FDA Fast-Tracks Biogen’s Breakthrough Cure for Exceedingly Rare ALS Variant

The US Food and Drug Administration (FDA) has granted accelerated approval to Biogen’s new treatment for a rare form of amyotrophic lateral sclerosis (ALS), allowing patients to access the therapy more quickly.

The approval was given to Biogen’s drug Torferson for use in those suffering from the neuromuscular disorder, which is also known as Lou Gehrig’s disease in the US. The accelerated program is designed to fast-track the approval of drugs for serious conditions with limited medication options, where the drug is shown to have an effect on an endpoint that is reasonably likely to predict a clinical benefit to patients.

ALS is a rare genetic disorder that attacks and kills the nerve cells controlling voluntary muscles, including those used to move, speak, eat, and breathe. The majority of people with the disease die from respiratory failure within three to five years from the onset of symptoms, although around 10% survive for more than a decade. It is thought to impact around 20,000 Americans annually, with 5,000 new cases diagnosed each year.

The most common form of the illness, sporadic ALS, can affect anyone, with only around 5-10% of cases thought to be hereditary. The new drug will target those with this rarer, hereditary form of the disease, which is estimated to account for around 1-2% of all ALS cases.

In a statement on the FDA’s website, the regulatory body stated that the approval, which came after the results of several clinical trials, demonstrated that the new drug is the “first approved treatment for this specific genetic form of ALS”.

Dr. Billy Dunn, Director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research, added that the decision to grant accelerated approval was made due to the seriousness of the condition, and the urgent need for new, pioneering therapies for ALS sufferers.

The news has been welcomed by Derek Andrews, President of The ALS Association Greater Philadelphia Chapter, who said it is a “landmark advancement in the development of an effective treatment for ALS”, and that it is “the first time a genetically-specific approach to treating ALS has ever been successfully applied”.

However, the FDA was keen to stress that, while the accelerated approval program enables patients to access the therapy more quickly, further clinical trials are necessary in order to fully assess the risks and benefits of this potentially groundbreaking new drug.

Biogen has already reaffirmed its commitment to pursuing additional research into the rare form of ALS, with the hope that it will eventually lead to a cure for this debilitating condition.

The FDA’s decision to grant accelerated approval to Torferson could potentially act as a catalyst for further investment in ALS research and the development of new treatments for this rare genetic disorder.

Indeed, the past few years have seen an increased focus on ALS, primarily due to the so-called “Ice Bucket Challenge”, a viral internet sensation that involved individuals pouring a bucket of iced water over their heads to raise awareness and funds for charity. The challenge, which took place in 2014, raised over $100 million globally for ALS charities, significantly raising its profile and helping to fund research into potential treatments for the disease.


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