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About EXONDYS 51

Weekly infusions of EXONDYS 51 (eteplirsen) have been shown to help the body make a shorter form of the dystrophin protein in some boys.

EXONDYS 51 packaging and vials

Understanding EXONDYS 51.

 

EXONDYS 51 is a treatment for Duchenne muscular dystrophy. It uses a technology called exon skipping to help the body make a shorter form of the dystrophin protein. EXONDYS 51 is given in a once-weekly infusion.

 

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 that has not been proven. 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.

Meet Dalton, age 22. Deletion of exons 48-50. Meet Dalton, age 22. Deletion of exons 48-50. Profile view of EXONDYS 51 patient Dalton, age 22, in his wheelchair, smiling and turned toward camera

Results from EXONDYS 51 clinical studies.

Researchers conducted clinical trials of EXONDYS 51 to review different aspects of the medicine, including whether it triggers skipping of exon 51 on the dystrophin gene. They also analyzed the amount of dystrophin produced, along with its safety profile. EXONDYS 51 was studied in boys who had a confirmed mutation in the dystrophin gene that could be treated by skipping exon 51. Learn what this means

Designed to skip exon 51.

Three clinical studies looked at whether exon skipping occurred on the dystrophin gene of boys treated with EXONDYS 51. In clinical studies, exon skipping occurred in all 36 evaluated study participants.

36/36 - Number of study participants with a muscle biopsy confirming exon skipping in the dystrophin gene.
36/36 - Number of study participants with a muscle biopsy confirming exon skipping in the dystrophin gene.
Exon skipping occurred in all 36 evaluated study participants

Dystrophin levels increased in some clinical trial participants.

A study of 12 boys with Duchenne muscular dystrophy compared the level of dystrophin in their bodies before the first infusion of EXONDYS 51 with the level of dystrophin after 48 weeks of treatment with EXONDYS 51. Weekly infusions of EXONDYS 51 helped the body make a shorter form of the dystrophin protein.

 EXONDYS 51 patient Liam, age 19, standing with arms crossed looking at camera
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*After 48 weeks, dystrophin levels were an average of 2.8x higher than the level before treatment began, but still remained at a level much lower than that produced by people without Duchenne. In another study of boys treated for 180 weeks, dystrophin levels were an average of 0.93% of the level produced by people without Duchenne. Difference in dystrophin = Week 48 % normal dystrophin/baseline % normal dystrophin.

 EXONDYS 51 patient Liam, age 19, standing with arms crossed looking at camera

What do the results tell us?

Participants showed an average dystrophin increase of 2.8x the baseline level, but levels still remained much lower than the level of people without Duchenne.

 

After 48 weeks the average dystrophin level was 0.44% of normal, but levels still remained much lower than the level of people without Duchenne.

 

Baseline dystrophin level was 0.16% of normal

 

Additional study results.

In a separate study of 12 patients, the average dystrophin level for participants after 180 weeks** was 0.93% of normal, but levels still remained much lower than the level of people without Duchenne. ** Baseline levels were not available for all participants.
In a separate study of 12 patients, the average dystrophin level for participants after 180 weeks** was 0.93% of normal, but levels still remained much lower than the level of people without Duchenne. ** Baseline levels were not available for all participants.

** Baseline levels were not available for all participants.

Graysen
Graysen

What about risks? Researchers assessed the risks and tolerability of EXONDYS 51. As with any medication, you should discuss the risks and side effects associated with EXONDYS 51 with your doctor.

Review Risks and Side Effects

WHAT IS EXONDYS 51® (eteplirsen)?

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 that has not been proven. 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.

IMPORTANT RISK INFORMATION

Allergic reactions, including rash, hives, fever, flushing, cough, difficulty breathing, wheezing, and low blood pressure have occurred in patients who were treated with EXONDYS 51. Seek immediate medical care if signs and symptoms of allergic reactions occur.

Side effects that happened at least 25% more often in 8 patients treated with EXONDYS 51 by intravenous infusion than in 4 patients treated with an inactive intravenous infusion were problems with balance (38%, 0%), vomiting (38%, 0%), and skin irritation (25%, 0%). The most common side effects were problems with balance and vomiting.

In patients who received EXONDYS 51 for 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.

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).

READ MORE

WHAT IS EXONDYS 51® (eteplirsen)?

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 that has not been proven. 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.

IMPORTANT RISK INFORMATION

Allergic reactions, including rash, hives, fever, flushing, cough, difficulty breathing, wheezing, and low blood pressure have occurred in patients who were treated with EXONDYS 51. Seek immediate medical care if signs and symptoms of allergic reactions occur.

Side effects that happened at least 25% more often in 8 patients treated with EXONDYS 51 by intravenous infusion than in 4 patients treated with an inactive intravenous infusion were problems with balance (38%, 0%), vomiting (38%, 0%), and skin irritation (25%, 0%). The most common side effects were problems with balance and vomiting.

In patients who received EXONDYS 51 for 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.

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).

Please see the full Prescribing Information for EXONDYS 51 (eteplirsen).