Seladelpar: First Approval.

Seladelpar (LIVDELZI) is an oral delpar [i.e. a selective peroxisome proliferator-activated receptor (PPAR)δ agonist] being developed by Gilead Sciences for the treatment of primary biliary cholangitis (PBC).

Futibatinib: A Review in Locally Advanced and Metastatic Cholangiocarcinoma.

Futibatinib (LYTGOBI) is an oral small molecule compound that selectively, irreversibly and potently inhibits the tyrosine kinase activity of fibroblast growth factor receptor (FGFR)1-4. It is approved in the EU, Japan and the USA for the treatment of adults with locally advanced or metastatic cholangiocarcinoma (CCA) harbouring an FGFR2 fusion or rearrangement who have progressed following systemic therapy. In the phase II part (FOENIX-CCA2) of a multinational phase I/II study in this patient population, monotherapy with futibatinib 20 mg once daily was associated with clinically meaningful and durable responses, sustained health-related quality of life (HR-QOL), and a manageable safety profile with supportive care and as-needed dose modifications.

Exagamglogene Autotemcel: First Approval.

Exagamglogene autotemcel (Casgevy™) is a genetically modified autologous CD34 cell enriched population. It contains human haematopoietic stem and progenitor cells edited ex vivo by CRISPR/Cas9 (a DNA double strand break-inducing nuclease system) to differentiate into erythroid cells that produce high levels of foetal hemoglobin. Developed by Vertex Pharmaceuticals and CRISPR Therapeutics, exagamglogene autotemcel received its first approval on 16 November 2023 in the UK for the treatment of transfusion-dependent β-thalassemia (TDT) in patients aged ≥ 12 years for whom haematopoietic stem cell (HSC) transplantation is appropriate and a human leukocyte antigen matched related HSC donor is not available.

Bosutinib: Pediatric First Approval.

Bosutinib (BOSULIF), an orally administered BCR-ABL tyrosine kinase inhibitor (TKI) developed by Pfizer Inc., is well established in the EU and the USA as a treatment for adults with newly diagnosed (ND) chronic phase (CP) Philadelphia chromosome-positive (Ph+) chronic myelogenous leukemia (CML), and for CP, accelerated phase and blast phase Ph+ CML that is resistant or intolerant (R/I) to prior therapy. In September 2023, based on clinical data from patients aged ≥ 1 to < 18 years, bosutinib was approved in the USA for the treatment of pediatric patients aged ≥ 1 year with CP Ph+ CML that is ND or R/I to prior therapy.

Solriamfetol: A Review in Excessive Daytime Sleepiness Associated with Narcolepsy and Obstructive Sleep Apnoea.

Solriamfetol (SUNOSI) is an oral selective dopamine and norepinephrine reuptake inhibitor approved in the EU and the USA for improving wakefulness in adults with excessive daytime sleepiness (EDS) associated with narcolepsy or obstructive sleep apnoea (OSA). In phase III studies, 12 weeks' therapy with solriamfetol within the recommended dosage range for narcolepsy (75 mg or 150 mg once daily) or OSA (37.5 mg, 75 mg or 150 mg once daily) provided early and sustained reductions in excessive sleepiness and improvements in wakefulness relative to placebo.

Delandistrogene Moxeparvovec: First Approval.

Delandistrogene moxeparvovec (delandistrogene moxeparvovec-rokl; ELEVIDYS) is an adeno-associated virus (AAV) vector-based gene therapy designed to deliver a gene encoding a micro-dystrophin protein [i.e. a shortened (138 kDa) version of the dystrophin protein expressed in normal muscle cells (427 kDa)] to all muscles involved in the pathology of Duchenne muscular dystrophy (DMD).

Eftrenonacog Alfa: A Review in Haemophilia B.

Eftrenonacog alfa (Alprolix) is an extended half-life recombinant factor IX (rFIX)-Fc fusion protein (hereafter referred to as rFIXFc). Administered as an intravenous bolus, it is approved for prophylactic use and the treatment of bleeding in patients with haemophilia B in various countries worldwide, including those of the EU, as well as the USA. In multinational, phase III trials, rFIXFc was effective for the prophylaxis, perioperative management or on-demand treatment of bleeding in male patients with severe haemophilia B regardless of age and irrespective of whether or not they had been previously treated with FIX replacement products.

Ruxolitinib Cream 1.5%: A Review in Mild to Moderate Atopic Dermatitis.

Ruxolitinib cream 1.5% (OPZELURA™) is a topical formulation of ruxolitinib, a potent, selective inhibitor of Janus kinase (JAK)1 and JAK2. The targeting of these kinases is associated with therapeutic benefits in patients with atopic dermatitis (AD).

Deucravacitinib: First Approval.

Deucravacitinib (SOTYKTU™) is a first-in-class, highly selective, oral tyrosine kinase 2 (TYK2) inhibitor. It acts via an allosteric mechanism, binding to the catalytically inactive pseudokinase regulatory domain of TYK2 and stabilizing an inhibitory interaction between the regulatory and catalytic domains. Deucravacitinib is being developed by Bristol Myers Squibb for the treatment of multiple immune-mediated diseases, including psoriasis, psoriatic arthritis, lupus and inflammatory bowel disease.

Amubarvimab/Romlusevimab: First Approval.

Amubarvimab /romlusevimab is a combination of two neutralizing recombinant human IgG1 monoclonal antibodies (amubarvimab and romlusevimab) against the spike protein of severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), the causative agent of coronavirus disease 2019 (COVID-19). Jointly developed by Brii Biosciences, Tsinghua University and the Third People's Hospital of Shenzhen, it has been approved (in December 2021) by the National Medical Products Administration of China for the treatment of mild COVID-19 in patients aged ≥ 18 years, and those aged 12-17 years with a bodyweight of ≥ 40 kg (conditional approval) who are at high risk of progressing to severe disease, including hospitalization or death. An Emergency Use Authorization application for amubarvimab/romlusevimab is currently under review in the USA.

Pimitespib: First Approval.

Pimitespib (Jeselhy) is an oral small molecule inhibitor of the α and β isoforms of heat shock protein 90 (HSP90). HSP90α and HSP90β regulate the stability and activity of a number of proteins that are crucial for tumour development. Pimitespib is being developed by Taiho Pharmaceutical for the treatment of solid tumours, including gastrointestinal stromal tumour (GIST), and in June 2022 it received its first approval in Japan for GIST that has progressed after chemotherapy.

Oteseconazole: First Approval.

Oteseconazole (VIVJOA™) is an orally administered azole antifungal agent developed by Mycovia Pharmaceuticals for the treatment of fungal infections. It inhibits cytochrome P450 (CYP) 51, thereby affecting the formation and integrity of the fungal cell membrane, but has a low affinity for human CYP enzymes due to its tetrazole metal-binding group. Oteseconazole is the first agent to be approved (in April 2022) for recurrent vulvovaginal candidiasis (RVVC) in the USA, where it is indicated to reduce the incidence of RVVC in females with a history of RVVC who are NOT of reproductive potential.