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Edaravone
At 17:00 EST on Friday May 5th. The FDA in the US made an unexpected announcement that they had approved a new drug for ALS - `Edavarone`.
This is excellent news, is most welcome and is the first new drug to be officially approved for ALS since 1995.
This brings a much-needed additional medication to a neurological condition for which there is a critically unmet need.
Previously, there was only one drug `Riluzole` licenced worldwide, and it is hoped that `Edavarone` will gain approved status globally before the end of 2017.
Whereas it is marvellous that another ALS drug has been approved, there are practical issues to be considered.
A respected Professor of Neurology and a Principal Investigator for ALS clinical trials, Dr. Jeremy Shefner, wisely observed: "As a community, we are all vulnerable to being overly hopeful and acceptingon face value, preliminary data that is expressed positively.”
The Edavarone clinical trial was in a selected group of patients who were in early stage of the disease, had to meet minimum criteria of time since diagnosis, forced vital capacity and minimum ALSFRS-R score.
PALS taking Edavarone, will have to travel to a clinic 20 days per week as it requires intra venous infusion (i.e., unlike a diabetes intra-muscular injection, it cannot be self-administered at home or by a medically unqualified carer) taking perhaps one hour each day.
A side effect referred to has drawn attention. Glucose appears in the urine. It means the level of glucose in the blood exceeds the ability of the kidneys to absorb it. It was not reported if those patients were diabetics or had some kidney issue.
It must be remembered that it can be difficult to obtain information as not all data is published. The following information is from various sources but it must be remembered that facts can change with the passage of time:
Believed correct May `17 / Riluzole / Edaravon / RCH4Licencing status / Licenced globally / Licenced US & Japan / Unlicenced
Dose form / Oral tablet / Slow venal infusion / Muscle injection
Dose frequency / Daily / 20 days per month / Once weekly
At home? / Yes / No / Yes
Monthly cost / ~ $1,100 / ~ $12,000 / Free of charge
ALSFRS-R score slowing / No. Extends time to tracheotomy / 33% slowed In clinical trial / 63% (2016)
Number of patient treatment-years / Many thousands / 35 treatment-years in a clinical trial / 47 treatment-years
Source of efficacy data / Clinical trial / Clinical trial / Patients own monthly monitoring reports
ALSFRS-R decline / efficacy charts published / No / No / Yes
Side effects reported / Generally minor / Urine glucose / None reported