Arnold Lee

Pariglasgene brecaparvovec (GENGLYCOS™, pariglasgene brecaparvovec-opnr) is an adeno-associated virus serotype 8 (AAV8) vector gene therapy developed by Ultragenyx Pharmaceutical for the treatment of glycogen storage disease type Ia. As glycogen storage disease type Ia occurs due to defects in glucose-6-phosphatase, pariglasgene brecaparvovec delivers a functional copy of the G6PC gene to stabilise blood glucose levels and reduce cornstarch intake in patients. Pariglasgene brecaparvovec received an accelerated approval in the USA on 19 August 2026. This article summarises the milestones in the development of pariglasgene brecaparvovec leading to this first approval to reduce daily cornstarch intake as an adjunct to nutritional management in adult and paediatric patients ≥ 8 years of age with glycogen storage disease type Ia.

Vusolimogene oderparepvec (TUDRIQEV™; vusolimogene oderparepvec-wtpg) is a genetically modified oncolytic viral therapy that targets cancer cells. It is being developed by Replimune for the treatment of skin malignancies and received its first approval for combination treatment with nivolumab on 6 August 2026 in the USA. The combination of vusolimogene oderparepvec and nivolumab resulted in objective responses in patients with unresectable advanced melanoma after progression with an anti-PD-1 regimen during the IGNYTE phase I/II trial. This article summarises the milestones in the development of vusolimogene oderparepvec leading to this first approval in combination with nivolumab for the treatment of adult patients with unresectable advanced cutaneous melanoma who experienced disease progression with a PD-1-blocking antibody-based regimen.

Camizestrant (Etcamah^®) is an orally available, potent selective estrogen receptor degrader (SERD) and complete estrogen receptor (ER) α antagonist being developed by AstraZeneca for the treatment of breast cancer. It received its first approval in the United Arab Emirates (UAE) in May 2025. At the time of writing, the drug had been subsequently approved in Saudi Arabia, Japan, the EU, Canada, the UK and the USA. In patients with ER-positive and human epidermal growth factor receptor 2 (HER2)-negative breast cancer with a detectable ESR1 mutation, switching to camizestrant while continuing the same CDK4/6 inhibitor significantly prolonged progression-free survival in comparison with continuing treatment with an aromatase inhibitor in combination with a CDK4/6 inhibitor. This article summarizes the milestones in the development of camizestrant leading to this first approval for the treatment of adults with locally advanced or metastatic hormone receptor positive (HR+), HER2-negative breast cancer with an ESR1 gene mutation that emerges during first-line hormonal therapy, in combination with CDK4/6 inhibitors.

Tradipitant (NEREUS™) is a small molecule neurokinin-1 (NK-1) receptor antagonist being developed by Vanda Pharmaceuticals. NK-1 receptor activation and substance P release in the central nervous system leads to nausea and vomiting symptoms associated with motion sickness. Tradipitant received its first approval in the USA in December 2025 for the prevention of vomiting induced by motion in adults, and is also being developed for the treatment of gastroparesis and prevention of vomiting induced by GLP-1 receptor agonists. In phase III trials, tradipitant reduced the incidence of vomiting associated with motion sickness. This article summarizes the milestones in the development of tradipitant leading to this first approval for the prevention of vomiting induced by motion in adults.

Depemokimab (depemokimab-ulaa; EXDENSUR) is an anti-IL-5 antibody being developed by GSK for the treatment of asthma, chronic rhinosinusitis with nasal polyps (CRSwNP), chronic obstructive pulmonary disease, eosinophilic granulomatosis with polyangiitis and hypereosinophilic syndrome. Add-on treatment with depemokimab reduced asthma-related exacerbations in patients with severe asthma with an eosinophilic phenotype, in addition to reducing the severity of nasal polyps and nasal obstruction in patients with CRSwNP. This article summarizes the milestones in the development of depemokimab leading to this first approval in the UK as an add-on maintenance treatment of asthma in adult and adolescent patients aged ≥ 12 years with type 2 inflammation characterised by an eosinophilic phenotype who are inadequately controlled on maximum moderate-dose or high-dose inhaled corticosteroids plus another asthma controller; and as add-on therapy with intranasal corticosteroids for the treatment of adults with severe CRSwNP for whom therapy with systemic corticosteroids and/or surgery do not provide adequate control.

Etuvetidigene autotemcel (WASKYRA^™) is an autologous haematopoietic stem cell (HSC)-based gene therapy being developed by Fondazione Telethon for the treatment of Wiskott–Aldrich syndrome (WAS). In December 2025, etuvetidigene autotemcel was approved in the USA for the treatment of paediatric patients aged 6 months and older and adults with WAS who have a mutation in the WAS gene for whom HSC transplantation (HSCT) is appropriate and no suitable human leukocyte antigen (HLA)-matched related stem cell donor is available. Etuvetidigene autotemcel was approved in the EU for the same indication in January 2026. This article summarizes the milestones in the development of etuvetidigene autotemcel leading to this first approval for the treatment of WAS.

Sibeprenlimab (VOYXACT^®; sibeprenlimab-szsi) is a fully humanized monoclonal antibody that binds to A PRoliferation Inducing Ligand (APRIL) being developed by Otsuka Pharmaceutical for the treatment of immunoglobulin A (IgA) nephropathy and Sjögren’s disease. Signalling via APRIL is implicated in the inflammatory processes that ultimately result in damage to the glomeruli in patients with IgA nephropathy. Treatment with sibeprenlimab suppressed APRIL levels and reduced proteinuria in patients with IgA nephropathy, according to interim analyses from the ongoing VISIONARY phase III clinical trial. This article summarizes the milestones in the development of sibeprenlimab leading to this first approval to reduce proteinuria in adults with primary IgA nephropathy at risk for disease progression.

Zongertinib (HERNEXEOS^®) is a small molecule, irreversible, HER2-specific tyrosine kinase inhibitor being developed by Boehringer Ingelheim for the treatment of advanced solid tumours with HER2 aberrations. Zongertinib selectively inhibits HER2-expressing cells, including those with HER2 mutations, while sparing cells expressing wild-type EGFR. It received its first approval under accelerated approval in August 2025 in the USA following positive results from the Beamion LUNG-1 phase Ia/Ib trial in patients with previously treated HER2-mutant non-small cell lung cancer (NSCLC). This article summarizes the milestones in the development of zongertinib leading to this first approval for the treatment of adult patients with unresectable or metastatic NSCLC whose tumours have HER2 tyrosine kinase domain activating mutations, as detected by an FDA-approved test, and who have received prior systemic therapy.

Rilzabrutinib (WAYRILZ™) is a small-molecule, reversible, highly specific, covalent BTK inhibitor being developed by Sanofi for the treatment of multiple immune-related and inflammatory disorders, including immune thrombocytopenia, warm autoimmune haemolytic anaemia, sickle cell disease, IgG4-related disease, asthma, chronic spontaneous urticaria, among others. As a BTK inhibitor, rilzabrutinib demonstrates multiple immunomodulatory effects, including reduced B cell activation and autoantibody production, inhibition of macrophage activity, and anti-inflammatory effects. In clinical trials, rilzabrutinib was associated with durable platelet responses in patients with previously treated immune thrombocytopenia. This article summarizes the milestones in the development of rilzabrutinib leading to its first approval for the treatment of adults with persistent or chronic immune thrombocytopenia who have had an insufficient response to previous treatment.