The United States Food and Drug Administration (FDA) recently approved two new drugs for the treatment of amyotrophic lateral sclerosis (ALS). Biogen's tofersen and Ionis Pharmaceuticals' Qalsody are now available to patients suffering from this rare and debilitating disease. This article will explore the benefits and potential side effects of these drugs and what they mean for the future of ALS treatment.
Qalsody: A New Drug for SOD1ALS
Qalsody, developed by Ionis Pharmaceuticals, is an antisense oligonucleotide (ASO) that targets the superoxide dismutase 1 (SOD1) protein. This protein is found in about 2% of all ALS patients, and mutations in the SOD1 gene are a common cause of familial ALS. The drug works by reducing the levels of the SOD1 protein, which is believed to slow down the progression of the disease.
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The FDA granted Qalsody accelerated approval based on data from a phase 1/2 clinical trial involving 80 patients with SOD1ALS. The study found that patients receiving Qalsody experienced a slower decline in their ALS Functional Rating Scale (ALSFRS-R) scores than those receiving a placebo. However, the drug has not been proven to improve survival or quality of life for patients with ALS.
Tofersen: A Promising New Treatment for Certain Types of ALS
Tofersen, developed by Biogen, is a gene-silencing drug that targets the SOD1 gene. The drug works by reducing the production of the SOD1 protein, which is believed to contribute to the development and progression of ALS. Unlike Qalsody, tofersen is not limited to patients with the SOD1 mutation and can be used to treat a broader range of ALS patients.
The FDA granted tofersen pre-approval in August 2021 based on data from a phase 3 clinical trial involving 183 patients with a confirmed SOD1 mutation. The study found that patients receiving tofersen experienced a slower decline in their ALSFRS-R scores than those receiving a placebo. Tofersen has not been proven to improve survival or quality of life for patients with ALS.
Potential Side Effects
As with all drugs, Qalsody and tofersen may cause side effects. The most common side effects reported in clinical trials were injection site reactions and flu-like symptoms. Patients receiving tofersen may also experience liver damage, and patients receiving Qalsody may experience thrombocytopenia, a condition in which the blood has a lower than normal number of platelets.
The approval of Qalsody and tofersen represents a significant step forward in the treatment of ALS. While these drugs have not been proven to improve survival or quality of life for patients with ALS, they offer hope to those suffering from this devastating disease. As researchers continue to explore new treatments for ALS, the approval of these drugs highlights the importance of ongoing research in this field.
Keywords: ALS, Qalsody, tofersen, SOD1, gene-silencing, gene therapy
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