The Food and Drug Administration said Friday it had approved the first new drug in 20 years to treat the paralyzing disease ALS.
"This is the first new treatment approved by the FDA for ALS in many years, and we are pleased that people with ALS will now have an additional option", he said.
Melinda Gates participates in the viral ALS ice bucket challenge in 2014.YouTube/Bill and Melinda Gates FoundationMay 5 (Reuters) -The FDA just approved a new drug for ALS.
The rare progressive condition attacks nerve cells located in the brain and spinal cord responsible for controlling voluntary muscles. It'll be sold under the brand name Radicava, and the drug should be available in the United States by August, MTPA Chief Commercial Officer Tom Larson said.
Edaravone is given as an intravenous infusion, with an initial treatment cycle of daily dosing for 14 days, followed by a 14-day drug-free period.
A clinical trial in Japan including 137 participants treated with edaravone showed clinically less decline on assessment of daily functioning compared to those receiving placebo.
It comes at a price.
The FDA approved Radicava less than a year after Mitsubishi Tanabe Pharma Corporation submitted a New Drug Application.
"If taken annually for 12 months or 13 cycles, according to the dosing and administration in the label, the cost before government discounts, will be $145,524", the company said.
The FDA sought out the drug after hearing about its use elsewhere.
In the USA, the drug had been granted orphan drug designation - raising questions about a potentially high price tag, despite the fact that generic versions of the drug are available in China and India, according to Alzforum. It appears to react with nerve-damaging compounds generated as part of the disease process.
The agency also noted the drug is associated with risks that require medical care such as hives, swelling, shortness of breath, and allergic reactions to sodium bisulfite - an ingredient that can cause anaphylactic symptoms that can be life-threatening in people with sulfite sensitivity.
ALS advocacy groups have been waiting eagerly for FDA's approval of Radicava.