Spinraza

About Spinraza

• Spinraza got  Food and Drug Administration (FDA) approval in 2016. It is classified as an antisense oligonucleotide medication and is used in the treatment of spinal muscular atrophy (SMA). 
• Spinal muscular atrophy (SMA) is a type of motor neuron disease affecting both children and adults. It holds the distinction of being the first drug to receive approval from the US FDA for this particular patient group.
• SMA, an inherited condition, leads to progressive skeletal muscle weakness and degeneration over time. The primary cause of SMA is often attributed to mutations within the SMN1 gene, resulting in insufficient functional SMN protein production necessary for the health and operation of motor neurons.
• In addition to SMN1, there exists another gene known as SMN2, which also generates SMN protein. However, only a small portion of the protein produced by SMN2 is fully functional. This is primarily due to the tendency for exon 7, a specific part of the gene, to be frequently excluded during protein synthesis, resulting in an incomplete and ineffective protein.
• Spinraza operates by correcting the SMN2-produced protein, ensuring it functions as intended. This is achieved by ensuring that the crucial missing information from exon 7 is included in the protein’s structure.
• Treatment involves healthcare professional supervision, potentially requiring regular injections or infusions. Consult a healthcare provider for specific guidance.

Strength: 

Injection: 12mg/5mL (2.4mg/mL) in a single-dose vial. 

Recommended Dosage:

Spinraza is administered through a procedure called intrathecal injection, and it should only be carried out by healthcare professionals who have the necessary expertise in performing lumbar punctures. This is an important precaution to ensure the safe and effective delivery of the medication into the cerebrospinal fluid that surrounds the spinal cord. Intrathecal administration allows Spinraza to directly target the motor neurons in the spinal cord, which is crucial for its effectiveness in treating spinal muscular atrophy (SMA). 

• To start Spinraza treatment, administer a 12mg (5 mL) dose four times initially. 
• The first three doses are given at 14-day intervals, followed by the fourth dose 30 days after the third one. 
• After your 4 loading doses, you will receive a maintenance dose of Spinraza every 4 months (3 times per year).

Important:

Before starting Spinraza treatment, it’s crucial to understand the associated risks and side effects. This drug is administered through intrathecal injection, which carries certain risks. The injection must not be administered in areas of the skin where there are signs of infection or inflammation. Ensure you have an experienced healthcare team for the procedure and follow your doctor’s instructions diligently. Report any side effects promptly, especially signs like headache, fever, or back pain related to the injection. Adhere to post-injection care instructions, closely monitor any changes in symptoms or behavior in children, and discuss concerns with your doctor.

Warnings & Precautions

• Prior to initiating Spinraza treatment and before each dose, your healthcare provider will conduct blood tests to monitor for signs of these risks. In the event of unexpected bleeding, seek immediate medical attention. 
• Do not take this treatment if you are allergic to Nusinersen or any of its active ingredients. 
• There is an increased risk of kidney damage, including potentially severe kidney inflammation, which has been noted with similar medicines. 
• Your healthcare provider will perform urine testing before starting Spinraza and before each dose to monitor for any indications of kidney damage. It is important to heed these warnings and precautions for your safety.
• Spinraza is not recommended for pregnant and breastfeeding women unless advised by a doctor due to unknown safety and efficacy in these populations.
• Spinraza  should be used cautiously in patients taking other medications that may interact with it. Please consult your healthcare provider for guidance on potential interactions and medication management.

Common Spinraza  Side Effects:

• Lower respiratory infection 
• Constipation
• Pyrexia
• Headache
• Vomiting
• Back pain

Use in Specific Population

• Spinraza has been proven to be safe and effective for pediatric patients ranging from newborns to 17 years of age. However, clinical studies of Spinraza did not encompass a significant number of individuals aged 65 and older to ascertain whether their response to the treatment differs from that of younger subjects.

Storage and Handling

• Store Spinraza in a refrigerator between 2°C to 8°C (36°F to 46°F) within its original carton to shield it from light. Avoid freezing. Keep Spinraza in its original carton to protect it from light until it is ready to be used. 
• If refrigeration is unavailable, it can be stored in its original carton, shielded from light, at or below 30°C (86°F) for a maximum of 14 days. 
• Before administration, unopened vials of Spinraza can be taken out of and returned to the refrigerator as needed. 
• However, if removed from the original carton, the total cumulative time outside of refrigeration should not exceed 30 hours at a temperature not exceeding 25°C (77°F).