Lenacapavir: First Approval.

Lenacapavir (Sunlenca) is a long-acting capsid inhibitor of human immunodeficiency virus type 1 (HIV-1) being developed by Gilead Sciences Inc. It is available as an oral tablet and injectable solution, with the latter being a slow-release formulation to allow bi-annual subcutaneous administration. In August 2022, lenacapavir received its first approval in the EU for use in combination with other antiretroviral(s) in adults with multi-drug resistant HIV infection, for whom it is otherwise not possible to construct a suppressive anti-viral regimen.

Ozanimod: A Review in Ulcerative Colitis.

Ozanimod (Zeposia) is the first sphingosine-1-phosphate receptor (S1PR) modulator to be approved for the treatment of adults with moderately to severely active ulcerative colitis in the USA, and in adults with moderately to severely active ulcerative colitis who have had an inadequate or lost response to, or were intolerant of, either conventional therapy or a biologic in the EU. An oral agent, ozanimod is administered once daily as induction and maintenance therapy. In the randomized, double-blind, multinational phase 2 Touchstone and phase 3 True North clinical trials, ozanimod was effective in inducing clinical remission and maintaining remission relative to placebo in adults with moderately to severely active ulcerative colitis.

Dabigatran Etexilate: A Review in Pediatric Venous Thromboembolism.

Although rare in children, venous thromboembolism (VTE) is markedly more likely in hospitalized patients, particularly with the use of central venous access devices. Dabigatran etexilate (Pradaxa) is one of the first direct non-vitamin K antagonist oral anticoagulants (DOAC) approved for use in pediatric patients. It is approved in the EU and USA for the treatment of VTE in patients who have been treated with a parenteral anticoagulant for ≥ 5 days, and for the prevention of recurrent VTE.

Nivolumab Plus Relatlimab: First Approval.

Nivolumab plus relatlimab (nivolumab and relatlimab-rmbw; Opdualag) is a fixed-dose, combination immunotherapy treatment being developed by Bristol Myers Squibb for the treatment of multiple types of advanced cancers. Both drugs are immunoglobulin G4 (IgG4) monoclonal antibodies developed to target immune checkpoints, with nivolumab targeting the programmed cell death protein 1 (PD-1) receptor and relatlimab being a newly developed, first-in-class drug targeting the lymphocyte-activation gene 3 (LAG-3) protein. In March 2022, nivolumab plus relatlimab received its first approval in the USA for the treatment of unresectable or metastatic melanoma in adult patients and paediatric patients aged ≥ 12 years who weigh ≥ 40 kg.

Risdiplam: A Review in Spinal Muscular Atrophy.

Risdiplam (Evrysdi) is the first oral drug developed to treat spinal muscular atrophy (SMA) and is approved in multiple countries worldwide. It is approved for the treatment of SMA in patients aged ≥ 2 months in the USA and the EU, with this approval further specified in the EU for the treatment of 5q-autosomal recessive SMA with a clinical diagnosis of SMA types 1, 2, or 3 or with one to four survival motor neuron 2 (SMN2) copies. As an SMN2 pre-mRNA splicing modifier, risdiplam increases the production of full-length SMN protein, the lack of which drives the pathophysiology of SMA.

Olaparib: A Review as First-Line Maintenance Therapy in Advanced Ovarian Cancer.

Olaparib (Lynparza) is a poly (adenosine diphosphate-ribose) polymerase (PARP) inhibitor approved for first-line maintenance treatment in adults with advanced ovarian cancer who are in complete or partial response to first-line, platinum-based chemotherapy. Originally approved as monotherapy, olaparib is also approved to be administered in combination with bevacizumab in patients whose cancer is associated with homologous recombination deficiency (HRD), defined by either a BRCA1/2 mutation and/or genomic instability. In phase III trials, olaparib monotherapy significantly improved progression-free survival (PFS) relative to placebo (SOLO-1), as did olaparib plus bevacizumab relative to placebo plus bevacizumab (PAOLA-1), in patients with advanced ovarian cancer who had responded to platinum-based chemotherapy.

Teriflunomide: Pediatric First Approval.

Teriflunomide (Aubagio), which was developed by Sanofi, is an oral immunomodulatory agent targeting the mitochondrial enzyme dihydroorotate dehydrogenase and available to adults to treat relapsing-remitting multiple sclerosis (MS). On 18 June 2021, teriflunomide received its first approval in this indication in pediatric patients aged ≥ 10 years in the EU. This article summarizes the milestones in the development of teriflunomide leading to this first pediatric approval for relapsing-remitting MS.

Olanzapine/Samidorphan: First Approval.

Samidorphan, which was developed by Alkermes, is an opioid receptor antagonist that has been co-formulated with olanzapine into a single-dose oral tablet to mitigate the risk of weight gain while providing the therapeutic effect of olanzapine. Olanzapine/samidorphan (LYBALVI) was recently approved in the USA for the treatment of schizophrenia and bipolar I disorder. This article summarizes the milestones in the development of samidorphan leading to this first approval of olanzapine/samidorphan.

Diroximel Fumarate in Relapsing Forms of Multiple Sclerosis: A Profile of Its Use.

Diroximel fumarate (Vumerity), an orally administered disease-modifying drug (DMD), expands the available treatment options for adults with relapsing forms of multiple sclerosis (MS), including clinically isolated syndrome, relapsing-remitting MS (RRMS), and active secondary progressive MS. It demonstrates bioequivalence to dimethyl fumarate and was developed to provide similar clinical benefits, but with an improved gastrointestinal (GI) tolerability profile. In RRMS patients who are treatment-naïve or were previously treated with interferon-β or glatiramer acetate, diroximel fumarate reduces annualized relapse rates, with most patients experiencing no relapses during treatment, and reduces the formation of new MS-associated brain lesions.

Fostamatinib: A Review in Chronic Immune Thrombocytopenia.

Fostamatinib (Tavalisse; Tavlesse) is the first spleen tyrosine kinase (Syk) inhibitor approved for the treatment of chronic immune thrombocytopenia (ITP) in adult patients who have had an insufficient response to previous treatment. By inhibiting Syk activation in macrophages, fostamatinib blocks autoantibody-mediated platelet phagocytosis. In the placebo-controlled phase III FIT1 and FIT2 trials, 24 weeks of oral fostamatinib therapy increased platelet count in previously treated adults with ITP.

Correction to: Romosozumab: A Review in Postmenopausal Osteoporosis.

Unfortunately the sections were published incorrectly in the original article.

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.

Topical Minocycline Foam 4%: A Review in Acne Vulgaris.

Topical minocycline foam 4% (Amzeeq™) is approved in the USA for the treatment of inflammatory lesions of non-nodular, moderate to severe acne vulgaris (acne) in patients aged ≥ 9 years. It was developed to minimize systemic minocycline absorption and toxicity, and its high lipid content allows efficient drug movement through sebum and into affected sites. The favorable in vitro resistance profile of oral minocycline seen in Cutibacterium acnes (C.

Levodopa Inhalation Powder: A Review in Parkinson's Disease.

Levodopa inhalation powder (Inbrija) is approved for the intermittent treatment of OFF episodes in patients with Parkinson's disease (PD) treated with levodopa/dopa-decarboxylase inhibitor (LD-DCI) in the EU and specifically with carbidopa/levodopa in the USA. The approved dosage is 84 mg taken as needed up to five times a day. Administered via a breath-actuated inhaler, this formulation enables levodopa to bypass the gastrointestinal (GI) tract and, instead, rapidly enter the bloodstream through the pulmonary system.

Correction to: Dapagliflozin: A Review in Type 1 Diabetes.

The article Dapagliflozin: A Review in Type 1 Diabetes, written by Julia Paik and Hannah A. Blair, was originally published Online First without Open Access.

Dapagliflozin: A Review in Type 1 Diabetes.

Oral dapagliflozin (Edistride, Forxiga) is approved in the EU at a dosage of 5 mg/day as an adjunct to insulin in adults with type 1 diabetes (T1D) and a body mass index (BMI) of ≥ 27 kg/m, when insulin alone does not provide adequate glycaemic control despite optimal insulin therapy. As a highly selective SGLT2 inhibitor, dapagliflozin decreases plasma glucose levels independently of insulin action and enables glycaemic control improvement without increasing the risks associated with intensive insulin therapy. In the phase III DEPICT-1 and -2 trials, dapagliflozin 5 mg/day as an adjunct to insulin improved glycaemic control and reduced total daily insulin dose and bodyweight relative to placebo in adults with inadequately controlled T1D, including in patients with a BMI of ≥ 27 kg/m, over 24 weeks of treatment.

Correction to: Triamcinolone Acetonide Extended-Release: A Review in Osteoarthritis Pain of the Knee.

An Online First version of this article was made available online at https://rd.springer.com/article/10.

Correction to: Damoctocog Alfa Pegol: A Review in Haemophilia A.

The article Damoctocog Alfa Pegol: A Review in Haemophilia A, written by Julia Paik and Emma D. Deeks, was originally published Online First without open access.

Volanesorsen: First Global Approval.

Volanesorsen (Waylivra), an antisense oligonucleotide inhibitor of apolipoprotein CIII (apoCIII) mRNA, is being developed by Ionis Pharmaceuticals through its subsidiary company, Akcea Therapeutics, to treat familial chylomicronemia syndrome (FCS), hypertriglyceridemia and familial partial lipodystrophy (FPL). In May 2019, volanesorsen was approved in the EU for the treatment of adult patients with FCS based on positive results from the multinational, phase III APPROACH and COMPASS studies. Other clinical trials are ongoing to assess its utility in hypertriglyceridemia, FPL and partial lipodystrophy.

Damoctocog Alfa Pegol: A Review in Haemophilia A.

Damoctocog alfa pegol (Jivi) is approved in the USA, EU, Japan and Canada for the treatment and prophylaxis of previously treated patients aged ≥ 12 years with haemophilia A. Formulated with a 60 kDa polyethylene glycol (PEG) moiety, damoctocog alfa pegol is an intravenously (IV) administered recombinant factor VIII (rFVIII) product with a longer terminal half-life than non-PEGylated FVIII and rFVIII products. In the multinational phase II/III PROTECT VIII trial, prophylaxis with damoctocog alfa pegol reduced the likelihood of bleeding in previously treated patients aged ≥ 12 years with severe haemophilia A, with dosing schedules ranging from twice weekly to once every 7 days.

Correction to: Triamcinolone Aceonide Extended-Release: A Review in Osteoarthritis Pain of the Knee.

An Online First version of this article was made available online at.