-If a loading dose is delayed or missed, administer it as soon as possible, with at least 14-days between doses and continue dosing as prescribed. -If a maintenance dose is delayed or missed, administer it as soon as possible and continue dosing every 4 months. Use: For the treatment of spinal muscular atrophy (SMA)
Full Answer
How long do nusinersen treatments for SMA last?
The 42 nusinersen-treated participants were adults with SMA type 2 or type 3 (table 1) who were treated for a range of 3–24 months (mean treatment duration 12.5 months). The mean age at treatment initiation was 33.7 years (range 17.7–66.1 years), 57% were male, and 62% had 3 copies of the SMN2gene.
What is nusinersen used to treat?
Nusinersen belongs to a group of medicines known as antisense oligonucleotides. It’s only used to treat 5q spinal muscular atrophy (5q SMA). People with SMA don’t have enough of a protein called ‘survival motor neuron’ (SMN) protein. This protein is essential for the nerve cells, known as motor neurons that help control muscles.
How often do you take nusinersen for muscle atrophy?
Nusinersen dosing information. Usual Adult Dose for Spinal Muscular Atrophy: Loading dose: 12 mg intrathecally every 14 days for 3 doses, then 12 mg intrathecally 30 days after the 3rd dose. Maintenance dose: 12 mg intrathecally once every 4 months.
What is the generic name for nusinersen?
Nusinersen (injection) Generic Name: nusinersen (injection) (NUE si NER sen) Brand Name: Spinraza. Medically reviewed by Drugs.com on Jul 26, 2018 – Written by Cerner Multum. Nusinersen works by helping the body produce a protein essential to the function of nerves that control muscle movement.
How effective is nusinersen?
Results from the probabilistic sensitivity analyses found that nusinersen had a zero likelihood of achieving a cost-effective ratio of less than $500,000 per QALY gained.
How often is nusinersen administered?
The first three loading doses should be administered at 14-day intervals. The 4th loading dose should be administered 30 days after the 3rd dose. A maintenance dose should be administered once every 4 months thereafter.
Does nusinersen cure SMA?
Spinraza (nusinersen), marketed by Biogen, was the first FDA-approved therapy to treat SMA. It is an SMN-enhancing therapy that works by targeting the SMN2 gene, causing it to make more complete protein.
Is nusinersen the same as Spinraza?
What Is Spinraza? Spinraza (nusinersen) injection is a survival motor neuron-2 (SMN2)- directed antisense oligonucleotide indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.
How effective is Spinraza?
An analysis of data from the NURTURE study (n=25) shows 92 percent of patients who initiated SPINRAZA treatment as pre-symptomatic infants maintained the ability to swallow after a median of 3.8 years.
How is nusinersen administered?
SPINRAZA is an intrathecal injection administered by, or under the direction of, healthcare professionals experienced in performing lumbar punctures. The recommended dosage is 12 mg (5 mL) per administration. Treatment with SPINRAZA begins with 4 loading doses. The first 3 doses are administered at 14-day intervals.
How quickly does Spinraza work?
Spinraza starts working to treat SMA as soon as it's injected. In clinical studies, more than half of the people who were given Spinraza showed a decrease in their SMA symptoms within 15 months of their first dose.
What is the life expectancy of a child with SMA?
The life expectancy of patients with spinal muscular atrophy (SMA) type I is generally considered to be less than 2 years.
How much is nusinersen?
Nusinersen list price in the USA is US$125,000 per injection which puts the treatment cost at US$750,000 in the first year and US$375,000 annually after that. According to The New York Times, this places nusinersen "among the most expensive drugs in the world".
Is Risdiplam better than nusinersen?
Overall, it was established that Risdiplam leads to higher survivability rates and had better performance on motor milestone response of the infants of over 85 percent if the treatment is administered early. As such, it is a treatment that gets a higher recommendation as compared to Nusinersen.
How does nusinersen Spinraza work?
Spinraza is a synthetic anti-sense oligonucleotide (a type of genetic material) that enables the SMN2 gene to produce full length protein, which is able to work normally. This replaces the missing protein, thereby relieving the symptoms of the disease.
How long do you take Spinraza?
The first 3 loading doses of SPINRAZA are administered every 14 days. The fourth and final loading dose is administered 30 days later. Maintenance doses are then administered every 4 months thereafter.
Usual Adult Dose for Spinal Muscular Atrophy
Loading dose: 12 mg intrathecally every 14 days for 3 doses, then 12 mg intrathecally 30 days after the 3rd dose Maintenance dose: 12 mg intrathecally once every 4 months Comments: -For administration, consider sedation; consider ultrasound or other imaging techniques to guide therapy. -Prior to administration, remove 5 mL from cerebrospinal fluid; administer as an intrathecal bolus injection over 1 to 3 minutes using a spinal anesthesia needle. -If a loading dose is delayed or missed, administer it as soon as possible, with at least 14-days between doses and continue dosing as prescribed. -If a maintenance dose is delayed or missed, administer it as soon as possible and continue dosing every 4 months. Use: For the treatment of spinal muscular atrophy (SMA).
Usual Pediatric Dose for Spinal Muscular Atrophy
Loading dose: 12 mg intrathecally every 14 days for 3 doses, then 12 mg intrathecally 30 days after the 3rd dose Maintenance dose: 12 mg intrathecally once every 4 months Comments: -For administration, consider sedation; consider ultrasound or other imaging techniques to guide therapy, especially in younger patients. -Prior to administration, remove 5 mL from cerebrospinal fluid; administer as an intrathecal bolus injection over 1 to 3 minutes using a spinal anesthesia needle -If a loading dose is delayed or missed, administer it as soon as possible, with at least 14-days between doses and continue dosing as prescribed. -If a maintenance dose is delayed or missed, administer it as soon as possible and continue dosing every 4 months. Use: For the treatment of spinal muscular atrophy (SMA).
Renal Dose Adjustments
Use with caution; for urinary protein concentration greater than 0.2 g/L consider repeat testing and further evaluation -Conduct quantitative spot urine protein testing (preferably with first morning urine specimen) at baseline and prior to each dose
Precautions
CONTRAINDICATIONS: -None Consult WARNINGS section for additional precautions.
Other Comments
Administration advice: -For intrathecal use only -This drug should be administered by, or under the direction of, a healthcare professional experienced in performing lumbar punctures. -Consider sedation as indicated by the clinical condition of the patient. -Consider ultrasound or other imaging techniques to guide intrathecal administration, particularly in younger patients. -Prior to administration, remove 5 mL of cerebrospinal fluid (CSF). -Administer as an intrathecal bolus injection over 1 to 3 minutes using a spinal anesthesia needle. -Do not administer in areas of the skin where there are signs of infection or inflammation. Storage requirements: -Protect from light -Store in the original carton in the refrigerator until time of use; do not freeze. -May be stored in original carton at or below 30C (86F) for up to 14 days -Prior to administration, unopened vials may be removed from and returned to the refrigerator, if necessary.
Further information
Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.
How long does it take to get Nusinersen?
A healthcare provider will give you this injection. The nusinersen injection takes about 1 to 3 minutes to complete. You may be given a sedative to help you relax and keep you comfortable during the injection.
How many doses of Nusinersen are given?
Nusinersen is usually given in a series of 4 "loading dose" injections, followed by "maintenance" injections. The first 3 loading doses are usually given 2 weeks apart, followed by a 4th loading dose given 1 month later.
What is Nusinersen used for?
Nusinersen is used to treat spinal muscular atrophy in adults, children, and infants. Nusinersen may also be used for purposes not listed in this medication guide.
What are the side effects of Nusinersen?
Nusinersen side effects. Get emergency medical help if you have signs of an allergic reaction: hives; difficult breathing; swelling of your face, lips, tongue, or throat. easy bruising, unusual bleeding (nose, mouth, vagina, or rectum), purple or red pinpoint spots under your skin;
Does Nusinersen cause rash?
a red skin rash; or. kidney problems--foamy urine; red or brown colored urine; swelling in your face, stomach, hands, or feet. Nusinersen may affect growth in children. Tell your doctor if your child is not growing at a normal rate while using nusinersen. Common side effects may include: headache; back pain;
Is Nusinersen a type 1 or type 2 drug?
Nusinersen represents the first efficacious marked approved drug in type 1 and type 2 SMA. Different knowledge gaps, such as results in older patients, in patients with permanent ventilation, in patients with neonatal forms, or in patients after spinal fusion, still need to be addressed.
Does Nusinersen prolong SMA?
Nusinersen treatment prolongs survival of patients with type 1 SMA and allows motor milestone acquisition. Patients with type 2 SMA also show progress on different motor scales after nusinersen treatment. Nusinersen was recently approved by the European Medicines Agency and the US Food and Drug Administration; it is now reimbursed in several ...
How long does Spinraza last?
Part C will test adults on Spinraza for more than one year, with these patients getting a single 50 mg dose four months after their most recent treatment, followed by 28 mg maintenance doses four and eight months later. Researchers expect to finish the DEVOTE study in July 2023.
Where is Spinraza administered?
Spinraza is administered as an intrathecal injection, or an injection directly into the spinal canal. Treatment must take place in hospitals or at centers whose staff are trained in performing lumbar punctures. The most common side effects of Spinraza are respiratory infections and constipation.
What is the function of Spinraza?
Spinraza contains an antisense oligonucleotide that allows the body to produce more of a protein called survival motor neuron (SMN). SMN is essential for the health of motor neurons, nerve cells that control the movement of muscles. People with SMA do not produce enough of this protein. Everyone has two genes that encode for ...
What is Spinraza used for?
Spinraza became the first therapy approved by the U.S. Food and Drug Administration (FDA) to treat SMA type 1, type 2, and type 3 — covering both infants and adults — in December 2016, under a priority review program for rare diseases.
How old are children when they get Zolgensma?
The first group will include 40 children up to nine months old, who have two copies of the SMN2 gene (likely to develop type 1 SMA) and were treated with Zolgensma before or at six months of age.
When will the SMA end?
It is due to conclude in August 2023. Interim findings released in May 2020 and reaching out for some participants to 6.5 years of treatment, showed sustained benefits — from motor skill gains to disease stabilization — across a range of SMA patients who were at ages now ranging from toddlers to young adults.
Is Spinraza safe for infants?
Its results showed that Spinraza had an acceptable safety and tolerability profile. It also led to encouraging signs of clinical efficacy, in the form of slowed progression, in infants with SMA type 1, the most severe form of the disease.
