Qalsody is the fourth drug approved by the FDA for the treatment of ALS and the first drug to treat hereditary ALS. So what are the symptoms of ALS?

What is amyotrophic lateral sclerosis?

Amyotrophic lateral sclerosis (ALS) is also called motor neuropathy (MND), which is commonly known as ALS. It is caused by damage to the upper and lower motor nerves, leading to gradual weakness and atrophy of the muscles of the bulb (the so-called bulb refers to the part of the muscles controlled by the medulla oblongata), limbs, trunk, chest and abdomen (1). The cause of the disease is still unknown. 20% of cases may be related to genetic and gene defects. The early symptoms are not obvious. Patients may only feel weakness, muscle twitching, easy fatigue and other symptoms, which gradually progress to systemic muscle atrophy and difficulty swallowing until respiratory failure.

ALS is believed to be related to multiple genes. Among them, patients carrying SOD1 gene mutations account for 2% of ALS patients. Most patients carrying this gene have a rapid progression of the disease and survive less than one year after onset. There is no specific drug for ALS, but the emergence of Qalsody has alleviated this phenomenon. What is Qalsody? How does it work?

What is Qalsody?

Qalsody was approved by the US FDA for marketing in the United States on April 25, 2023 for the treatment of adult amyotrophic lateral sclerosis (ALS) with superoxide dismutase 1 (SOD1) gene mutations, and has been granted accelerated approval and orphan drug designation by the FDA.

Qalsody is an antisense oligonucleotide that binds to SOD1 mRNA to cause degradation of SOD1 mRNA, thereby reducing SOD1 protein synthesis to achieve the purpose of treating the disease.

The above is the basic information of Qalsody.