The US FDA has recently approved Radicava, the first new drug to treat patients with amyotrophic lateral sclerosis (ALS) in over two decades.1

ALS is a rare but fatal disease that attacks and kills the nerve cells that control voluntary muscles.

The nerves lose the ability to activate specific muscles, which causes weakness and eventually paralysis.

ALS became well known in 1939 when baseball player Lou Gehrig became a sufferer, and physicist Stephen Hawking was diagnosed with the condition in 1963.

In Europe and the US the disease affects approximately two people per 100,000 per year, with most aged between 40 and 70 at the time of diagnosis.2

Fast track to success

Radicava is not a cure, but has been shown to slow the progression of the disease.

In a Japanese study patients receiving the treatment experienced a 33% reduction in the rate of decline of physical function.3

After learning about the study, the FDA contacted the drug developer to fast track the marketing of the drug in the US, in a move that has been highly praised by patient groups.

Limiting factors

While the new treatment is long overdue, it may not be well received by all.

Radicava is an intravenous infusion that has to be given by a healthcare professional, which makes it expensive to administer. 

Mitsubishi Tanabe is reportedly seeking a list price of $1,000 per infusion of the drug, which equates to around $146,000 per year, before rebates, discounts and patient assistance programs.4

It also carries a risk of dangerous side effects, including hives, swelling and allergic reactions.

First of many?

In 2015, the viral Ice Bucket Challenge helped fund groundbreaking research that led to the discovery of the gene associated with the disease.

It is now hoped that this will lead to further breakthroughs in the coming years, and that Radicava can open the floodgates to more new therapies that could transform the lives of sufferers.