On April 25, 2023, the U.S. Food and Drug Administration (FDA) granted accelerated approval to Qalsody (tofersen), developed by Biogen, for treating amyotrophic lateral sclerosis (ALS) linked to mutations in the SOD1 gene. This groundbreaking therapy is the first to target a genetic cause of ALS, and we eagerly anticipate its approval in Japan.

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ALS, a devastating neurodegenerative disease, progressively weakens muscles throughout the body, impairing the ability to walk, eat, speak, laugh, and ultimately breathe.

For those living with ALS, the emotional and physical toll is immense, as the disease leaves cognitive functions intact, forcing patients to confront their deteriorating physical abilities head-on. Imagine the challenge of facing such a diagnosis, knowing that the journey ahead involves gradual loss of independence.

The introduction of Qalsody represents a beacon of hope for ALS patients, offering a glimpse of light in the darkness. However, with ALS patients typically having only two to five years of life expectancy post-diagnosis, the urgency for Qalsody's approval in Japan cannot be overstated. Every moment counts.

We implore you to lend your voice to this cause by signing our petition for the swift approval of Qalsody, supporting ALS patients and their families in Japan in their fight against this fearsome disease.

Together with the Japan Amyotrophic Lateral Sclerosis Association (JALSA), we will present these signatures to the Minister of Health, Labour, and Welfare. Your support not only fuels hope among ALS patients and their loved ones but also champions the advancement of ALS research and treatment.

Even after submitting signatures on November 20, 2023, we continue to seek your support through this ongoing petition.

The accelerated approval of Qalsody promises not only relief for ALS patients but also assurance for their families, potentially paving the way for further breakthroughs in ALS research and treatment for various forms of the disease.

Join us in our fight for a future where ALS can be fully conquered. Your support is invaluable.

*Supplementary Information: The FDA's accelerated approval of Qalsody highlights its potential to significantly impact ALS treatment, offering hope through scientific advancement.

Amyotrophic lateral sclerosis (ALS) is a progressive disease that causes muscle weakness and atrophy and patients with ALS lose the ability to move, speak, eat, and eventually breathe. Most of them die from respiratory failure in 2 to 5 years without respiratory apparatus since the muscles of the respiratory system gradually weaken.

There are approximately 10,000 patients with ALS in Japan, 350,000 patients in the whole world, and 10% of them are familial ALS. ALS due to a mutation in the superoxide dismutase 1 (SOD1) gene (SOD1-ALS) is the most common familial ALS in Japan, and more frequent in the Asian populations than the Western.

It is thought that a mutation in the SOD1 gene contribute to the death of motor neurons, leading to muscle weakness. Qalsody (tofersen) is an antisense oligonucleotide (ASO) and binds to SOD1 mRNA to reduce the amount of SOD1 protein. Clinical study showed that Qalsody-treated patients experienced a reduction in the amount of cerebrospinal SOD1 protein and plasma neurofilament light chain (NfL), a marker of neurodegeneration, compared to changes in placebo-treated patients. The U.S. Food and Drug Administration (FDA) approved Qalsody (tofersen) under accelerated approval based on these clinically significant results in patients with SOD1-ALS.

（Translated by Eiyu Nakayama)