induced thrombocytope...
Source: Drugs of Today - December 14, 2018 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research

Delafloxacin meglumine for the treatment of acute bacterial skin and skin structure infections (ABSSSI).
Authors: Kaul G, Kapoor E, Dasgupta A, Chopra S Abstract Delafloxacin meglumine (Baxdela, WQ-3034, ABT-492, RX-3341; Melinta Therapeutics) was approved by the U.S. Food and Drug Administration (FDA) in June 2017 for the treatment of acute bacterial skin and skin structure infections on the basis of data from two phase III trials. Delafloxacin is a broad-spectrum anionic fluoroquinolone and its distinct chemical structure increases its potency in acidic environments. It is known to inhibit DNA replication and repair by targeting DNA gyrase and topoisomerase IV. Delafloxacin is administered via both oral and parenter...
Source: Drugs of Today - December 14, 2018 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research

Estradiol vaginal inserts (Imvexxy): effective in genitourinary syndrome of menopause without increasing systemic estrogen levels.
Authors: Paton DM Abstract Many women suffer from the genitourinary syndrome of the menopause (GSM), in particular pathological changes that occur in the vagina due to the reduction in their production of estrogens. TherapeuticsMD has developed Imvexxy, small softgel capsules that contain solubilized estradiol and that are inserted vaginally by women twice a week, every 3 to 4 days. These vaginal inserts have been found to readily supply estradiol to the vagina and to result in significant improvement in the GSM signs and symptoms these women had been experiencing. At the same time, pharmacokinetic studies have dem...
Source: Drugs of Today - December 14, 2018 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research

An update on sargramostim for treatment of acute radiation syndrome.
Authors: Singh VK, Seed TM Abstract The potential use by terrorists of an improvised nuclear device, a radiological dispersal device, or an unintended nuclear/radiological accident in heavily populated areas is a national security threat of major consequences. Although this type of security threat is considered to be low-risk, it would have a devastating impact. Health issues would be a major concern; medical care would be necessary for all those who received considerable radiation exposure (> 1 Gy) leading to hematopoietic acute radiation syndrome (ARS). In the past few years, the U.S. Food and Drug Administrat...
Source: Drugs of Today - December 14, 2018 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research

New therapies for anaplastic thyroid cancer.
Authors: Agrawal VR, Hreno J, Patil T, Bowles DW Abstract Anaplastic thyroid cancer is a rare but extremely aggressive type of thyroid cancer. Treatment typically consists of surgery, external beam radiotherapy and cytotoxic chemotherapy. However, available literature suggests only modest survival benefit for cytotoxic chemotherapy. Recent advances have suggested the combination of BRAF and MEK inhibition may have a profound and durable effect on patients with BRAFV600E-mutated anaplastic thyroid cancer, with a response rate of 69%. Other systemic treatments, including immunotherapies, have also shown promising but...
Source: Drugs of Today - December 14, 2018 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research

Apalutamide for the treatment of patients with castration-resistant prostate cancer.
Authors: Hauke R Abstract Patients with recurrent, nonmetastatic prostate cancer after curative intent therapy can experience a heterogeneous clinical course ranging from indolent disease that can be observed for years to a rapidly progressive disease that is metastatic in a relatively short time. Patients with short prostate-specific antigen (PSA) doubling times are at risk for early development of metastatic disease and are frequently placed on androgen deprivation therapy. Although castration-resistant disease inevitably occurs in these patients, most therapies for castration-resistant disease have shown benefit...
Source: Drugs of Today - November 7, 2018 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research

Emicizumab for routine prophylaxis to prevent bleeding episodes in patients with hemophilia A.
Authors: Díaz-Ricart M, Isola IM, Escolar G Abstract Hemophilia A is an X-linked bleeding disorder caused by defects in the gene encoding factor VIII (FVIII). Routine prophylaxis with exogenous FVIII requires frequent intravenous injections. One of the most challenging issues in the treatment of hemophilia A is the development of alloantibodies against infused FVIII. Presence of inhibitors results in an ineffective factor replacement therapy and increases the risk of morbidity and mortality in these patients. Therefore, there is growing interest in the development of new strategies for the prophylaxis and pr...
Source: Drugs of Today - November 7, 2018 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research

Sodium zirconium cyclosilicate: a new potassium binder for the treatment of hyperkalemia.
Authors: Cases A, Gorriz JL Abstract Hyperkalemia is one of the most common electrolyte disturbances, especially among some groups of patients, such as in those with chronic kidney disease, diabetes or heart failure. Hyperkalemia has been associated with increased risks of mortality, arrhythmias, hospitalization and costs, as well as the need to down titrate/discontinue renin-angiotensin-aldosterone system inhibitors (RAASIs), despite their well-known cardiovascular and nephroprotective benefits. Current potassium binders have limitations (slow onset of action, limited selectivity for potassium binding, risk of dru...
Source: Drugs of Today - November 7, 2018 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research

An update of treatments of hepatocellular carcinoma in patients refractory to sorafenib.
Authors: Ielasi L, Sansone V, Granito A, Benevento F, De Lorenzo S, Tovoli F Abstract Hepatocellular carcinoma (HCC) remains among the neoplastic diseases with the most unfavorable prognosis. Historically, systemic treatments for HCC have been scarce. In particular, sorafenib was the only registered drug for the treatment of unresectable HCC until 2017. Last year, regorafenib was approved by the global regulatory agencies as second-line therapy. Since then, further randomized, controlled trials have been successful in this patient population. This paper deals with the most recent data concerning cabozantinib and ra...
Source: Drugs of Today - November 7, 2018 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research