FDA approves first new drug to treat ALS in 20 years
The Food and Drug Administration said Friday that it had approved the first new drug in 20 years to treat ALS paralyzing disease.
The drug is not a cure but may slow the inexorable worsening of the disease, which gradually paralyzes patients completely. This is always fatal and there is no cure.
Amyotrophic lateral sclerosis (ALS), sometimes known as Lou Gehrig's disease, is rare and affects between 12,000 and 15,000 Americans, according to the Centers for Disease Control and Prevention.
The new drug, called Radicava or edaravone, was developed in Japan, where it was approved to treat strokes.
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"After learning about the use of edaravone to treat ALS in Japan, we quickly engaged with the drug developer to apply for marketing in the US," said Dr. Eric Bastings, Deputy Director of the Division of Neurological Products at the FDA.
"This is the first new FDA-approved treatment for ALS for many years, and we are delighted that people with ALS have an additional option," he said.
This is actually the first new drug approved for AL since 1995, when Riluzole, sold under the brand name Rilutek, was approved.
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Radicave is administered in the form of an intravenous infusion, with two weeks of daily treatment followed by a two week break.
Tests on a very small group of 137 patients showed that those who received the drug had slower declines compared to those who did not.
It comes at a price. Mitsubishi Tanabe Pharma America says the drug will cost more than $ 1,000 per infusion.
"If taken every year for 12 months or 13 cycles, depending on the dosage and administration in the label, the cost before government remittances will be $ 145,524," the company said.
]"We offer co-production assistance for commercially insured patients in order to reduce their labor cost," added the company. "The price of Radicava is at the midpoint of an orphan drug in the US and represents the investment that MT Pharma America is making to deliver it to US patients."
An orphan drug is unlikely to have many buyers, so the FDA speeds up the approval.
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Radicava is what is called a free radical scavenger. It appears to react with compounds harmful to the nerves generated as part of the disease process.
It can have serious side effects.
"Radicava is also associated with serious risks that require immediate medical attention, such as a hives, swelling or shortness of breath and allergic reactions to sodium bisulfite, an ingredient of the medication." Sodium bisulfite can cause symptoms Anaphylactic drugs that can be life threatening in people suffering from sulfite sensitivity, "the FDA said.
Rilutek has a different method of action, helping to stop the death of nerve cells, but only for a short time and only prolongs the lives of patients within a few months on average.
ALS advocacy groups are eagerly awaiting approval by the FDA of Radicava.
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