What is Exondys 51 used to treat?
EXONDYS 51 is used to treat Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation in the dystrophin gene that can be treated by skipping exon 51. EXONDYS 51 was approved under accelerated approval. Accelerated approval allows for drugs to be approved based on a marker...
What is accelerated approval for Exondys 51?
Accelerated approval allows for drugs to be approved based on a marker that is considered reasonably likely to predict a clinical benefit. EXONDYS 51 treatment increased the marker, dystrophin, in skeletal muscle in some patients. Verification of a clinical benefit may be needed for EXONDYS 51 to continue to be approved.
Can Exondys 51 help Duchenne muscular dystrophy?
Bringing possibility to Duchenne. EXONDYS 51 is the first FDA-approved Duchenne muscular dystrophy treatment for patients who have a confirmed genetic mutation in the dystrophin gene that can be treated by skipping exon 51. In some patients, it helps the body make a shorter form of the dystrophin protein.
What is exon 51 used for in Duchenne?
EXONDYS 51 is the first FDA-approved Duchenne muscular dystrophy treatment for patients who have a confirmed genetic mutation in the dystrophin gene that can be treated by skipping exon 51. In some patients, it helps the body make a shorter form of the dystrophin protein.
How do you get Exondys 51?
GETTING YOUR PATIENTS STARTED ON EXONDYS 51 (ETEPLIRSEN).Download the SareptAssist START Form. Download Form.With your patient, fill out the START Form and fax it to 1-800-621-5203.A dedicated SareptAssist Case Manager will contact your patient to welcome them to the program and explain how they can help.
How much is Exondys?
Exondys 51 (eteplirsen) – between $750,000 and $1.5m a year EXONDYS 51® is approved for the treatment of Duchenne muscular dystrophy.
Is EXONDYS 51 effective?
Does Exondys 51 cure Duchenne muscular dystrophy? No, Exondys 51 doesn't cure Duchenne muscular dystrophy (DMD). Currently, there isn't a cure for DMD or any other type of muscular dystrophy. People with DMD lack a specific protein called dystrophin that causes muscle weakness and loss over time.
How does EXONDYS 51 work?
Treatment with EXONDYS 51 (eteplirsen). A lack of dystrophin causes muscle cells to become damaged and weakened over time. EXONDYS 51 uses exon-skipping technology to allow the body to make a shorter form of the dystrophin protein in some patients by skipping over a specific exon on the dystrophin gene.
What is the name of the gene that is used to treat Duchenne muscular dystrophy?from exondys51hcp.com
EXONDYS 51 (eteplirsen) is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. This indication is approved under accelerated approval based on an increase in dystrophin in skeletal muscle observed in some patients treated with EXONDYS 51. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials.
What are the reactions to oxydyl 51?from exondys51hcp.com
Hypersensitivity reactions, including rash and urticaria, pyrexia, flushing, cough, dyspnea, bronchospasm, and hypotension, have occurred in patients who were treated with EXONDYS 51. If a hypersensitivity reaction occurs, institute appropriate medical treatment and consider slowing the infusion or interrupting the EXONDYS 51 therapy.
What is the copay assistance program?from exondys51hcp.com
The Co-Pay Assistance Program was created for eligible individuals with commercial health insurance in the United States who are prescribed EXONDYS 51 therapy. This program may help with some out-of-pocket costs related to receiving therapy, such as co-pays, co-insurance, and deductibles.
What is a PAP program?from sarepta.com
The PAP program can help patients, who are uninsured or rendered uninsured and meet certain eligibility requirements, obtain access to drug. You may be responsible for additional costs associated with administration of the drug. Your Case Manager can provide information on your eligibility, as well as help facilitate program enrollment and answer questions you may have on the application form if you are eligible.
What is Sareptassist case manager?from sarepta.com
When you contact SareptAssist, you will be connected to a dedicated Case Manager with experience in rare diseases, including knowledge of insurance plans and healthcare networks. Your Case Manager will work closely with you to provide one-on-one, personalized support as you navigate the complexities of starting and staying on therapy.
What does a case manager do after insurance is confirmed?from sarepta.com
Once your insurance benefits have been confirmed, your Case Manager will work closely with the providing pharmacy (specialty or hospital pharmacy) to facilitate treatment access and coordinate drug delivery to your treatment location.
Why is the Duchenne family important?from sarepta.com
It is our fundamental goal to empower you with information and support that may help manage the stresses of starting a new therapy so you can focus on what's important — life with your child."
What is Exondys 51 used for?
Exondys 51 (eteplirsen) is a member of the miscellaneous uncategorized agents drug class and is commonly used for Duchenne Muscular Dystrophy.
What is the number to contact for Exondys 51?
Exondys 51 Patient Co-Pay Assistance Program: Eligible commercially insured patients may receive assistance with some out-of-pocket costs related to receiving therapy; for additional information contact the program at 888-727-3782.
How much does a free drug card save?
The free Drugs.com Discount Card works like a coupon and can save you up to 80% or more off the cost of prescription medicines, over-the-counter drugs and pet prescriptions.
Is Exondys 51 a generic?
Exondys 51 is available as a brand name drug only, a generic version is not yet available. For more information, read about generic Exondys 51 availability . This Exondys 51 price guide is based on using the Drugs.com discount card which is accepted at most U.S. pharmacies.
What is the purpose of Exon 51?
EXONDYS 51 is used to treat Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation in the dystrophin gene that can be treated by skipping exon 51. EXONDYS 51 was approved under accelerated approval. Accelerated approval allows for drugs to be approved based on a marker that is considered reasonably likely to predict a clinical benefit. EXONDYS 51 treatment increased the marker, dystrophin, in skeletal muscle in some patients. Verification of a clinical benefit may be needed for EXONDYS 51 to continue to be approved.
What are the side effects of elongys 51?
In patients who received EXONDYS 51for up to 4 years in clinical studies, the following side effects were reported in at least 10% of patients and occurred more often than at the same dose of EXONDYS 51: vomiting, bruising, scratches, joint pain, rash, IV site pain, and infections of the upper airways.
How to report side effects of a drug?
You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. You may also report side effects to Sarepta Therapeutics at 1-888-SAREPTA (1-888-727-3782).
Is Duchenne muscular dystrophy rare?
Duchenne muscular dystrophy is a rare disease— but you’re not facing it alone. Many families have been in your shoes, and over the years, they’ve created a strong community of patient advocates, educators, and service providers.
What is eteplirsen?from drugs.com
Eteplirsen works by helping the body produce a protein called dystrophin . Dystrophin is a protein that helps stabilize the structure of muscle tissue and is necessary for proper muscle development and function. Without dystrophin, muscle fibers can become weak and easily damaged. This can affect voluntary movement such as walking.
What happens if I overdose?from drugs.com
Seek emergency medical attention or call the Poison Help line at 1-800-222-1222.
Will I need to use this drug long term?from medicalnewstoday.com
Exondys 51 is meant to be used as a long-term treatment. If you and your doctor determine that Exondys 51 is safe and effective for you, you’ll likely use it long term.
What is exon 51?from drugs.com
An exon is a part of a gene. In this case, exon 51 refers to a specific exon in the DMD gene. (For more information on how Exondys 51 works, including an explanation of exon skipping, see the “ How Exondys 51 works ” section above.) Before receiving Exondys 51, your doctor will order a genetic test.
What is the most common form of muscular dystrophy?from medicalnewstoday.com
DMD is the most common form of muscular dystrophy. This term refers to a group of genetic (inherited) disorders that cause loss of muscle mass over time. A person with a family history of muscular dystrophy has a greater risk for developing this condition.
How does Duchenne muscular dystrophy occur?from en.wikipedia.org
Duchenne muscular dystrophy is caused when a mutation in the DMD gene changes the DMD RNA so that it no longer codes for functional dystrophin protein, usually due to a mutation that alters the reading frame of the RNA downstream of the mutation. If an exon with an appropriate number of bases lies near the mutation, by removing that exon the downstream reading frame can be corrected and production of partially functional dystrophin can be restored. This is the general strategy used for designing exon-skipping oligos for DMD; as there are 79 exons in the longest splice form of the dystrophin transcript, many different oligos are needed to address the range of mutations present in the population of people with DMD.
What to do if you miss an appointment for Exondys 51?from medicalnewstoday.com
If you miss an appointment for your Exondys 51 dose, call your doctor as soon as you can. They’ll work to reschedule your infusion as soon as possible. They’ll also give you instructions on rescheduling future doses.
LET SAREPTASSIST CONNECT YOU WITH A REIMBURSEMENT SPECIALIST
If you have questions about reimbursement, just give SareptAssist a call at 1-888-SAREPTA (1-888-727-3782). Case Managers are available to help Monday – Friday 8:30am – 6:30pm ET.
INDICATION
EXONDYS 51 (eteplirsen) is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. This indication is approved under accelerated approval based on an increase in dystrophin in skeletal muscle observed in some patients treated with EXONDYS 51.
IMPORTANT SAFETY INFORMATION
Hypersensitivity reactions, including rash and urticaria, pyrexia, flushing, cough, dyspnea, bronchospasm, and hypotension, have occurred in patients who were treated with EXONDYS 51. If a hypersensitivity reaction occurs, institute appropriate medical treatment and consider slowing the infusion or interrupting the EXONDYS 51 therapy.
Will I need to use this drug long term?
Exondys 51 is meant to be used as a long-term treatment. If you and your doctor determine that Exondys 51 is safe and effective for you, you’ll likely use it long term.
What to do if you miss an appointment for Exondys 51?
If you miss an appointment for your Exondys 51 dose, call your doctor as soon as you can. They’ll work to reschedule your infusion as soon as possible. They’ll also give you instructions on rescheduling future doses.
How long does it take for Exondys 51 to work?
In clinical studies, the dystrophin levels of people using Exondys 51 weren’t measured until after 48 weeks .
How does exondys 51 work?
The drug works by helping the body “skip” exon 51 while reading the genetic instructions for making dystrophin. †. In the process, the missing exons are also skipped.
What is the active drug in Exondys 51?
Exondys 51 is available in one strength: 50 milligrams per milliliter (mg/mL). The active drug in Exondys 51 is eteplirsen. Eteplirsen belongs to a class of medications known as antisense oligonucleotides.
What is Exondys 51 used for?
Exondys 51 uses. The Food and Drug Administration (FDA) approves prescription drugs such as Exondys 51 to treat certain conditions. Exondys 51 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 Exondys 51 is given?
Exondys 51 is given in a dose of 30 milligrams (mg) per kilogram (kg) of body weight. One kg is equal to about 2.2 pounds (lb). As an example, a person weighing 50 kg (about 110 lb) would receive an Exondys 51 dose of 1,500 mg. The dose is given once weekly over the course of 35 to 60 minutes.
