Treatments
- Spinraza®. Spinraza is the first approved medicine for spinal muscular atrophy and the only one approved outside of the US.
- Zolgensma®. Zolgensma (AVXS-101) is the only approved gene therapy for spinal muscular atrophy. ...
- Experimental medicines in clinical development. ...
- Off-label uses of approved medicines. ...
- Compounds in early development. ...
Call 1-888-INFO-FDA (1-888-463-6332).
What is Spinraza used for?
Spinraza (nusinersen) is a new medication that was recently approved by the US Food and Drug Administration (FDA) for the treatment of all forms of SMA. Multiple research studies, called clinical trials, have demonstrated that the drug significantly slows the disease’s progression and, in many cases, improves the strength of individuals with SMA.
Is Spinraza approved in the US?
Spinraza is FDA-approved for all ages and types of SMA. Spinraza is given via an intrathecal (IT) injection, which is an injection into directly into the cerebrospinal fluid through the lower back. Individuals receive four “loading doses” within the first 2 months of treatment. Once those loading doses are completed, they receive a ...
What is the efficacy of Spinraza in the treatment of SMA?
Discover SPINRAZA: the first FDA-approved treatment for SMA 7+ years Safety and efficacy evaluated in the longest clinical trial in SMA to date 3x/year SPINRAZA is directly delivered to the central nervous system (CNS) where motor neuron loss begins. After 4 initial loading doses, SPINRAZA is given 3 times a year. From 3 days † to 80 years old ‡§
What is the recommended dosage for Spinraza?
Apr 11, 2021 · Spinraza is a brand-name prescription drug. It’s FDA-approved to treat spinal muscular atrophy (SMA) in adults and children. SMA is a rare genetic disorder * that affects the central nervous...
What does Spinraza do for 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.Jun 14, 2021
Is Spinraza a cure?
Classified as a rare disease, spinal muscular atrophy (SMA) is a genetic condition that chiefly affects motor neurons and is the leading genetic cause of death in infants worldwide. The disease is chronic, severe and currently has no cure.Sep 28, 2017
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.
Does Spinraza stop SMA?
Spinraza (nusinersen) continues to safely and effectively stop disease progression in children with spinal muscular atrophy (SMA) who were treated before 6 weeks old and symptom onset, according to updated data from the NURTURE trial.Jun 15, 2020
Is Spinraza FDA approved?
On December 23, the FDA announced that it has approved SpinrazaTM (nusinersen) to treat spinal muscular atrophy, making it the first-ever FDA-approved therapy for SMA.Sep 21, 2020
Who is eligible for Spinraza?
To establish eligibility for Spinraza, patients must undergo genetic testing, routine blood work, urine tests, cardiac tests, and X-rays. These can be performed during a clinic appointment.
What is the life expectancy of a child with SMA?
Infants with type 1 SMA usually die before their second birthday. Children with type 2 or type 3 SMA may live full lives depending on the severity of symptoms. People who develop SMA during adulthood (type 4) often remain active and enjoy a normal life expectancy.Apr 26, 2021
How much does Spinraza cost?
The company set Spinraza's price at $125,000 per injection, which adds up to $750,000 for the first year of treatment and $375,000 after that.
How effective is Spinraza?
At the time the recommended dose's use stated in SHINE, 36% of children who were on Spinraza treatment since ENDEAR were able to sit without support, 8% could stand with assistance, and 5% were able to walk with assistance.May 20, 2020
How is SMA treated?
The FDA has approved three medications to treat SMA: nusinersen (Spinraza), onasemnogene abeparvovec-xioi (Zolgensma) and risdiplam (Evrysdi). Both are forms of gene therapy that affect the genes involved in SMA.Nov 4, 2020
Is Spinraza an ASO?
As an antisense oligonucleotide (ASO), SPINRAZA targets an underlying cause of motor neuron loss by binding to a specific sequence in the SMN2 gene to increase the production of functional SMN protein in the central nervous system (CNS).
What is the difference between Spinraza and Zolgensma?
Zolgensma and Spinraza are both used to treat SMA, but Zolgensma is only approved for use in patients less than two years of age, whereas Spinraza is approved for use in pediatric and adult patients.Dec 21, 2020
What is Spinraza used for?
SPINRAZA is a drug for the treatment of spinal muscular atrophy (SMA). Spinal muscular atrophy is a rare genetic disease that affects mostly children and young adults. It is caused by a low level of protein in the motor neuron that are responsible for normal muscle functioning.
How often is Spinraza given?
This is known as an intrathecal injection. The first three doses are given every 14 days, followed by a fourth dose 30 days later. Injections are then administered every 120 days.
How many infants were in the Spinraza trial?
FDA approved SPINRAZA based primarily on one controlled clinical trial of 121 infants with SMA. The trial was conducted in the United States, Canada, France, Germany, Spain, and Japan. All 121 patients were considered in the assessment of SPINRAZA’s safety (safety population).
What is Snapshot for a drug?
The Snapshot is intended as one tool for consumers to use when discussing the risks and benefits of the drugs. Do not rely on Snapshots to make decisions regarding medical care. Always speak to your health provider about the risks and benefits of a drug. Refer to SPINRAZA Prescribing Information for complete information.
What is a comparator in clinical trials?
COMPARATOR: A previously available treatment or placebo used in clinical trials that is compared to the actual drug being tested. EFFICACY: How well the drug achieves the desired response when it is taken as described in a controlled clinical setting, such as during a clinical trial.
What is a placebo pill?
PLACEBO: An inactive substance or “sugar pill” that looks the same as, and is given the same way as, an active drug or treatment being tested. The effects of the active drug or treatment are compared to the effects of the placebo. SUBGROUP: A subset of the population studied in a clinical trial.
How often do you receive Spinraza?
Individuals receive four “loading doses” within the first 2 months of treatment. Once those loading doses are completed, they receive a maintenance dose every 4 months for the duration of the individual’s life. Spinraza is an antisense oligonucleotide.
What is Spinraza 2021?
It is an SMN-enhancing therapy that works by targeting the SMN2 gene, causing it to make more complete protein.
Can you take Spinraza after diagnosis?
Individuals who began Spinraza as soon after diagnosis as possible had better results than those who waited to begin treatment. Several sites across the U.S. are dosing individuals with Spinraza. A number of these sites can be found on our map locator tool.
How does Spinraza work?
Spinraza works by targeting a certain protein that helps nerve cells function correctly. For more information, see the “ How Spinraza works ” section below. Spinraza comes as a liquid solution inside of a vial. The drug is given as an intrathecal injection (an injection directly into the fluid in your spinal cord).
How long does Spinraza last?
In general, the life expectancy for a person with SMA type 1 that hasn’t been treated is less than 2 years. Spinraza is approved to treat SMA in adults and children. The drug received approval for this use from the Food and Drug Administration (FDA) in 2016.
What is the FDA approved for?
The Food and Drug Administration (FDA) approves prescription drugs such as Spinraza to treat certain conditions. Spinraza may also be used off-label for other conditions. Off-label drug use means using a drug for a purpose other than what it’s been approved for by the FDA.
How much Spinraza should I give myself?
You won’t give yourself injections of the drug. Spinraza comes in one strength: 12 milligrams (mg) per 5 milliliters (mL) of solution. To learn how often Spinraza is typically used, see the “ Spinraza dosage ” section below.
How long does it take to get Spinraza?
(This is an injection directly into the fluid in the spinal cord .) The injection takes about 1 to 3 minutes.
What is SMA in medical terms?
About SMA. SMA is a rare genetic disorder * that affects the central nervous system, which includes your spinal cord and brain. Specifically, SMA is a type of motor neuron disease. This means it results from problems with the neurons ( nerve cells) that control muscle movement.
What is SMA in a syringe?
Specifically, SMA is a type of motor neuron disease. This means it results from problems with the neurons ( nerve cells) that control muscle movement. Over time, SMA leads to muscle weakness and trouble walking, speaking, breathing, and swallowing.
What is Spinaza ASO?
SPINRAZA is an antisense oligonucleotide (ASO) designed to treat SMA caused by mutations in chromosome 5q that lead to SMN protein deficiency. Using in vitro assays and studies in transgenic animal models of SMA, SPINRAZA was shown to increase exon 7 inclusion in SMN2 messenger ribonucleic acid (mRNA) transcripts and production of full-length SMN protein.
Can Spinraza cause kidney damage?
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.
Is Spinraza a double blind study?
The efficacy of SPINRAZA was demonstrated in two double-blind, sham-procedure controlled clinical trials in symptomatic infantile-onset and later-onset SMA patients (Study 1 and Study 2) and was supported by open-label clinical trials conducted in presymptomatic and symptomatic SMA patients. The overall findings from these trials support the effectiveness of SPINRAZA across the range of SMA patients, and appear to support the early initiation of treatment with
Is Spinraza safe for pregnant women?
There are no adequate data on the developmental risk associated with the use of SPINRAZA in pregnant women. When nusinersen was administered by subcutaneous injection to mice throughout pregnancy and lactation, developmental toxicity (long-term neurobehavioral impairment) was observed at all doses tested (see Data). In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2 to 4% and 15 to 20%, respectively. The background risk of major birth defects and miscarriage for the indicated population is unknown.
Can you compare the adverse reactions of Spinraza to other drugs?
Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of SPINRAZA cannot be directly compared to rates in clinical trials of other drugs and may not reflect the rates observed in practice.
Can you bleed with Spinaza?
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.
Does Spinraza cause renal toxicity?
Inform patients and caregivers that SPINRAZA could cause renal toxicity. Inform patients and caregivers of the importance of obtaining urine testing at baseline and prior to each dose to monitor for signs of potential renal toxicity [see Warnings and Precautions (5.2)]. 49655-08
