Sarepta Therapeutics is a global biotechnology company with a mission: to engineer precision medicine for rare diseases that devastate lives and cut futures short. They are one of the leading companies in this space whose products are approved by US FDA in Duchenne Muscular Dystrophy (DMD) treatment.
Ikris Pharma Network (IPN) is an authorized partner to provide access to their medicines to Indian patients through Named-Patient Import.
Sarepta Products
EXONDYS 51®(Eteplirsen Injection)
Exondys 51 (also eteplirsen or AVI-4658), developed by Sarepta, is an exon skipping therapy that is authorized to treat patients with Duchenne muscular dystrophy (DMD) who have genetic mutations that are amenable to exon 51 skipping; about 13 percent of the total Duchenne Muscular Dystrophy population.
This medicinal product works to address the underlying cause of Duchenne by escalating the level of dystrophin protein fabricated in muscle cells.
Exondys 51 is administered via a weekly infusion into the bloodstream at a standard dosage of 30 mg per kg. Each infusion typically lasts approximately 35-60 minutes.
The most common adverse reactions associated with the treatment of Exondys 51 include vomiting and balance problems.
Exondys 51 at-a-Glance:
- An injectable solution, approved in the United States
- Manufactured and commercialized by Sarepta Therapeutics
- Designated as an orphan drug (a medicine to be used in rare diseases) in the United States
VYONDYS 53® (Golodirsen Injection)
Vyondys 53 (also golodirsen or SRP-4053) is an authorized exon skipping therapy developed by Sarepta to treat individuals with Duchenne muscular dystrophy (DMD) whose disease is amenable to exon 53 skipping; representing approximately 8 percent of DMD patients.
Vyondys 53 therapy is designed to address the root cause of Duchenne by enabling the body to make a functional version of the dystrophin protein whose lack typically causes the disease.
Vyondys 53 is administered at a standard dosage of 30 mg per kg of body weight, once weekly via an infusion into the bloodstream that typically lasts 35-60 minutes.
The most common adverse reactions associated with the treatment of Vyondys 53 include falls, abdominal pain, headache, fever, the common cold, vomiting, cough, and nausea.
Vyondys 53 at-a-Glance:
- Approved in the United States
- Manufactured and commercialized by Sarepta Therapeutics
- Designated as an orphan medicine in the United States
AMONDYS 45®(Casimersen Injection)
Amondys 45, previously Casimersen, is an antisense oligonucleotide from Sarepta Therapeutics’s phosphorodiamidate morpholino oligomer (PMO) platform, used to treat Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation amenable to exon 45 skipping.
Amondys 45 is the 3’rd Sarepta exon-skipping treatment that the U.S. FDA approved for DMD patients. The other two treatments are Exondys 51 (Eteplirsen), for all those amenable to exon 51 skipping, and Vyondys 53 (Golodirsen), for all those with mutations amenable to exon 53 skipping.
Amondys 45 is administered by intravenous (into-a-vein) infusion over 35-60 minutes, available in single-dose vials containing 100 mg/2 mL. The standard dose is 30 mg/kg taken once weekly.
The most common adverse reactions associated with the therapy of Amondys 45 are fever, upper respiratory tract infections, cough, headache, joint pain, and pain in the mouth and throat.
Amondys 45 at-a-Glance:
- Third Sarepta exon-skipping therapy, approved by US FDA for patients with DMD
- Manufactured and commercialized by Sarepta Therapeutics
- Designated as an orphan drug in the United States
