Contributions
Abstract: EP1776
Type: ePoster
Abstract Category: Therapy - disease modifying - 31 Treatment of progressive MS
Objectives: MD1003 (biotin 100 mg three times per day) was granted an early access program called Qizenday® cohort Temporary Authorisation for Use (ATUc) in July 2016, following a named patient use called nominative ATU (ATUn) granted in 2015, by the French National Agency for Medicines and Health Products Safety, for treatment of adult primary (P) and secondary (S) progressive multiple sclerosis (PMS) patients 1 year relapse free. ATUc baseline characteristics at inclusion, and efficacy and safety results from both the ATUn and ATUc, are presented.
Methods: The ATUc included 5483 patients (61% female) between 13 July 2016 and 12 January 2017 (SPMS: 3080 [56%]; PPMS: 1524 [28%]; PMS type not reported: 880 [16%]); 2781 patients (51%) already received MD1003 as an ATUn. Further patients are currently included in the ATUc. To date, mean baseline values were: age: 57 years; time from diagnosis: 18 years (SPMS: 21 years; PPMS: 13 years); Expanded Disability Status Scale (EDSS) score: 6.1 (SPMS: 6.2; PPMS: 5.9) and walking distance (WD): 218.1m.
Here we report follow-up changes in EDSS, WD and clinical global impression (CGI) over a period of 1 year.
Further results covering the period until July 13 2017, with additional patients and a longer observation period, will be presented.
Results: To date, mean change in EDSS remained stable over 1 year while an increase in WD related to duration of MD1003 exposure was observed. An increased proportion of patients had improved CGI related to MD1003 exposure duration.
Safety data were consistent with the MD1003 summary of product characteristics.
Discussion: In this large cohort of patients, the efficacy and safety of MD1003 are consistent with the MS-SPI trial results.
Disclosure:
David Brassat received honoraria, consulting fees and travel grants from Bayer, Biogen, Medday, Merck-Serono, Novartis, Sanofi-Genzyme, Roche and Teva.
Patrick Hautecoeur has received board membership fees, honoraria, and paid travel accommodation from Biogen, Merck and Sanofi.
Françoise Durand-Dubief has received honoraria and consulting fees from Biogen, Sanofi-Genzyme, Novartis, Teva, Merck and Roche.
Giovanni Castelnovo has received honoraria and consulting fees from Novartis, Biogen, Teva, Sanofi, Genzyme, Merck-Serono, Merz, Ipsen and Bayer, and research support from Novartis, Merz and Ipsen.
Nathalie Derache has received speaker's honoraria from Merck-Serono, Biogen Idec, Novartis, Teva and Sanofi-Genzyme.
Bertrand Bourre serves on a scientific advisory board for Merck Serono and has received funding for travel and honoraria from Biogen Idec, Merck Serono, Novartis, Sanofi-Genzyme and Teva.
Emmanuelle Le Page has received honorarium as speaker or consultant from Biogen Idec, Sanofi Genzyme, Merck Serono, Novartis, Teva and Roche.
Cecil Donze has nothing to disclose.
Bertrand Audouin has nothing to disclose.
Jean-Christophe Ouallet has received consultancy fees, speaker fees, research grants (non-personal), and honoraria from Novartis, Biogen, Merck, Roche, Teva and Genzyme.
Nicolas Collongues has received board membership fees, honoraria, and paid travel accommodation from Biogen, Merck, Roche, Novartis and Sanofi.
Olivier Simon is an employee of Medday.
Guillaume Brion is an employee of Medday.
Patrick Vermersch has received honoraria and consulting fees from Biogen, Sanofi-Genzyme, Bayer, Novartis, Teva, Merck-Serono, Roche, Medday and Almirall, and research support from Biogen, Bayer, Novartis, Sanofi-Genzyme, Roche and Merck-Serono.
Abstract: EP1776
Type: ePoster
Abstract Category: Therapy - disease modifying - 31 Treatment of progressive MS
Objectives: MD1003 (biotin 100 mg three times per day) was granted an early access program called Qizenday® cohort Temporary Authorisation for Use (ATUc) in July 2016, following a named patient use called nominative ATU (ATUn) granted in 2015, by the French National Agency for Medicines and Health Products Safety, for treatment of adult primary (P) and secondary (S) progressive multiple sclerosis (PMS) patients 1 year relapse free. ATUc baseline characteristics at inclusion, and efficacy and safety results from both the ATUn and ATUc, are presented.
Methods: The ATUc included 5483 patients (61% female) between 13 July 2016 and 12 January 2017 (SPMS: 3080 [56%]; PPMS: 1524 [28%]; PMS type not reported: 880 [16%]); 2781 patients (51%) already received MD1003 as an ATUn. Further patients are currently included in the ATUc. To date, mean baseline values were: age: 57 years; time from diagnosis: 18 years (SPMS: 21 years; PPMS: 13 years); Expanded Disability Status Scale (EDSS) score: 6.1 (SPMS: 6.2; PPMS: 5.9) and walking distance (WD): 218.1m.
Here we report follow-up changes in EDSS, WD and clinical global impression (CGI) over a period of 1 year.
Further results covering the period until July 13 2017, with additional patients and a longer observation period, will be presented.
Results: To date, mean change in EDSS remained stable over 1 year while an increase in WD related to duration of MD1003 exposure was observed. An increased proportion of patients had improved CGI related to MD1003 exposure duration.
Safety data were consistent with the MD1003 summary of product characteristics.
Discussion: In this large cohort of patients, the efficacy and safety of MD1003 are consistent with the MS-SPI trial results.
Disclosure:
David Brassat received honoraria, consulting fees and travel grants from Bayer, Biogen, Medday, Merck-Serono, Novartis, Sanofi-Genzyme, Roche and Teva.
Patrick Hautecoeur has received board membership fees, honoraria, and paid travel accommodation from Biogen, Merck and Sanofi.
Françoise Durand-Dubief has received honoraria and consulting fees from Biogen, Sanofi-Genzyme, Novartis, Teva, Merck and Roche.
Giovanni Castelnovo has received honoraria and consulting fees from Novartis, Biogen, Teva, Sanofi, Genzyme, Merck-Serono, Merz, Ipsen and Bayer, and research support from Novartis, Merz and Ipsen.
Nathalie Derache has received speaker's honoraria from Merck-Serono, Biogen Idec, Novartis, Teva and Sanofi-Genzyme.
Bertrand Bourre serves on a scientific advisory board for Merck Serono and has received funding for travel and honoraria from Biogen Idec, Merck Serono, Novartis, Sanofi-Genzyme and Teva.
Emmanuelle Le Page has received honorarium as speaker or consultant from Biogen Idec, Sanofi Genzyme, Merck Serono, Novartis, Teva and Roche.
Cecil Donze has nothing to disclose.
Bertrand Audouin has nothing to disclose.
Jean-Christophe Ouallet has received consultancy fees, speaker fees, research grants (non-personal), and honoraria from Novartis, Biogen, Merck, Roche, Teva and Genzyme.
Nicolas Collongues has received board membership fees, honoraria, and paid travel accommodation from Biogen, Merck, Roche, Novartis and Sanofi.
Olivier Simon is an employee of Medday.
Guillaume Brion is an employee of Medday.
Patrick Vermersch has received honoraria and consulting fees from Biogen, Sanofi-Genzyme, Bayer, Novartis, Teva, Merck-Serono, Roche, Medday and Almirall, and research support from Biogen, Bayer, Novartis, Sanofi-Genzyme, Roche and Merck-Serono.