FDA Approves Radicava (edaravone)

Monday May 8, 2017 — A new drug was approved by the Federal Drug Administration on Friday May 5, 2017 – the first drug to treat ALS approved since 1995. The only other drug on the market today is Riluzole (or, Rilutek, Sanofi-Aventis U.S. LLC) which purports to extend the lives of ALS patients by 3-4 months. (1)

While that may sound good, the price isn’t. A generic prescription can cost US$1200 for a 30 day supply at your local CVS; up to US$8,000 for 60 tablets (2). Moreover, Riluzole was tested and designed to treat patients with genetic factors of the disease that were known at the time; namely the SOD1 gene. Therefore the drug is ineffective for many cases of Familial-ALS (the type my family has) caused by the C9orf72 chromonal mutation; the main reason for that is the big breakthrough in Familial-ALS cases happened in 2012-2013, fifteen years after Rilutek came onto the market.(3) The discovery of the C9orf72 repeat accounted for about 45% of familial ALS cases.

The new drug, edaravone, (Radicava, Mitsubishi Tanabe Pharma America, Inc.) comes at an even steeper price; US$150,000 for a 14-day intravenous dose(4). That’s US$10,715 per injection delivered at your local hospital (not from CVS). And the drug is targeted to newly diagnosed patients, who have not progressed yet to, say a wheelchair or traecheotomy.

Edaravone was fast-tracked through a six-month clinical trial in Japan, after being used to treat stroke victims since 2006. Using only the data from Japan, the US FDA approved edaravone with no clinical trial. Perhaps this is the best news overall; the FDA did not require any human testing and rushed to market in the United States. So it remains to be seen how efficient, popular, safe and effective edaravone will be. At such a cost, these “live-trials” will be limited to a small number of wealthy patients. However, that could change through fund-raising and charitable donations. We can act together to raise money and sponsor patients in the early stages of this devastating disease to provide equal care for all, regardless of cost.

Former President Obama addresses an audience after being presented with the 2017 Profile in Courage award during ceremonies at the John F. Kennedy Presidential Library and Museum on Sunday in Boston. (Steven Senne/AP)

In the words of President Barack Obama, “It doesn’t take a whole load of courage to aid those who are already powerful, already comfortable, already influential. But it does require courage to champion the vulnerable, and the sick, and the infirm. Those who have no access to the corridors of power.” (5)

 

Written by: William Cunningham, May 8, 2017

 



(1) [https://www.verywell.com/als-lou-gehrigs-disease-life-expectancy-2223973]
(2) 50 mg (60 ea): $8,087.80. [https://online.epocrates.com/u/1061197/Rilutek/Pricing+Manufacturer] & see [https://www.goodrx.com/rilutek]
(3) [http://www.neurology.org/content/47/4_Suppl_2/117S.extract] Dr Teepu Siddique, “I do not see any data here…in familial cases [that it will] affect the outcome in any fashion.”
(4) Forbes May 5, 2017 [https://www.forbes.com/sites/matthewherper/2017/05/05/fda-approves-first-new-drug-to-treat-als-in-22-years]
(5) Former President Barack Obama was honored with the John F. Kennedy Profile in Courage Award in Boston Sunday, May 7, 2017.

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