Tag: Tofersen

Antisense oligonucleotides (ASOs) have emerged as a significant therapeutic platform, with several drugs already approved by the FDA for the treatment of rare genetic disorders. This innovative technology provides a way to target the root cause of certain diseases at the RNA level, fundamentally changing how various genetic conditions are treated with ASO.

Biogen has set the wholesale acquisition cost for a single dose of Tofersen, also known as Qalsody, at $14,230. This steep price makes understanding **how much does a Tofersen injection cost** a critical concern for patients and their families seeking treatment for SOD1-ALS.

Every year, approximately 5,000 people in the U.S. receive a diagnosis of Amyotrophic Lateral Sclerosis (ALS), a progressive and fatal neurodegenerative disease. When faced with this news, many want to know, 'What is the best drug for ALS?' The answer is not a single drug, but a personalized treatment plan combining multiple medications and supportive therapies.

In the United States, approximately 2% of ALS cases are the genetic form known as SOD1-ALS [1.8.3]. For these patients, a targeted therapy approved in 2023 represents a major advancement. So, what is the newest drug for ALS, and how does it fit into the evolving landscape of treatment?

Tofersen, an antisense oligonucleotide marketed as Qalsody, was approved by the FDA for the treatment of SOD1-ALS, a rare and devastating form of amyotrophic lateral sclerosis. Like all potent medications, it is associated with a range of potential side effects, from common discomforts to serious neurological complications.

In a combined analysis of the VALOR trial and its open-label extension, starting tofersen earlier slowed the decline in clinical function, respiratory function, and muscle strength in SOD1-ALS patients compared to delayed initiation. This critical finding helps clarify **how effective is tofersen** beyond the initial trial results.

According to manufacturer Biogen, the wholesale acquisition cost for a single vial of Qalsody (tofersen) is $14,230. This puts the first-year list price for tofersen at nearly $200,000, creating significant discussion around the cost of this rare disease treatment.