Duchenne Treatments

Available Sarepta Therapies

Explore classes of therapy for Duchenne muscular dystrophy (DMD).

Treatments

Gene therapy
EXONDYS 51
VYONDYS 53
AMONDYS 45

VYONDYS 53 (golodirsen)

Prescribing Information

VYONDYS 53 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 53 skipping. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with VYONDYS 53. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials.1

Designed to Skip Exon 53

VYONDYS 53 is designed to skip exon 53, correcting the out-of-frame mutation that causes a lack of functional dystrophin and intended to allow for the production of an internally truncated dystrophin protein in patients living with DMD.1-3

The effect of VYONDYS 53 on dystrophin production was evaluated in two studies in patients with a confirmed mutation of the DMD gene that is amenable to exon 53 skipping.1

VYONDYS 53 modified gene expression in all patients evaluated, as measured by RT-PCR

100% (N=25)

of patients biopsied demonstrated exon skipping with VYONDYS 531

RT-PCR=reverse transcription polymerase chain reaction.

Overview of patients treated with VYONDYS 53 in clinical trials1,a

VYONDYS53 Clinical Trials Table

aAll patients in clinical trials have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping.

bMoA confirmed by RT-PCR. Skipping occurred at the correct location as confirmed by immunofluorescence staining.

MoA = mechanism of action.

mRNA = messenger ribonucleic acid.

Dystrophin Expression Over Baseline in Patients Treated With VYONDYS 531,4

Patients treated with VYONDYS 53 showed an average dystrophin increase of 15.96 fold

VYONDYS 53 Dystrophin Levels
  • Dystrophin levels measured via western blot can be affected by differences in sample processing, analytical technique, reference materials, and quantification methodologies1
  • Comparing dystrophin results from different assay protocols will require a standardized reference material and additional bridging studies1

VYONDYS 53 Increased Dystrophin Production Over Baseline1

After 48 Weeks on Treatment

VYONDYS 53 Dystrophin Levels over baseline chart
  • Dystrophin levels measured via western blot can be affected by differences in sample processing, analytical technique, reference materials, and quantification methodologies1
  • Comparing dystrophin results from different assay protocols will require a standardized reference material and additional bridging studies1

Hypersensitivity Reactions

Hypersensitivity reactions, including rash, pyrexia, pruritus, urticaria, dermatitis, and skin exfoliation have occurred in VYONDYS 53-treated patients, some requiring treatment. If a hypersensitivity reaction occurs, institute appropriate medical treatment and consider slowing the infusion or interrupting the VYONDYS 53 therapy.1

Kidney Toxicity

Kidney toxicity was observed in animals who received golodirsen. Although kidney toxicity was not observed in the clinical studies with VYONDYS 53, the clinical experience with VYONDYS 53 is limited, and kidney toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides. Kidney function should be monitored in patients taking VYONDYS 53. Because of the effect of reduced skeletal muscle mass on creatinine measurements, creatinine may not be a reliable measure of kidney function in DMD patients. Serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio should be measured before starting VYONDYS 53. Consider also measuring glomerular filtration rate using an exogenous filtration marker before starting VYONDYS 53. During treatment, monitor urine dipstick every month, and serum cystatin C and urine protein-to-creatinine ratio every three months. Only urine expected to be free of excreted VYONDYS 53 should be used for monitoring of urine protein. Urine obtained on the day of VYONDYS 53 infusion prior to the infusion, or urine obtained at least 48 hours after the most recent infusion, may be used. Alternatively, use a laboratory test that does not use the reagent pyrogallol red, as this reagent has the potential to cross react with any VYONDYS 53 that is excreted in the urine and thus lead to a false positive result for urine protein.1

If a persistent increase in serum cystatin C or proteinuria is detected, refer to a pediatric nephrologist for further evaluation.1

Adverse Reactions

In the VYONDYS 53 clinical development program, 58 patients received at least one intravenous dose of VYONDYS 53, ranging between 4 mg/kg (0.13 times the recommended dosage) and 30 mg/kg (the recommended dosage). All patients were male and had genetically confirmed DMD. Age at study entry was 6 to 13 years. Most (86%) patients were Caucasian.1

VYONDYS 53 was studied in 2 double-blind, placebo-controlled studies.1

The following adverse reactions occurred in at least 20% of VYONDYS 53-treated patients and at a rate greater than placebo.1

VYONDYS 53 Safety

Other adverse reactions that occurred at a frequency greater than 5% of VYONDYS 53-treated patients and at a greater frequency than placebo were: administration site pain, back pain, pain, diarrhea, dizziness, ligament sprain, contusion, influenza, oropharyngeal pain, rhinitis, skin abrasion, ear infection, seasonal allergy, tachycardia, catheter site related reaction, constipation, and fracture.1

Please see the full Prescribing Information for VYONDYS 53 (golodirsen).

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IMPORTANT SAFETY INFORMATION

VYONDYS 53 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 53 skipping. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with VYONDYS 53. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials.

IMPORTANT SAFETY INFORMATION

Hypersensitivity:
Hypersensitivity reactions, including rash, pyrexia, pruritus, urticaria, dermatitis, and skin exfoliation have occurred in VYONDYS 53-treated patients, some requiring treatment. If a hypersensitivity reaction occurs, institute appropriate medical treatment and consider slowing the infusion or interrupting the VYONDYS 53 therapy.

Kidney Toxicity:
Kidney toxicity was observed in animals who received golodirsen. Although kidney toxicity was not observed in the clinical studies with VYONDYS 53, the clinical experience with VYONDYS 53 is limited, and kidney toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides. Kidney function should be monitored in patients taking VYONDYS 53. Because of the effect of reduced skeletal muscle mass on creatinine measurements, creatinine may not be a reliable measure of kidney function in DMD patients. Serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio should be measured before starting VYONDYS 53. Consider also measuring glomerular filtration rate using an exogenous filtration marker before starting VYONDYS 53. During treatment, monitor urine dipstick every month, and serum cystatin C and urine protein-to-creatinine ratio every three months. Only urine expected to be free of excreted VYONDYS 53 should be used for monitoring of urine protein. Urine obtained on the day of VYONDYS 53 infusion prior to the infusion, or urine obtained at least 48 hours after the most recent infusion, may be used. Alternatively, use a laboratory test that does not use the reagent pyrogallol red, as this reagent has the potential to cross react with any VYONDYS 53 that is excreted in the urine and thus lead to a false positive result for urine protein.

If a persistent increase in serum cystatin C or proteinuria is detected, refer to a pediatric nephrologist for further evaluation.

Adverse Reactions:
Adverse reactions observed in at least 20% of treated patients and greater than placebo were (VYONDYS 53, placebo): headache (41%, 10%), pyrexia (41%, 14%), fall (29%, 19%), abdominal pain (27%, 10%), nasopharyngitis (27%, 14%), cough (27%, 19%), vomiting (27%, 19%), and nausea (20%, 10%).

Other adverse reactions that occurred at a frequency greater than 5% of VYONDYS 53-treated patients and at a greater frequency than placebo were: administration site pain, back pain, pain, diarrhea, dizziness, ligament sprain, contusion, influenza, oropharyngeal pain, rhinitis, skin abrasion, ear infection, seasonal allergy, tachycardia, catheter site related reaction, constipation, and fracture.

Please see the full Prescribing Information for VYONDYS 53 (golodirsen).

INDICATION

VYONDYS 53 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 53 skipping. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with VYONDYS 53. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials.

References: 1. VYONDYS 53 [package insert]. Cambridge, MA: Sarepta Therapeutics, Inc. 2021. 2. Aartsma-Rus A, Van Deutekom JC, Fokkema IF, et al. Entries in the Leiden Duchenne muscular dystrophy mutation database: an overview of mutation types and paradoxical cases that confirm the reading-frame rule. Muscle Nerve. 2006;34(2):135-144. 3. Wood MJA, Gait MJ, Yin H. RNA-targeted splice-correction therapy for neuromuscular disease. Brain. 2010;133(pt 4):957-972. 4. Frank DE, Mercuri E, Servais L, et al. Golodirsen induces exon skipping leading to sarcolemmal dystrophin expression in patients with genetic mutations amenable to exon 53 skipping. Poster presented at: Annual Clinical Genetics Meeting of the American College of Medical Genetics and Genomics; April 2-6, 2019; Seattle, Washington.