US FDA approved a new novel drug Exondys 51 for the treatment of Duchenne muscular dystrophy (DMD). This drug contains eteplirsen as an active ingredient. Exondys 51/eteplirsen specifically targets mutations that occurs in DMD gene.This drug has been approved only for the treatment and management of Duchenne muscular dystrophy (DMD) but it does not mean that it cures it forever.
Duchenne muscular dystrophy (DMD) is rare kind of disorder but it is very devastating degenerative disease. In this disease some mutational changes occur in DMD gene which leads to alteration in DMD RNA transcription process. This altered DMD RNA becomes non-functional or in-active and does not bind or recognize dystrophin protein. This is the main root cause of Duchenne muscular dystrophy (DMD).
Exondys 51/eteplirsen Mechanism of Action or Pharmacology
Exondys 51/eteplirsen is morpholino antisense oligomer that causes cleavage of exon 51 (part of a gene that will encode a part of the final mature RNA) during pre mRNA splicing phase of dystrophin protein.This process will generate a functional but modified form of dystrophin protein (skipping exon therapeutically) that can reinstate the binding process that was impaired due to genetic mutations.
The whole process initiated by this drug does not cure this problem but reduce the severity, progression and degeneration. Majority of patients suffering from DMD have this kind of mutation and can be treated effectively with the help of this therapeutic drug Exondys 51/eteplirsen.
Exondys 51/eteplirsen Side effectsAccording to clinical trials this drug exhibited some minor side effects but no life threatening side effect was reported at that time. These minor side effects were
- Balance disorder
This drug is administered in form of infusion through intra-venous route only.
Exondys 51/eteplirsen DosageThis drug is formulated as a single dose vial with potency of 50mg/ml. In adult DMD patients its standard dosage is 30mg/Kg through intravenous route once in a week. Dose titration may be considered depending upon the patient condition and severity of disease.
Exondys 51/eteplirsen Cost/PriceThis drug is administered by your healthcare practitioner (HCP), which usually means:
- It may comes under special items category.
- You cannot fill this prescription in a regular pharmacy. Your doctor, hospital, or clinic will provide this medication because of possible complications or other issues.
- This drug is likely to be covered under your medical benefit if you have insurance.