Frequently Asked Questions About Eteplirsen (Exondys 51) for DMD Treatment

Let’s learn together the answers to the frequently asked questions by Duchenne muscular dystrophy (DMD) patients about Eteplirsen (Exondys 51), which was approved by the US Food and Drug Administration on September 19, 2016. [Learn More: FDA Approves Exondys 51]

Eteplirsen, also known by its brand name Exondys 51, is a medication used to treat Duchenne muscular dystrophy (DMD). DMD is a genetic disorder characterized by progressive muscle weakness and degeneration.

Eteplirsen works by helping the body produce a protein called dystrophin, which is essential for muscle function. This medication is administered through intravenous infusion and has shown promising results in improving muscle function and slowing disease progression in some patients with DMD.

What is Exondys 51 (Eteplirsen)?

Eteplirsen (marketed under the brand Exondys 51) is an exon-skipping medication intended to treat certain types of Duchenne muscular dystrophy (DMD) caused by a certain mutation. Eteplirsen only targets specific mutations and is effective in treating approximately 13% of DMD cases.

Is Eteplirsen (Exondys 51) Approved by EMA in Europe?

The application Sarepta submitted for Exondys 51 was denied by the European Medicines Agency (EMA). [Read More: Exondys 51 Refused By EMA]

Why Did EMA Reject Exondys 51 Application?

The CHMP was concerned that the main study, which involved just 12 patients, did not compare Exondys with placebo beyond 24 weeks, during which there was no meaningful difference between Exondys and placebo in the 6-minute walking distance. The methods for comparing results of the main studies with historical data were not satisfactory for showing that the medicine was effective. [Read More: Reasons Why EMA Rejected Exondys 51 Application]

Is it possible to treat all types of DMD with Exondys 51, or only some of them?

Exondys 51 is an exon skipping medication that selectively targets the dystrophin gene’s exon 51, a region of genetic coding. Skipping exon 51 may help 13 percent of males with DMD, according to estimates. [Learn More: What is Exon Skipping]

Which exon skipping therapy is right for my son?

There are more exon skipping medications in the drug development pipeline that may affect various types of DMD. Currently, scientists are developing ways to target multiple exons and exon skipping medications to target exons 44, 45, 50, 52, 53, and 55. Other strategy-based DMD therapeutics are also being developed. [Discover More: List of All Researches for Duchenne]

Who can use Exondys 51?

The first exon-skipping treatment for DMD was approved in 2016 when the U.S. Food and Drug Administration provisionally approved Exondys 51 for patients with mutations amenable to exon 51 skipping.

Early indications that it might raise dystrophin protein levels led to the approval under the fast approval procedure. However, more clinical trials confirming the therapy’s impact on motor function will be necessary for ongoing licensure for this indication.

Who should not use Exondys 51?

There are no contraindications listed in the Exondys 51 prescribing instructions. However, patients may need to have their infusion delayed or treatment stopped if they develop allergic responses, which include hives, coughing, chest pain, and elevated heart rate. [Learn More: Warnings and Precautions]

Is there an age range that Exondys 51 is effective for?

Boys between the ages of 7 and 13 were the subjects of the clinical trials that backed the drug’s approval when the investigations began. In order to ascertain the effectiveness of Exondys 51 in young boys and men aged 4 to 21, Sarepta Therapeutics is presently undertaking a variety of clinical investigations. Talking to your doctor about what could be best for you or your child is the best course of action right now.

How much Exondys 51 treatment cost, and will insurance pay for it?

Depending on the pharmacy, a supply of two milliliters of Exondys 51 (50 mg/mL) intravenous solution costs approximately $1,694, according to Drug.com. The SareptAssist Patient Support and Assistance program, established by Therapuetics, offers case managers to assist in navigating the challenges of starting therapy.

In Which Countries Is Exondys 51 Available?

In order to create a managed access program (MAP) for its lead drug Exondys 51 (eteplirsen) for the treatment of Duchenne muscular dystrophy (DMD), Sarepta Therapeutics has teamed up with Idis Managed Access, a branch of Clinigen Group. Patients who reside in Argentina, Brazil, Canada, Colombia, France, Germany, Greece, Iceland, Italy, Mexico, Spain, Turkey, and the United Kingdom will be able to use the MAP for the time being. Sarepta anticipates adding more nations to the program’s list in the future. The treating physician must complete all requests on the patient’s behalf.