Qalsody: a novel antisense oligonucleotide (ASO) therapy

Qalsody (tofersen), a novel antisense oligonucleotide (ASO) therapy, has emerged as a beacon of hope in the fight against Amyotrophic lateral sclerosis (ALS). Qalsody received FDA approval in 2023 for the treatment of adults with ALS who have a mutation in the superoxide dismutase 1 (SOD1) gene. This significant milestone marked the first approved therapy specifically targeting a genetic cause of ALS.

FDA Approval and Manufacturing Details

On April 25, 2023, FDA-approved Qalsody based on the data from the Phase 3 VALOR clinical trial, which demonstrated a statistically significant slowing of the decline in pulmonary function, a key measure of ALS progression, compared to placebo. The study also showed a trend towards improved survival in the Qalsody group.

Biogen manufactures Qalsody at its state-of-the-art facility in Massachusetts, ensuring the highest standards of quality and consistency.

What Is Amyotrophic lateral sclerosis? 

Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, is a progressive neurodegenerative disorder that affects nerve cells in the spinal cord and brain. As the disease progresses, these nerve cells die, leading to muscle weakness, atrophy, and eventually paralysis. ALS is a devastating disease with no cure, and the average life expectancy after diagnosis is three to five years.

Symptoms of ALS:

• Muscle Weakness: One of the earliest symptoms is often weakness in a limb, affecting tasks like lifting or walking.

• Difficulty Speaking and Swallowing: ALS can impact the muscles used for speech and swallowing, leading to slurred speech and difficulty eating.

• Muscle Cramps and Twitching: Many individuals with ALS experience muscle cramps and twitching, known as fasciculations.
• Tripping and Difficulty Walking: ALS can affect the legs and lead to tripping or difficulty with balance and walking.

• Fatigue: Due to muscle weakness and the effort required for basic movements, individuals with ALS may experience fatigue more easily.

• Unintentional Weight Loss: Difficulty with eating and swallowing can contribute to unintentional weight loss.

• Stiffness and Joint Pain: Some people with ALS may experience stiffness and joint pain as a result of muscle changes.

• Respiratory Issues: As the disease progresses, muscles involved in breathing can be affected, leading to respiratory problems.

Indication and Mechanism of Action

Qalsody is specifically indicated for the treatment of adults with ALS who have a mutation in the SOD1 gene. SOD1 is an enzyme that helps protect cells from damage caused by free radicals. Mutations in the SOD1 gene can lead to the production of abnormal SOD1 protein, which can contribute to the development of ALS.

Qalsody works by targeting a specific messenger RNA (mRNA) molecule that encodes the abnormal SOD1 protein. By binding to this mRNA, Qalsody prevents the production of the abnormal SOD1 protein, thereby reducing its harmful effects on nerve cells.

Dosage Information 

• QALSODY is administered intrathecally at a recommended dose of 100 mg (15 mL) per administration. Treatment initiation involves three loading doses at 14-day intervals, followed by a maintenance dose every 28 days. 

• To administer, warm to room temperature, use within 4 hours, and remove approximately 10 mL of cerebrospinal fluid before injecting intrathecally over 1 to 3 minutes. It comes in a 100 mg/15 mL injection single-dose vial. No specific contraindications are mentioned.

• It is crucial to inform your doctor about all medications you are taking, including prescription and over-the-counter medications, herbal supplements, and vitamins, before starting Qalsody.

Usage in Specific Populations

• Pregnancy: The available data on QALSODY’s use in pregnant women is insufficient to assess the developmental risks related to major birth defects, miscarriage, or other adverse outcomes for both the mother and fetus.

• Lactation: Information regarding the presence of tofersen or its metabolites in human milk, the impact on the breastfed infant, or effects on milk production is not available.

• Pediatric Use: The safety and efficacy of QALSODY in pediatric patients have not been established.

• Geriatric Use: No adjustments to the dosage are required for elderly patients..

Warning And Precaution

QALSODY may lead to severe side effects. Contact your doctor promptly if you experience:

1. Inflammation of the spinal cord (Myelitis) and/or Nerve Roots (Radiculitis):

  These conditions may occur during QALSODY treatment. Consult your healthcare provider to understand the symptoms. Treatment interruption or discontinuation may be necessary.

2. Swelling of the Optic Nerve (Papilledema) and Increased Intracranial Pressure:

  These conditions may arise in patients treated with QALSODY. Discuss symptoms related to papilledema or elevated intracranial pressure with your healthcare provider.

3. Inflammation of the Brain Linings (Aseptic Meningitis):

  Aseptic meningitis may occur in patients treated with QALSODY. Consult your healthcare provider to learn about symptoms associated with aseptic meningitis.

Side Effects

The most common side effects associated with Qalsody include:

•  Peripheral neuropathy (nerve damage in the hands and feet)
•  Fatigue
•  Upper respiratory tract infection
•  Arthralgia
•  Cerebrospinal fluid white blood cell increased
•  Myalgia
•  Headache
•  Back pain
•  Neck pain

These side effects are usually mild and manageable. However, if you experience any severe or persistent side effects, contact your doctor immediately.

How To Buy Qalsody Online?

Obtaining Qalsody  has been streamlined through online platforms, especially for those residing in the US and Europe. To initiate the process, you can contact the SANSFRO team or other reputable firms specializing in importing medications from these regions.

For comprehensive details on the cost of Qalsody  in the Indian market and information regarding procurement, feel free to contact our dedicated Patient Support Team at ‎(+91) 93157 05373 or through email at help@sansfro.com. Our team is committed to providing accurate and up-to-date cost information for Qalsody  in the Indian market. Ensure a smooth experience accessing this essential medication by connecting with our trusted services.

Conclusion

Qalsody represents a significant breakthrough in the treatment of ALS, offering a targeted and effective approach that specifically addresses the genetic cause of the disease. Its FDA approval marks a turning point in the fight against ALS, providing renewed hope for patients and their families. With continued research and clinical experience, Qalsody has the potential to further enhance ALS management and improve patient outcomes.

Reference:

1. https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/215887s000lbl.pdf
2. https://www.drugs.com/qalsody.html