Opicapone: A Review in Parkinson's Disease.

Oral opicapone (Ongentys), a potent third-generation, peripheral catechol-O-methyltransferase (COMT) inhibitor, is approved as adjunctive therapy to preparations of levodopa/dopa-decarboxylase inhibitor (L-dopa/DDCI) in adults with Parkinson's disease (PD) and end-of dose (EoD) motor fluctuations. In pivotal global trials (BIPARK 1 and BIPARK 2; 14-15 weeks' duration), open-label extensions (OLEs) of BIPARK, and in the real-world setting (OPTIPARK; 3-6 months), opicapone 50 mg once daily was an effective and generally well tolerated adjunctive therapy to L-dopa/DDCI plus other PD therapy in adults with PD and EoD motor fluctuations. Adjunctive opicapone provided better efficacy than placebo for improvements in ON- and OFF-state time and fulfilled noninferiority to adjunctive entacapone for improvements in OFF time in BIPARK 1.

Lumasiran: First Approval.

Lumasiran (Oxlumo™) is a subcutaneously administered small interfering RNA (siRNA) targeting the mRNA for hydroxyacid oxidase 1 gene (HAO1; encodes glycolate oxidase) and was developed by Alnylam Pharmaceuticals for the treatment of primary hyperoxaluria type 1 (PH1). By silencing the gene encoding glycolate oxidase, lumasiran depletes glycolate oxidase and thereby inhibits the synthesis of oxalate, which is the toxic metabolite that is directly associated with the clinical manifestations of PH1. On 19 November 2020, lumasiran received its first global approval in the EU for the treatment of PH1 in all age groups.

Darolutamide: A Review in Non-Metastatic Castration-Resistant Prostate Cancer.

Oral darolutamide (Nubeqa™) is a novel second-generation, nonsteroidal, selective androgen receptor (AR) inhibitor indicated for the treatment of non-metastatic castration-resistant prostate cancer (nmCRPC). In the pivotal multinational, phase 3 ARAMIS trial in men with nmCRPC, relative to placebo plus ongoing androgen deprivation therapy (ADT), darolutamide (+ ADT) significantly prolonged metastasis-free survival (MFS) at the time of the primary analysis and overall survival (OS) at the time of the final OS analysis and was generally well tolerated in extended follow-up. Albeit long-term data from the real-world setting are required to fully define the safety profile of darolutamide, current evidence from the final ARAMIS analysis indicates that darolutamide has a low propensity for CNS-related adverse events (AEs) associated with other currently approved second-generation AR inhibitors.

Siponimod: A Review in Secondary Progressive Multiple Sclerosis.

Oral siponimod (Mayzent), a next-generation, selective sphingosine 1-phosphate receptor (S1PR) 1 and 5 modulator, is approved in several countries for the treatment of secondary progressive multiple sclerosis (SPMS), with specific indications varying between individual countries. In the pivotal EXPAND trial (median duration double-blind treatment 18 months) in a broad spectrum of patients with SPMS, once-daily oral siponimod 2 mg (initial dose titration over 6 days) was significantly more effective than placebo in reducing clinical and MRI-defined outcomes of disease activity and disability progression, including 3-month confirmed disability progression on the Expanded Disability Status Scale (EDSS), and was generally well tolerated in the core phase of the study. These beneficial effects of siponimod appeared to be sustained during up to 5 years of treatment in the ongoing open-label extension phase of EXPAND.

Correction to: Romosozumab: A Review in Postmenopausal Osteoporosis.

Unfortunately the sections were published incorrectly in the original article.

Rivaroxaban: A Review for Secondary CV Prevention in CAD and PAD.

Secondary cardiovascular (CV) prevention in patients with vascular disease [e.g. coronary (CAD) and peripheral (PAD) artery disease] is crucial and typically involves antiplatelet therapy with aspirin; however, managing residual ischaemic and bleeding risks in CV disease (CVD) remains a challenge.

Romosozumab: A Review in Postmenopausal Osteoporosis.

Romosozumab (Evenity), a humanized monoclonal antibody, promotes bone formation and inhibits bone resorption by inhibiting sclerostin, a protein involved in the regulation of bone formation. Subcutaneous romosozumab is approved in several countries, including those of the EU for treating severe osteoporosis as well as in the USA for osteoporosis in postmenopausal women at high risk of fracture. In pivotal phase III trials (FRAME and ARCH), 12 months' once-monthly romosozumab 210 mg significantly reduced vertebral and clinical fracture risk versus placebo and oral alendronate in postmenopausal women with osteoporosis.

Correction to: Galcanezumab: A Review in the Prevention of Migraine and Treatment of Episodic Cluster Headache.

The name of the first reviewer which previously read.

Voretigene Neparvovec: A Review in RPE65 Mutation-Associated Inherited Retinal Dystrophy.

Voretigene neparvovec (Luxturna), a recombinant adeno-associated virus vector-based gene therapy, delivers a functioning copy of the human retinal pigment epithelium-specific 65 kDa (RPE65) gene into retinal cells of patients with reduced or absent levels of RPE65 protein, providing the potential to restore the visual cycle. A single-dose subretinal injection of voretigene neparvovec administered in each eye is approved in several countries worldwide for the treatment of vision loss in adult and paediatric patients with confirmed biallelic RPE65 mutation-associated inherited retinal dystrophy (IRD) and with sufficient viable retinal cells. In the pivotal phase III trial, significant improvements from baseline were seen in the mean bilateral multi-luminance mobility test scores in the voretigene neparvovec group compared with the control group at 1 year.

Galcanezumab: A Review in the Prevention of Migraine and Treatment of Episodic Cluster Headache.

Galcanezumab (Emgality) is a humanized monoclonal antibody targeting the calcitonin gene-related peptide (CGRP), thereby inhibiting its physiological activity, with CGRP playing a key role in the pathophysiology of migraine and headache disorders. In pivotal phase 3 trials, recommended dosages of subcutaneous galcanezumab once monthly were significantly more effective than placebo as preventive therapy in adults with episodic (EVOLVE-1 and -2; over 6 months) or chronic (REGAIN; over 3 months) migraine (±  aura), including in patients who had failed several prior preventive migraine drugs (CONQUER; over 3 months). The beneficial effects of galcanezumab preventive treatment in reducing the number of monthly migraine headache days (MHDs) and improving health-related quality of life (HR-QOL) were sustained during up to 1 year of treatment.

Rimegepant: First Approval.

The orally disintegrating tablet (ODT) formulation of rimegepant (NURTEC ODT) is a small molecule, highly-selective, calcitonin gene-related peptide antagonist that was developed by Biohaven Pharmaceutical Holding Company Ltd as an acute treatment for migraine. A conventional tablet formulation of the drug is being investigated for the acute treatment (under FDA review in the USA) and prevention of migraine and the treatment of refractory trigeminal neuralgia. In February 2020, rimegepant ODT received its first global approval in the USA for the acute treatment of migraine (± aura) in adults.

Certolizumab Pegol: A Review in Moderate to Severe Plaque Psoriasis.

Certolizumab pegol (Cimzia) is a PEGylated, Fab'-only, recombinant humanized antibody against TNF-α. Subcutaneous certolizumab pegol is indicated for the treatment of various immune-mediated inflammatory diseases (IMIDs), including moderate to severe plaque psoriasis. In pivotal phase III trials in adults with moderate to severe plaque psoriasis, significantly more patients receiving certolizumab pegol 200 mg or 400 mg once every 2 weeks than placebo recipients achieved a ≥ 75% reduction in PASI score (PASI75 responder) and a PGA score of clear/mostly clear with a ≥ 2 point improvement from baseline (PGA0/1 responder) at week 12 (CIMPACT) or 16 (CIMPASI-1 and -2).

Correction to: Ubrogepant: First Approval.

The article Ubrogepant: First Approval, written by Lesley J. Scott, was originally published published Online First without Open Access. After publication online Allergan Sales, LLC requested that the article be Open Choice to make the article an open access publication.

Lemborexant: First Approval.

Lemborexant (DAYVIGO™) is an orally administered, dual orexin receptor (OXR) antagonist that exhibits reversible competitive antagonism at OXR1 and OXR2 (> affinity at OXR2) that was discovered and developed by Eisai Inc. for the treatment of adult patients with insomnia. In December 2019, lemborexant received its first approval (with final interim scheduling) in the USA for the treatment of adult patients with insomnia, characterized by difficulties with sleep onset and/or sleep maintenance.

Givosiran: First Approval.

Givosiran (Givlaari™) is an aminolevulinate synthase 1 (ALAS1)-directed small interfering RNA (siRNA) covalently linked to a ligand to enable specific delivery of the siRNA to hepatocytes. This results in downregulation of ALAS1 mRNA and prevents accumulation of neurotoxic δ-aminolevulinic acid and porphobilinogen levels that are associated with acute porphyria attacks. Givosiran is being developed by Alnylam Pharmaceuticals for the treatment of acute hepatic porphyria (AHP).

Ubrogepant: First Approval.

Ubrogepant (Ubrelvy™) is an orally administered, small molecule, highly-selective, calcitonin gene-related peptide (CGRP) antagonist that was developed by Allergan under license to Merck & Co. as an acute treatment for migraine. In December 2019, ubrogepant received its first global approval in the USA for the acute treatment of migraine (± aura) in adults.

Dulaglutide: A Review in Type 2 Diabetes.

Subcutaneous dulaglutide (Trulicity) is a once-weekly glucagon-like peptide-1 receptor agonist that is approved in numerous countries as an adjunct to diet and exercise for the treatment of adults with type 2 diabetes (T2D). In the clinical trial and real-world settings, once-weekly subcutaneous dulaglutide, as monotherapy or add-on therapy to other antihyperglycaemic agents (including oral antihyperglycaemic drugs and insulin), was an effective and generally well tolerated treatment in adults with inadequately controlled T2D, including in high-risk patients [e.g.

Dolutegravir/Lamivudine Single-Tablet Regimen: A Review in HIV-1 Infection.

The oral once-daily, fixed-dose single-tablet regimen (STR) of dolutegravir/lamivudine (Dovato), combining a second generation integrase single-strand transfer inhibitor (INSTI) and a nucleoside reverse transcriptase inhibitor (NRTI), is indicated as a complete regimen for the treatment of HIV-1 infection in adults and adolescents (> 12 years of age weighing at least 40 kg) with no known or suspected resistance to the INSTI class or lamivudine. In GEMINI trials in antiretroviral therapy (ART)-naïve HIV-1-infected adults, treatment with dolutegravir plus lamivudine provided rapid and sustained virological suppression and was noninferior to dolutegravir plus tenofovir disoproxil fumarate/emtricitabine at 48 weeks, irrespective of baseline patient or disease characteristics. Virological suppression was sustained at 96 weeks in these ongoing trials.

Trifarotene: First Approval.

Topical trifarotene (Aklief) is a first-in-class, fourth-generation retinoid [selective retinoic acid receptor (RAR)-γ agonist] being developed by Galderma Research and Development LLC for the treatment of acne vulgaris. In October 2019 trifarotene received its first global approval in the USA for the topical treatment of acne vulgaris in patients 9 years of age and older. This article summarizes the milestones in the development of trifarotene leading to its first global approval for acne vulgaris.

Venetoclax: A Review in Relapsed/Refractory Chronic Lymphocytic Leukemia.

Venetoclax (Venclyxto; Venclexta) is a first-in-class, oral, selective B cell lymphoma-2 (BCL-2) inhibitor. The drug is approved in numerous countries, including those of the EU and in the USA, for the treatment of adults with relapsed or refractory (RR) chronic lymphocytic leukemia (CLL); the specific indication(s) for venetoclax may vary between individual countries. Venetoclax monotherapy or combination therapy with rituximab was an effective treatment, provided durable responses, and had a manageable safety profile in pivotal clinical trials in adults with RR CLL, including in patients with adverse prognostic factors.

Correction to: Migalastat: A Review in Fabry Disease.

The article Migalastat: A Review in Fabry Disease, written by Emma H. McCafferty, Lesley J. Scott, was originally published Online First without open access.

Correction to: Osimertinib as first-line therapy in advanced NSCLC: a profile of its use.

[This corrects the article DOI: 10.1007/s40267-018-0536-9.].

Teriflunomide: A Review in Relapsing-Remitting Multiple Sclerosis.

Teriflunomide (Aubagio) is a disease-modifying immunomodulatory drug with anti-inflammatory properties that selectively and reversibly inhibits the mitochondrial enzyme dihydro-orotate dehydrogenase, with consequent inhibition of de novo pyrimidine synthesis and reduced lymphocyte proliferation. Based on extensive evidence from randomized controlled trials (RCTs) and the real-world setting, oral teriflunomide is an effective and generally well tolerated treatment in patients with relapsing multiple sclerosis (MS), with these benefits maintained during long-term treatment (≥ 10 years) and no new safety signals identified. In pivotal RCTs in this patient population, teriflunomide provided significantly better efficacy than placebo (TEMSO and TOWER) and was as effective as interferon β-1a (TENERE) in terms of improvements in clinical outcomes (such as reduced annualized relapse rates, prevention of disability progression) and/or MRI-assessed disease activity measures.