Spinraza (Nusinersen) Treatment for Spinal Muscular Atrophy (SMA)
Is my child acceptable for Nusinersen treatment?
Nusinersen (Spinraza) is the first medication approved for the treatment of spinal muscular atrophy (SMA), it is specifically indicated for the treatment of 5q SMA. 95% of SMA patients are diagnosed with 5q SMA, which is caused by a mutation in the survival motor neuron 1 (SMN1) gene on chromosome 5q, leading to a deficiency in the survival motor neuron (SMN) protein.
Indications for Nusinersen treatment in China:
- Patients who are diagnosed with 5q SMA.
- Pediatric and adult patients of all ages.
How Spinraza works?
Spinraza (nusinersen) is an antisense oligonucleotide medication that works to increase the production of the survival motor neuron (SMN) protein. The SMN protein is essential for the health and function of motor neurons, which control muscle movement. Spinraza specifically targets individuals with spinal muscular atrophy (SMA), a genetic disorder characterized by a deficiency of the SMN protein.
Here’s how Spinraza works:
- Mechanism of Action: Spinraza modifies the splicing of the pre-messenger RNA (mRNA) molecules derived from the survival motor neuron 2 (SMN2) gene. In SMA patients, the SMN2 gene produces insufficient amounts of functional SMN protein due to a specific splicing pattern. Spinraza helps correct this splicing pattern, resulting in increased production of full-length SMN protein.
- Intrathecal Administration: Spinraza is administered through a lumbar puncture procedure, also known as an intrathecal injection. The medication is delivered directly into the cerebrospinal fluid surrounding the spinal cord, allowing it to reach motor neurons in the central nervous system.
- Treatment Course: Spinraza treatment typically involves an initial loading phase followed by maintenance doses. During the loading phase, multiple doses are administered over a specified period to increase SMN protein levels. Maintenance doses are then given at regular intervals to sustain the therapeutic effect.
- Efficacy: By increasing the production of SMN protein, Spinraza aims to improve motor neuron function, delay disease progression, and potentially enhance muscle strength in individuals with SMA. Improved motor function outcomes have been observed in clinical trials, including increased survival, improved motor milestones achievement, and reduced need for respiratory intervention in some SMA patients.
How is Spinraza delivered?
Spinraza for 5q SMA involves intrathecal injections, meaning the medication is injected into the cerebrospinal fluid surrounding the spinal cord. It is approved for use in patients of all ages, including infants, children, and adults with 5q SMA. The specific dosing and treatment regimen may vary depending on factors such as age, weight, and disease severity. Treatment with nusinersen aims to improve motor function, increase survival, and slow down the progression of the disease.
What is Spinraza?
Spinraza (nusinersen) is a medication used for the treatment of spinal muscular atrophy (SMA). SMA is a genetic neuromuscular disorder characterized by the progressive weakening and loss of muscle function. Spinraza is specifically designed to increase production of the survival motor neuron (SMN) protein, which is deficient in individuals with SMA.
Spinraza is administered through intrathecal injection, which means it is injected directly into the cerebrospinal fluid surrounding the spinal cord. By increasing the production of SMN protein, Spinraza aims to improve motor function and slow down the progression of SMA.
Spinraza has shown positive results in clinical trials, particularly in infants and children with SMA, leading to its approval as a treatment option for SMA by various regulatory authorities worldwide. It is important to note that the use of Spinraza should be done under the supervision of healthcare professionals experienced in treating SMA.
Important safety information
Increased risk of bleeding complications has been observed after administration of similar medicines. Your healthcare provider should perform blood tests before you start treatment with SPINRAZA and before each dose to monitor for signs of these risks. Seek medical attention if unexpected bleeding occurs.
Increased risk of kidney damage, including potentially fatal acute inflammation of the kidney, has been observed after administration of similar medicines. Your healthcare provider should perform urine testing before you start treatment with SPINRAZA and before each dose to monitor for signs of this risk.
The most common side effects of SPINRAZA include lower respiratory infection, fever, constipation, headache, vomiting, back pain, and post-lumbar puncture syndrome.
These are not all of the possible side effects of SPINRAZA. Call your healthcare provider for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.
Before taking SPINRAZA, tell your healthcare provider if you are pregnant or plan to become pregnant.
Please note that individual results may vary based on several factors, including severity of disease, initiation of treatment, and duration of therapy.*
*Pivotal trials did not include adult patients with spinal muscular atrophy (SMA).
In a SPINRAZA® (nusinersen) pivotal trial, people with later-onset SMA showed meaningful improvements in overall motor function as measured by Hammersmith Functional Motor Scale—Expanded (HFMSE) compared to placebo at 15 months. Additionally, in a pivotal trial in people with early-onset SMA, a majority of infants were motor milestone responders at 13 months.
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