Qalsody

Indications for Qalsody

Qalsody is used to treat amyotrophic lateral sclerosis (ALS) in adults who have a confirmed mutation in the superoxide dismutase 1 (SOD1) gene. ALS is a progressive neurodegenerative disease that affects the nerve cells in the brain and spinal cord responsible for controlling voluntary muscle movement.

This medication is intended only for patients with the SOD1-ALS genetic variant. Before starting treatment, a healthcare provider will perform a genetic test to confirm that the patient has this specific mutation.

Accelerated Approval

The Food and Drug Administration (FDA) granted accelerated approval for Qalsody based on its ability to reduce levels of neurofilament light chain (NfL) in the blood. NfL is a marker of nerve injury and neurodegeneration. Because it was approved under this pathway, the drug's continued approval may depend on the results of ongoing clinical trials to verify that it provides a definitive clinical benefit, such as slowing the loss of physical function.

Targeting the SOD1 Gene

Qalsody is designed for individuals with amyotrophic lateral sclerosis (ALS) who have a mutation in the superoxide dismutase 1 (SOD1) gene. In people with this mutation, the body produces a defective or toxic version of the SOD1 protein. Over time, this protein builds up and damages the motor neurons, which are the nerve cells responsible for controlling muscle movement.

How the Medication Works

Qalsody is a type of medicine called an antisense oligonucleotide (ASO). It works by interfering with the genetic instructions the body uses to create the SOD1 protein. The process generally follows these steps:

• Binding: After being injected into the spinal fluid, the medication binds to the specific messenger RNA (mRNA) that carries the instructions for making the SOD1 protein.
• Degradation: Once the medication attaches to the mRNA, it triggers the body to break down those instructions before they can be used to build the protein.
• Reduction: By destroying these instructions, the medication reduces the overall amount of toxic SOD1 protein produced in the body.

By lowering the levels of this toxic protein, the treatment aims to slow the progression of the disease. Clinical data has shown that treatment with Qalsody also leads to a reduction in neurofilament light chain, which is a biological marker in the blood that indicates nerve cell injury and death.

Common Side Effects

The most common side effects reported by people using Qalsody include:

• Pain in the back, arms, or legs
• Feeling tired or weak (fatigue)
• Muscle and joint pain
• Increased white blood cell count in the spinal fluid

Serious Side Effects

Serious side effects can occur and may require urgent medical care. Contact your healthcare provider immediately if you experience symptoms of the following conditions:

• Inflammation of the spinal cord or nerve roots: Symptoms may include numbness, tingling, pain, or weakness in the lower body, or changes in bowel or bladder control.
• Increased pressure in the brain: This condition (papilledema) can cause vision changes, blurred vision, double vision, or severe headaches.
• Inflammation of the brain or spinal cord lining: This includes meningitis or chemical meningitis. Symptoms may include fever, headache, stiff neck, nausea, vomiting, or sensitivity to light.

Serious Neurological Events

Treatment with Qalsody has been linked to serious inflammatory conditions of the nervous system. These include:

• Myelitis and Radiculitis: Inflammation of the spinal cord (myelitis) or nerve roots (radiculitis) can occur. Symptoms may include sensory changes, muscle weakness, or bowel and bladder dysfunction.
• Papilledema and Elevated Intracranial Pressure: Some patients may experience increased pressure inside the skull or swelling of the optic nerve (papilledema). This can lead to vision changes or headaches.
• Aseptic Meningitis: This is an inflammation of the membranes covering the brain and spinal cord that is not caused by an infection. Symptoms often include fever, headache, stiff neck, nausea, and vomiting.

Monitoring and Management

Healthcare providers typically perform regular evaluations to check for new or worsening neurological symptoms. If serious neurological events are suspected, treatment may be interrupted or discontinued based on the severity of the condition.

Lumbar Puncture Risks

Because Qalsody is administered via a lumbar puncture (spinal tap), there are risks associated with the procedure itself, such as headache, back pain, or infection at the injection site.

Pregnancy

There are no adequate studies on the use of Qalsody in pregnant women to determine if there is a drug-associated risk of birth defects, miscarriage, or other adverse outcomes. In animal studies, no negative effects on the development of the fetus were observed in mice or rabbits at doses higher than the recommended human dose.

Breastfeeding

It is not known whether Qalsody passes into human breast milk, how it affects milk production, or if it has any effect on a breastfed infant. Research in animals has shown that the medication is present in the milk of lactating mice. If you are breastfeeding or planning to breastfeed, you should discuss the potential risks and benefits with your healthcare provider to determine the best course of action.

Dosage Forms

Qalsody is a sterile, preservative-free solution for intrathecal injection. It is administered directly into the fluid-filled space around the spinal cord by a healthcare professional.

This medication is supplied in vials. The solution is clear and colorless to slightly yellow.

FDA Approval Status

The FDA granted accelerated approval to Qalsody in April 2023. It is the first treatment for amyotrophic lateral sclerosis (ALS) to be approved based on the reduction of a biomarker, specifically plasma neurofilament light chain (NfL), which indicates nerve injury. This medication is indicated for adults with ALS who have a mutation in the superoxide dismutase 1 (SOD1) gene.

Accelerated Approval Requirements

Under the accelerated approval pathway, the FDA requires the manufacturer to conduct a confirmatory clinical trial. This trial is intended to verify that the reduction in NfL levels leads to a meaningful clinical benefit for patients. The FDA may withdraw approval if the confirmatory trial fails to demonstrate such benefits.

Safety Warnings

The FDA-approved labeling for Qalsody includes warnings for serious neurologic adverse reactions. Healthcare providers are advised to monitor patients for symptoms of:

• Myelitis and radiculitis: Inflammation of the spinal cord or nerve roots.
• Papilledema and increased intracranial pressure: Swelling of the optic nerve and high pressure within the skull.
• Aseptic meningitis: Inflammation of the brain and spinal cord membranes not caused by infection.

There are currently no Boxed Warnings or Risk Evaluation and Mitigation Strategies (REMS) required for this medication.

Insurance and Out-of-Pocket Costs

The price of Qalsody can vary significantly based on your health insurance plan. Most insurance providers require prior authorization before they will cover this medication. Your out-of-pocket expenses, such as copays or coinsurance, will depend on your plan's specific benefits for specialty medications.

Administration Fees

Qalsody is administered through an intrathecal injection into the spinal canal by a healthcare professional. Because of this, the total cost of treatment includes not only the drug itself but also the professional fees for the medical procedure and the costs associated with the clinic or hospital visit.

Patient Assistance Programs

Financial assistance may be available through the drug manufacturer for eligible patients. These programs are designed to help reduce the cost of the medication for those with or without insurance. You can speak with your healthcare provider or contact the manufacturer directly to learn about support services and eligibility requirements.