Alpelisib (Piqray™)—an orally available phosphatidylinositol 3-kinase (PI3K) inhibitor with specific activity against PI3K alpha (PI3Kα)—is being developed by Novartis for the treatment of breast ...cancer. Alpelisib has demonstrated efficacy in combination with fulvestrant as treatment for hormone receptor (HR)-positive, human epidermal growth factor receptor-2 (HER2)-negative breast cancer in patients with a PIK3CA mutation and was recently approved for this indication in the USA. This article summarizes the milestones in the development of alpelisib leading to this first approval.
Selpercatinib (RETEVMO™) is a receptor tyrosine kinase RET (rearranged during transfection) inhibitor being developed by Loxo Oncology for the treatment of cancers harbouring RET alterations. Based ...on results from the phase I/II LIBRETTO-001 trial, selpercatinib was recently approved by the US FDA for the treatment of
RET
fusion-positive non-small-cell lung cancer,
RET
fusion-positive thyroid cancer and
RET
-mutant medullary thyroid cancer. This article summarizes the milestones in the development of selpercatinib leading to this first approval.
Setmelanotide: First Approval Markham, Anthony
Drugs (New York, N.Y.),
02/2021, Letnik:
81, Številka:
3
Journal Article
Recenzirano
Setmelanotide (IMCIVREE™, Rhythm Pharmaceuticals) is a melanocortin-4 (MC4) receptor agonist developed for the treatment of obesity arising from proopiomelanocortin (POMC), proprotein convertase ...subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency. The drug has received its first approval in the USA for chronic weight management in patients 6 years and older with obesity caused by POMC, PCSK1 and LEPR deficiency and has been granted PRIority MEdicines (PRIME) designation by the European Medicines Agency for the treatment of obesity and the control of hunger associated with deficiency disorders of the MC4 receptor pathway. Setmelanotide is also being developed in other rare genetic disorders associated with obesity including Bardet–Biedl Syndrome, Alström Syndrome, POMC and other MC4R pathway heterozygous deficiency obesities, and POMC epigenetic disorders. This article summarizes the milestones in the development of setmelanotide leading to this first approval for obesity caused by POMC, PCSK1 and LEPR deficiency.
Belantamab mafodotin (BLENREP
™
; belantamab mafodotin-blmf) is a first-in-class monoclonal antibody-drug conjugate (ADC) that has been developed for the treatment of multiple myeloma by ...GlaxoSmithKline. The ADC comprises an antibody targeting B-cell maturation antigen (BCMA) conjugated to the microtubule inhibitor monomethyl auristatin F (MMAF). The antibody moiety binds to BCMA on the tumour cell surface, delivering the cytotoxic microtubule inhibitor MMAF to the therapeutic target. Based on preliminary results from the multinational DREAMM-2 trial, belantamab mafodotin was approved in early August 2020 in the USA for the treatment of relapsed or refractory multiple myeloma in adult patients who have received at least four prior therapies, including an anti CD38 monoclonal antibody, a proteasome inhibitor, and an immunomodulatory agent. The ADC was also approved in the EU for this indication in late August 2020. This article summarizes the milestones in the development of belantamab mafodotin leading to this first approval.
Savolitinib: First Approval Markham, Anthony
Drugs (New York, N.Y.),
09/2021, Letnik:
81, Številka:
14
Journal Article
Recenzirano
Savolitinib (Orpathys
®
; HUTCHMED, AstraZeneca) is a receptor tyrosine kinase mesenchymal epithelial transition factor (MET) inhibitor being developed for the treatment of metastatic non-small cell ...lung cancer (NSCLC), papillary and clear cell renal cell carcinoma (RCC), gastric cancer and colorectal cancer. Based on the results of a pivotal phase II trial in patients with NSCLC/pulmonary sarcomatoid carcinoma, savolitinib was recently granted approval in China (conditional on the results of a phase III trial) for the treatment of metastatic NSCLC with MET exon 14-skipping alterations in patients who have progressed after or who are unable to tolerate platinum-based chemotherapy. This article summarizes the milestones in the development of savolitinib leading to this first approval.
Tisotumab vedotin (Tivdak™) is an antibody-drug conjugate comprising a fully human monoclonal antibody specific for tissue factor (TF-011) conjugated to monomethyl auristatin E (MMAE) that has been ...engineered to target tissue factor expressing tumours. Based on the results of a phase II trial, tisotumab vedotin has been granted accelerated approval in the USA for the treatment of adult patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy. This article summarizes the milestones in the development of tisotumab vedotin leading to this first approval.
Erdafitinib (Balversa™, Janssen Pharmaceutical Companies) is a pan-fibroblast growth factor receptor (FGFR) inhibitor that was recently approved in the USA for the treatment of locally advanced or ...metastatic FGFR3 or FGFR2 urothelial carcinoma. The drug is also being investigated as a treatment for other cancers including cholangiocarcinoma, liver cancer, non-small cell lung cancer, prostate cancer, lymphoma and oesophageal cancer. This article summarizes the milestones in the development of erdafitinib leading to this first approval for the treatment of urothelial carcinoma.
Pralsetinib: First Approval Markham, Anthony
Drugs (New York, N.Y.),
11/2020, Letnik:
80, Številka:
17
Journal Article
Recenzirano
Pralsetinib (GAVRETO™, Blueprint Medicines Corporation) is a selective rearranged during transfection (RET) inhibitor being developed for the treatment of various solid tumours. RET is a well ...described proto-oncogene present in multiple cancers including non-small cell lung cancer (NSCLC), papillary thyroid cancer, and medullary thyroid carcinoma (MTC). Pralsetinib was recently granted accelerated approval for the treatment of metastatic RET fusion-positive NSCLC in the USA and is under regulatory review in the USA for RET fusion-positive thyroid cancer and RET mutation-positive MTC; pralsetinib is under regulatory review in the EU for RET fusion-positive NSCLC. This article summarizes the milestones in the development of pralsetinib leading to this first approval.
REGN-EB3: First Approval Markham, Anthony
Drugs,
2021/1, Letnik:
81, Številka:
1
Journal Article
Recenzirano
Odprti dostop
REGN-EB3 (INMAZEB
®
, Regeneron Pharmaceuticals) is a combination of three fully-human monoclonal antibodies—atoltivimab (REGN3470), maftivimab (REGN3479), and odesivimab (REGN3471) —that target ...Ebola virus glycoprotein. Based on the results of the PALM study conducted during an Ebola outbreak in the Democratic Republic of Congo, REGN-EB3 was recently approved by the US FDA as a treatment for Ebola virus infection. This article summarizes the milestones in the development of REGN-EB3 leading to this first approval for the treatment of infection caused by
Zaire ebolavirus
(Ebola virus) in adult and paediatric patients.
Cerliponase alfa (Brineura™) is a recombinant human tripeptidyl peptidase-1 (TPP1) being developed by BioMarin Pharmaceutical Inc. for use in patients with neuronal ceroid lipofuscinosis type 2 ...(CLN2), a paediatric neurodegenerative disease caused by a deficiency in TPP1. CLN2 is characterised by progressive impairment of motor function, language deficiencies, seizures, ataxia, blindness and early death, and intracerebroventricular infusion of cerliponase alfa has been shown to reduce the progression of functional decline. This article summarizes the milestones in the development of cerliponase alfa leading to its first global approval in the USA for the treatment of motor function loss in paediatric patients ≥3 years of age with CLN2, and subsequent approval in the EU for CLN2 in all ages.
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