Meet Max.
Max is a boy with DMD (Deletion of exon 52)

Meet Max.
Max is a boy with DMD (Deletion of exon 52)

About Exondys 51 (HCP)

ABOUT EXONDYS 51

INDICATION

EXONDYS 51 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. A clinical benefit of EXONDYS 51 has not been established. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials.

A genetic test is required to confirm that a patient's mutation of the DMD gene is amenable to exon 51 skipping. For more information about genetic testing resources for patients, visit Parent Project Muscular Dystrophy’s (PPMD) Decode Duchenne website.

DOSING AND ADMINISTRATION

The recommended dose of EXONDYS 51 is 30 mg/kg administered once weekly as a 35 to 60 minute intravenous infusion. If a dose of EXONDYS 51 is missed, it may be administered as soon as possible after the scheduled time.

Application of a topical anesthetic cream to the infusion site prior to administration of EXONDYS 51 may be considered.

The 50 mg/kg once weekly dosing regimen is not recommended.

EXONDYS 51 is a clear and colorless solution that may have some opalescence, and is available as follows:
  • Injection: 100 mg/2 mL (50 mg/mL) solution in a single-dose vial
  • Injection: 500 mg/10 mL (50 mg/mL) solution in a single-dose vial

For more information, download the full Prescribing Information.

SareptAssist can help your patients find a treatment center or facilitate the at-home infusion process.

Get your patients started. Call 1-888-SAREPTA
(1-888-727-3782) for more information.

Case Managers are available Monday through Friday, 8:30am - 6:30pm ET.

GET YOUR PATIENTS STARTED IN 3 STEPS:

SUPPORT, BY YOUR PATIENTS' SIDE

Treating Duchenne muscular dystrophy is complex. Our team is here to help your patients navigate the process of starting and staying on therapy. SareptAssist provides each family with a personal Case Manager who can answer questions and provide information on:

  • Insurance options
  • On-site or at-home therapy options
  • Out-of-pocket costs
  • Financial assistance options
  • The prior authorization process
  • Other Sarepta resources and programs

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INDICATION

EXONDYS 51 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. A clinical benefit of EXONDYS 51 has not been established. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials.

IMPORTANT SAFETY INFORMATION

Adverse reactions in DMD patients (N=8) treated with EXONDYS 51 30 or 50 mg/kg/week by intravenous (IV) infusion with an incidence of at least 25% more than placebo (N=4) (Study 1, 24 weeks) were (EXONDYS 51, placebo): balance disorder (38%, 0%), vomiting (38%, 0%) and contact dermatitis (25%, 0%). The most common adverse reactions were balance disorder and vomiting. Because of the small numbers of patients, these represent crude frequencies that may not reflect the frequencies observed in practice. The 50 mg/kg once weekly dosing regimen of EXONDYS 51 is not recommended.

In the 88 patients who received ≥30 mg/kg/week of EXONDYS 51 for up to 208 weeks in clinical studies, the following events were reported in ≥10% of patients and occurred more frequently than on the same dose in Study 1: vomiting, contusion, excoriation, arthralgia, rash, catheter site pain, and upper respiratory tract infection.

There have been reports of transient erythema, facial flushing, and elevated temperature occurring on the day of EXONDYS 51 infusion.

Please see the full Prescribing Information for EXONDYS 51.

IMPORTANT SAFETY INFORMATION

Adverse reactions in DMD patients (N=8) treated with EXONDYS 51 30 or 50 mg/kg/week by intravenous (IV) infusion with an incidence of at least 25% more than placebo (N=4) (Study 1, 24 weeks) were (EXONDYS 51, placebo): balance disorder (38%, 0%), vomiting (38%, 0%) and contact dermatitis (25%, 0%). The most common adverse reactions were balance disorder and vomiting. Because of the small numbers of patients, these represent crude frequencies that may not reflect the frequencies observed in practice. The 50 mg/kg once weekly dosing regimen of EXONDYS 51 is not recommended.

INDICATION

EXONDYS 51 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. A clinical benefit of EXONDYS 51 has not been established. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials.

IMPORTANT SAFETY INFORMATION

Adverse reactions in DMD patients (N=8) treated with EXONDYS 51 30 or 50 mg/kg/week by intravenous (IV) infusion with an incidence of at least 25% more than placebo (N=4) (Study 1, 24 weeks) were(EXONDYS 51, placebo): balance disorder (38%, 0%), vomiting (38%, 0%) and contact dermatitis (25%, 0%). The most common adverse reactions were balance disorder and vomiting. Because of the small numbers of patients, these represent crude frequencies that may not reflect the frequencies observed in practice. The 50 mg/kg once weekly dosing regimen of EXONDYS 51 is not recommended.

In the 88 patients who received ≥30 mg/kg/week of EXONDYS 51 for up to 208 weeks in clinical studies, the following events were reported in ≥10% of patients and occurred more frequently than on the same dose in Study 1: vomiting, contusion, excoriation, arthralgia, rash, catheter site pain, and upper respiratory tract infection.

There have been reports of transient erythema, facial flushing, and elevated temperature occurring on the day of EXONDYS 51 infusion.

Please see the full Prescribing Information for EXONDYS 51.