Topical Collection "The Story of Successful Drugs and Recent FDA-Approved Molecules"

Editors

Dr. Jean Jacques Vanden Eynde
Website1 Website2
Collection Editor
Formerly head of the Department of Organic Chemistry (FS), University of Mons-UMONS, 7000 Mons, Belgium
Interests: heterocycles; microwave-induced synthesis; medicinal chemistry; green chemistry
Special Issues and Collections in MDPI journals
Dr. Klaus Kopka
Website
Collection Editor
Helmholtz-Zentrum Dresden-Rossendorf (HZDR) e.V., Institute of Radiopharmaceutical Cancer Research, 01328 Dresden, Germany
Interests: radiopharmaceutical sciences; labeling chemistry; medicinal chemistry; PET tracers; radiopharmaceuticals for diagnostics and endoradiotherapy; small molecules; peptides; antibodies and derivatives thereof
Special Issues and Collections in MDPI journals
Prof. Dr. Maria Emília de Sousa
Website
Collection Editor
1. Laboratório de Química Orgânica e Farmacêutica, Departamento de Ciências, Químicas, Faculdade de Farmácia, Universidade do Porto, Rua Jorge Viterbo Ferreira 228, 4050-313, Porto, Portugal
2. Interdisciplinar de Investigação Marinha e Ambiental (CIIMAR/CIMAR), Universidade do Porto, Terminal de Cruzeiros do Porto de Leixões, Avenida General Norton de Matos, S/N 4450-208 Matosinhos, Portugal
Interests: medicinal chemistry; organic synthesis; heterocycles, P-glycoprotein; anticancer; anticoagulants; chiral drugs; marine natural products
Special Issues and Collections in MDPI journals
Dr. Annie Mayence
Website
Collection Editor
Formerly professor at the Haute Ecole Provinciale de Hainaut-Condorcet, 7330 Saint-Ghislain, Belgium
Interests: medicinal chemistry; organic synthesis; parasitic diseases; orphan drugs
Special Issues and Collections in MDPI journals
Prof. Dr. Joachim Jose
Website
Collection Editor
PharmaCampus Institute of Pharmaceutical and Medicinal Chemistry, Westfälische Wilhelms-Universität, Corrensstr. 48, 48149, Muenster, Germany
Interests: autodisplay; assay development and inhibitor testing; whole cell biocatalysts for synthesis of drugs and building blocks; directed evolution of enzyme inhibitors and biocatalysts; biosensor development and diagnostic tools
Special Issues and Collections in MDPI journals
Dr. Guangshun Wang
Website
Collection Editor
Department of Pathology & Microbiology, University of Nebraska Medical Center, Omaha, NE 68198-5900, USA
Interests: host defense antimicrobial peptides; structural bioinformatics; biomolecular NMR
Special Issues and Collections in MDPI journals

Topical Collection Information

Dear Colleagues,

In 2018, the FDA approved 59 novel drugs, a record high, demonstrating the vigor of pharmaceutical industry and associated research groups. Small molecules were the most represented entities among the approvals, but there is an increasing number of antibodies and oligonucleotides on the medical market. Interestingly, there is a growing interest in the development of orphan drugs and the treatment of orphan diseases.

The aim of this Special Issue is to offer to our readers short but comprehensive and updated reviews on compounds recently approved by the FDA and drugs attracting particular attention because of their success.

Dr. Jean Jacques Vanden Eynde
Prof. Dr. Klaus Kopka
Prof. Dr. Maria Emília De Sousa
Dr. Annie Mayence
Prof. Dr. Joachim Jose
Dr. Guangshun Wang
Guest Editors

Manuscript Submission Information

Manuscripts should be submitted online at www.mdpi.com by registering and logging in to this website. Once you are registered, click here to go to the submission form. Manuscripts can be submitted until the deadline. All papers will be peer-reviewed. Accepted papers will be published continuously in the journal (as soon as accepted) and will be listed together on the collection website. Research articles, review articles as well as short communications are invited. For planned papers, a title and short abstract (about 100 words) can be sent to the Editorial Office for announcement on this website.

Submitted manuscripts should not have been published previously, nor be under consideration for publication elsewhere (except conference proceedings papers). All manuscripts are thoroughly refereed through a single-blind peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. Pharmaceuticals is an international peer-reviewed open access monthly journal published by MDPI.

Please visit the Instructions for Authors page before submitting a manuscript. The Article Processing Charge (APC) for publication in this open access journal is 1000 CHF (Swiss Francs). Submitted papers should be well formatted and use good English. Authors may use MDPI's English editing service prior to publication or during author revisions.

Keywords

  • antibody
  • aptamer
  • oligonucleotide
  • orphan disease
  • orphan drug
  • peptide
  • small molecule

Published Papers (14 papers)

2020

Jump to: 2019

Open AccessBrief Report
Is There a Role for the Therapeutic Drug Monitoring of Colistin? An Overview
Pharmaceuticals 2020, 13(3), 42; https://doi.org/10.3390/ph13030042 - 06 Mar 2020
Abstract
Colistin is used as a last-line antibiotic for the treatment of Gram-negative multiresistant bacteria. Due to its high nephrotoxicity, Therapeutic Drug Monitoring (TDM) is recommended for dose adjustment. We aimed to evaluate the available evidence of TDM in patients given colistin to treat [...] Read more.
Colistin is used as a last-line antibiotic for the treatment of Gram-negative multiresistant bacteria. Due to its high nephrotoxicity, Therapeutic Drug Monitoring (TDM) is recommended for dose adjustment. We aimed to evaluate the available evidence of TDM in patients given colistin to treat Gram-negative infections. In this paper, we offer an overview, using an electronic search of the literature (published up to June 2019, without language restrictions) that compares the clinical outcomes and measurements of colistin TDM. Ultimately, the Therapeutic Drug Monitoring (TDM) of colistin in Plasma could prevent nephrotoxicity risk. Full article
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Open AccessReview
2019 FDA TIDES (Peptides and Oligonucleotides) Harvest
Pharmaceuticals 2020, 13(3), 40; https://doi.org/10.3390/ph13030040 - 05 Mar 2020
Cited by 3
Abstract
2019 has been an excellent year in terms of peptides and oligonucleotides (TIDES) approved by the FDA. Despite the drop in the number of total drugs approved by the FDA in 2019 in comparison with 2018 (48 vs. 59), the total number of [...] Read more.
2019 has been an excellent year in terms of peptides and oligonucleotides (TIDES) approved by the FDA. Despite the drop in the number of total drugs approved by the FDA in 2019 in comparison with 2018 (48 vs. 59), the total number of TIDES authorized increased (seven vs. three). Year after year, TIDES are increasingly present in therapy, as imaging agents, theragnostic and constituent moieties of other complex drugs, such as antibody drug conjugates. This means a consolidation of these kinds of drugs in the pharmaceutical arena, paving the way in the coming years for the approval of others for diverse medical indications. Here the TIDES approved in 2019 are analyzed in terms of chemical structure, medical target, mode of action, and adverse effects. Full article
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Open AccessReview
[68Ga]Ga-DOTA-TOC: The First FDA-Approved 68Ga-Radiopharmaceutical for PET Imaging
Pharmaceuticals 2020, 13(3), 38; https://doi.org/10.3390/ph13030038 - 03 Mar 2020
Cited by 1
Abstract
In the United States, [68Ga]Ga-DOTA-TOC has been approved by the Food and Drug Administration (FDA) in 2019 as the first 68Ga-radiopharmaceutical for imaging of somatostatin receptor (SSTR) positive gastroenteropancreatic neuroendocrine tumors while employing positron emission tomography (PET). In Europe (Austria, [...] Read more.
In the United States, [68Ga]Ga-DOTA-TOC has been approved by the Food and Drug Administration (FDA) in 2019 as the first 68Ga-radiopharmaceutical for imaging of somatostatin receptor (SSTR) positive gastroenteropancreatic neuroendocrine tumors while employing positron emission tomography (PET). In Europe (Austria, Germany, France), [68Ga]Ga-DOTA-TOC was already approved back in 2016. This radiopharmaceutical combines the radionuclide 68Ga with the somatostatin analogue DOTA-TOC for specific imaging of tumor cells expressing SSTRs. Such a targeting approach can also be used for therapy planning in the case of both localized as well as disseminated disease and potentially for the evaluation of treatment response. Full article
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2019

Jump to: 2020

Open AccessArticle
Application of Thin-Layer Chromatography in Combination with Densitometry for the Determination of Diclofenac in Enteric Coated Tablets
Pharmaceuticals 2019, 12(4), 183; https://doi.org/10.3390/ph12040183 - 16 Dec 2019
Cited by 2
Abstract
Diclofenac belongs to the drug class non-steroidal anti-inflammatory drugs widely used in Europe as well as all over the world. Thus, it is important to conduct research on its quality control of available pharmaceutical preparations like for example enteric coated tablets. Among various [...] Read more.
Diclofenac belongs to the drug class non-steroidal anti-inflammatory drugs widely used in Europe as well as all over the world. Thus, it is important to conduct research on its quality control of available pharmaceutical preparations like for example enteric coated tablets. Among various analytical techniques, thin-layer chromatography (TLC) is ideal for this task due to their short time analysis, ease of operation and low cost. Hence, the aim of this study was to develop the optimal conditions of analysis and quantitative determination of diclofenac sodium in enteric tablets by using TLC in combination with densitometry. Of all chromatographic systems tested, the best is the one which consists of silica gel 60F254 and cyclohexane: chloroform:methanol:glacial acetic acid (6:3:0.5:0.5 v/v) as the mobile phase, which allows the successful separation of examined diclofenac sodium as active component and the largest number (twelve) of its degradation products as potential impurities of its pharmaceutical products. This indicates that the newly developed method is more effective than previously reported assays by Starek and Krzek. Linearity range was found to be 4.00–18.00 μg/spot for diclofenac sodium. The results of the assay of enteric tablet formulations equals 98.8% of diclofenac sodium in relation to label claim is in a good agreement with pharmaceutical requirements. Full article
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Open AccessArticle
Metabolism and Pharmacokinetic Study of the Boron-Containing Prodrug of Belinostat (ZL277), a Pan HDAC Inhibitor with Enhanced Bioavailability
Pharmaceuticals 2019, 12(4), 180; https://doi.org/10.3390/ph12040180 - 08 Dec 2019
Cited by 1
Abstract
ZL277 is a prodrug of belinostat with enhanced bioavailability and efficacy as a pan histone deacetylase (HDAC) inhibitor. In this study, we investigated the metabolism and pharmacokinetics of ZL277 in liver S9 fractions, liver microsomes, liver cytosol, and in mice. Metabolic products were [...] Read more.
ZL277 is a prodrug of belinostat with enhanced bioavailability and efficacy as a pan histone deacetylase (HDAC) inhibitor. In this study, we investigated the metabolism and pharmacokinetics of ZL277 in liver S9 fractions, liver microsomes, liver cytosol, and in mice. Metabolic products were identified and quantified by a combination of liquid chromatography and tandem mass spectrometry. The in vitro metabolic profile of ZL277 includes ZL277-B(OH)2-452, the major oxidative metabolite ZL277-OH-424, the active ingredient belinostat, belinostat amide, belinostat acid, and methylated belinostat in liver S9 fractions. Both ZL277-OH-424 and belinostat underwent further glucuronidation in liver microsome, whereas only ZL277-OH-424, but not belinostat, underwent some level of sulfation in rat liver cytosols. These metabolites were examined in plasma and in a breast tumor model in vivo. They were also examined in urine and feces from mice treated with ZL277. The pharmacokinetic study of ZL277 showed the parameters of active drug belinostat with a half-life (t1/2) of 10.7 h, an area under curve value (AUC) of 1506.9 ng/mL*h, and a maximum plasma concentration (Cmax) of 172 ng/mL, reached 3 h after a single dose of 10 mg/kg. The hydrolysis product of the prodrug, ZL277-B(OH)2-452 showed an AUC of 8306 ng/mL*h and Cmax of 931 ng/mL 3 h after drug administration. Full article
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Open AccessBrief Report
Tafenoquine: A 2018 Novel FDA-Approved Prodrug for the Radical Cure of Plasmodium vivax Malaria and Prophylaxis of Malaria
Pharmaceuticals 2019, 12(3), 115; https://doi.org/10.3390/ph12030115 - 30 Jul 2019
Abstract
Tafenoquine (an 8-aminoquinoline) was approved by the Food and Drug Administration (FDA) in 2018 for the radical cure of Plasmodium vivax malaria and preventive action against malaria. Despite the fact that the mechanism of action of the drug remains unclear, all studies indicated [...] Read more.
Tafenoquine (an 8-aminoquinoline) was approved by the Food and Drug Administration (FDA) in 2018 for the radical cure of Plasmodium vivax malaria and preventive action against malaria. Despite the fact that the mechanism of action of the drug remains unclear, all studies indicated that a metabolite is responsible for its efficacy. Routes for the preparation of the drug are described. Full article
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Open AccessReview
Lutathera®: The First FDA- and EMA-Approved Radiopharmaceutical for Peptide Receptor Radionuclide Therapy
Pharmaceuticals 2019, 12(3), 114; https://doi.org/10.3390/ph12030114 - 29 Jul 2019
Cited by 10
Abstract
As the first radiopharmaceutical for Peptide Receptor Radionuclide Therapy (PRRT), Lutathera® was approved by the EMA in 2017 and the FDA in 2018 for the treatment of somatostatin receptor (SSTR) positive gastroenteropancreatic neuroendocrine tumors. Using the concept of PRRT, Lutathera® combines [...] Read more.
As the first radiopharmaceutical for Peptide Receptor Radionuclide Therapy (PRRT), Lutathera® was approved by the EMA in 2017 and the FDA in 2018 for the treatment of somatostatin receptor (SSTR) positive gastroenteropancreatic neuroendocrine tumors. Using the concept of PRRT, Lutathera® combines the radionuclide 177Lu with the somatostatin analogue DOTA-TATE, thus delivering ionizing radiation specifically to tumor cells expressing somatostatin receptors. As a result, DNA single- and double-strand breaks are provoked, in case of double-strand breaks leading to cell death of the tumor and its SSTR-positive lesions. Full article
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Open AccessReview
Psychotropic Drugs for the Management of Chronic Pain and Itch
Pharmaceuticals 2019, 12(2), 99; https://doi.org/10.3390/ph12020099 - 24 Jun 2019
Cited by 3
Abstract
Clinical observations have shown that patients with chronic neuropathic pain or itch exhibit symptoms of increased anxiety, depression and cognitive impairment. Such patients need corrective therapy with antidepressants, antipsychotics or anticonvulsants. It is known that some psychotropic drugs are also effective for the [...] Read more.
Clinical observations have shown that patients with chronic neuropathic pain or itch exhibit symptoms of increased anxiety, depression and cognitive impairment. Such patients need corrective therapy with antidepressants, antipsychotics or anticonvulsants. It is known that some psychotropic drugs are also effective for the treatment of neuropathic pain and pruritus syndromes due to interaction with the secondary molecular targets. Our own clinical studies have identified antipruritic and/or analgesic efficacy of the following compounds: tianeptine (atypical tricyclic antidepressant), citalopram (selective serotonin reuptake inhibitor), mianserin (tetracyclic antidepressant), carbamazepine (anticonvulsant), trazodone (serotonin antagonist and reuptake inhibitor), and chlorprothixene (antipsychotic). Venlafaxine (serotonin-norepinephrine reuptake inhibitor) is known to have an analgesic effect too. The mechanism of such effect of these drugs is not fully understood. Herein we review and correlate the literature data on analgesic/antipruritic activity with pharmacological profile of these compounds. Full article
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Open AccessBrief Report
Tegsedi (Inotersen): An Antisense Oligonucleotide Approved for the Treatment of Adult Patients with Hereditary Transthyretin Amyloidosis
Pharmaceuticals 2019, 12(2), 78; https://doi.org/10.3390/ph12020078 - 21 May 2019
Cited by 3
Abstract
Tegsedi (Inotersen) is a chemically modified antisense oligonucleotide that inhibits the hepatic production of transthyretin (TTR). Several single-point mutations in TTR destabilize its structure, leading to the aggregation and accumulation of amyloid deposits in the nervous system, heart, kidneys and eyes. In July [...] Read more.
Tegsedi (Inotersen) is a chemically modified antisense oligonucleotide that inhibits the hepatic production of transthyretin (TTR). Several single-point mutations in TTR destabilize its structure, leading to the aggregation and accumulation of amyloid deposits in the nervous system, heart, kidneys and eyes. In July 2018, Tegsedi was approved by the European Commission for use in adults with stage one and two polyneuropathies. Later on, in October 2018, the FDA and Health Canada also approved its use for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR) in adults in the U.S. and Canada. Tegsedi was developed by Ionis Pharmaceuticals, the company that holds the global marketing license, together with its subsidiary Akcea Therapeutics. Full article
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Open AccessBrief Report
Duvelisib: A 2018 Novel FDA-Approved Small Molecule Inhibiting Phosphoinositide 3-Kinases
Pharmaceuticals 2019, 12(2), 69; https://doi.org/10.3390/ph12020069 - 06 May 2019
Cited by 4
Abstract
Duvelisib (Copiktra®) is a dual inhibitor of phosphoinositide 3-kinases (PI3Kδ and PI3Kγ). In 2018, duvelisib was first approved by the Food and Drug Administration (FDA) for the treatment of adult patients with relapsed or refractory chronic lymphocytic leukaemia (CLL)/ small lymphocytic [...] Read more.
Duvelisib (Copiktra®) is a dual inhibitor of phosphoinositide 3-kinases (PI3Kδ and PI3Kγ). In 2018, duvelisib was first approved by the Food and Drug Administration (FDA) for the treatment of adult patients with relapsed or refractory chronic lymphocytic leukaemia (CLL)/ small lymphocytic lymphoma (SLL) after at least two prior therapies. Duvelisib has also been approved under accelerated track for relapsed or refractory follicular lymphoma (FL) after at least two prior systemic therapies. In this review, we provide a series of information about duvelisib, such as the development of clinical trials for LLC/SLL and FL and the steps used for its synthesis. Full article
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Open AccessBrief Report
Omadacycline: A Newly Approved Antibacterial from the Class of Tetracyclines
Pharmaceuticals 2019, 12(2), 63; https://doi.org/10.3390/ph12020063 - 21 Apr 2019
Cited by 5
Abstract
Omadacycline (Nuzyra®) is a new aminomethylcycline, approved by the U. S. Food and Drug Administration in 2018, as a tetracycline antibacterial. It can be used in community-acquired pneumonia and in acute bacterial skin and skin-structure infections. It was developed and is commercialized by [...] Read more.
Omadacycline (Nuzyra®) is a new aminomethylcycline, approved by the U. S. Food and Drug Administration in 2018, as a tetracycline antibacterial. It can be used in community-acquired pneumonia and in acute bacterial skin and skin-structure infections. It was developed and is commercialized by Paratek Pharmaceuticals. It is a semisynthetic compound, derived from minocycline, capable of evading widely distributed efflux and target protection antibacterial resistance mechanisms and has demonstrated activity in a broad spectrum of bacteria. Full article
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Open AccessReview
Long-Acting Anti-HIV Drugs Targeting HIV-1 Reverse Transcriptase and Integrase
Pharmaceuticals 2019, 12(2), 62; https://doi.org/10.3390/ph12020062 - 20 Apr 2019
Cited by 8
Abstract
One of the major factors contributing to HIV-1 drug resistance is suboptimal adherence to combination antiretroviral therapy (cART). Currently, recommended cART for HIV-1 treatment is a three-drug combination, whereas the pre-exposure prophylaxis (PrEP) regimens consist of one or two antivirals. Treatment regimens require [...] Read more.
One of the major factors contributing to HIV-1 drug resistance is suboptimal adherence to combination antiretroviral therapy (cART). Currently, recommended cART for HIV-1 treatment is a three-drug combination, whereas the pre-exposure prophylaxis (PrEP) regimens consist of one or two antivirals. Treatment regimens require adherence to a once or twice (in a subset of patients) daily dose. Long-acting formulations such as injections administered monthly could improve adherence and convenience, and thereby have potential to enhance the chances of expected outcomes, although long-lasting drug concentrations can also contribute to clinical issues like adverse events and development of drug resistance. Globally, two long-acting antivirals have been approved, and fifteen are in clinical trials. More than half of investigational long-acting antivirals target HIV-1 reverse transcriptase (HIV-1 RT) and/or integrase (HIV-1 IN). Here, we discuss the status and potential of long-acting inhibitors, including rilpivirine (RPV), dapivirine (DPV), and 4-ethynyl-2-fluoro-2-deoxyadenosine (EFdA; also known as MK-8591), which target RT, and cabotegravir (CAB), which targets IN. The outcomes of various clinical trials appear quite satisfactory, and the future of long-acting HIV-1 regimens appears bright. Full article
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Open AccessReview
2018 FDA Tides Harvest
Pharmaceuticals 2019, 12(2), 52; https://doi.org/10.3390/ph12020052 - 05 Apr 2019
Cited by 15
Abstract
In 2018, the United States Food and Drug Administration (FDA) approved a total of 59 new drugs, three of them (5%) are TIDES (or also, -tides), two oligonucleotides and one peptide. Herein, the three TIDES approved are analyzed in terms of medical target, [...] Read more.
In 2018, the United States Food and Drug Administration (FDA) approved a total of 59 new drugs, three of them (5%) are TIDES (or also, -tides), two oligonucleotides and one peptide. Herein, the three TIDES approved are analyzed in terms of medical target, mode of action, chemical structure, and economics. Full article
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Open AccessBrief Report
Baricitinib: A 2018 Novel FDA-Approved Small Molecule Inhibiting Janus Kinases
Pharmaceuticals 2019, 12(1), 37; https://doi.org/10.3390/ph12010037 - 12 Mar 2019
Cited by 1
Abstract
In 2018, Baricitinib was approved by the Food and Drig Administration (FDA) for the treatment of rheumatoid arthritis. Baricitinib exerts its action by targeting Janus kinases (JAK). In this study, we describe the necessary steps for preparing the drug using two alternative routes. [...] Read more.
In 2018, Baricitinib was approved by the Food and Drig Administration (FDA) for the treatment of rheumatoid arthritis. Baricitinib exerts its action by targeting Janus kinases (JAK). In this study, we describe the necessary steps for preparing the drug using two alternative routes. Full article
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