Optimising bench science to withstand regulatory scrutiny

Borg, John J.; Melchiorri, Daniela; Sepodes, Bruno; Caramella, Carla; Tomino, Carlo; Micallef, Benjamin; Serracino‐Inglott, Anthony; Nisticò, Robert

doi:10.1016/j.phrs.2018.10.014

Cited by 2 publications

(2 citation statements)

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“…Recruitment of naive patients in LHON clinical trials has been reported as a slow process since there are few LHON patients in the EU. Researchers have to consider an added complexity that patients carrying the m.14484T>C point mutation have a 37% to 58% chance of spontaneous recovery of their VA which might introduce biases in the primary end point 6 that are used to study LHON in clinical trials (best recovery in VA determined using the Early Treatment Diabetic Retinopathy Study [ETDRS] charts measured by a logMAR). 7 Currently, LHON is an unmet medical need.…”

Section: Treatment Of Leber's Hereditary Optic Neuropathy: An Overviementioning

confidence: 99%

“…10 Raxone was issued a centralised marketing authorisation due to its previous designation as orphan medicinal product. 6 Raxone (idebenone 150 mg film-coated tablets) is indicated for the treatment of visual impairment in adolescent and adult patients with LHON. 11 Raxone acts as an electron carrier in the ETC, shuttling the leaking electrons onto complex III, diverting the dysfunctional complex I.…”

Section: Modulating Agent Of Mitochondrial Electron Transport Chainmentioning

confidence: 99%

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Treatment of Leber’s hereditary optic neuropathy: An overview of recent developments

Zuccarelli

Vella-Szijj

Serracino‐Inglott

et al. 2020

European Journal of Ophthalmology

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Leber’s hereditary optic neuropathy (LHON) is a rare, maternally-inherited optic neuropathy caused by mitochondrial DNA point mutations and which can cause blindness. Currently, Raxone (idebenone) is the only available medicinal product authorised to treat LHON within the European Union and LHON remains an unmet medical need. The aim of this article was to summarise interventional clinical trials published over the past 5 years (between 2014 and 2019) with the primary purpose of treating LHON. Therapeutic approaches discussed include modulating agents of the mitochondrial electron transport chain such as Raxone, cysteamine bitartrate and KH176, inhibitors of apoptosis such as elamipretide, gene therapy medicinal products such as GS010 and scAAV2P1ND4 and retinal tissue regeneration medicinal products such as bone marrow-derived stem cells.

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Section: Treatment Of Leber's Hereditary Optic Neuropathy: An Overviementioning

confidence: 99%

Section: Modulating Agent Of Mitochondrial Electron Transport Chainmentioning

confidence: 99%

Treatment of Leber’s hereditary optic neuropathy: An overview of recent developments

Zuccarelli

Vella-Szijj

Serracino‐Inglott

et al. 2020

European Journal of Ophthalmology

Self Cite

View full text Add to dashboard Cite

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Outcomes and endpoints in clinical trials supporting the marketing authorisation of treatments in paediatric acute lymphoblastic leukaemia

Micallef

Nisticò

Bjerrum

et al. 2022

Drug Discovery Today

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Optimising bench science to withstand regulatory scrutiny

Cited by 2 publications

References 4 publications

Treatment of Leber’s hereditary optic neuropathy: An overview of recent developments

Treatment of Leber’s hereditary optic neuropathy: An overview of recent developments

Outcomes and endpoints in clinical trials supporting the marketing authorisation of treatments in paediatric acute lymphoblastic leukaemia

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