Antisense Oligonucleotide

Muscular Dystrophy, Duchenne

<section class="Section" data-sectioncode="34089-3"><a name="s23"></a><a name="section-9"></a><p></p><h1>11 DESCRIPTION</h1><p class="First">EXONDYS 51 (eteplirsen) injection is a sterile, aqueous, preservative-free, concentrated solution for dilution prior to intravenous administration. EXONDYS 51 is clear and colorless, and may have some opalescence, and may contain trace amounts of small, white to off-white amorphous particles. EXONDYS 51 is supplied in single dose vials containing 100 mg or 500 mg eteplirsen (50 mg/mL). EXONDYS 51 is formulated as an isotonic, phosphate buffered saline solution with an osmolality of 260 to 320 mOsm and a pH of 7.5. Each milliliter of EXONDYS 51 contains 50 mg eteplirsen; 0.2 mg potassium chloride, 0.2 mg potassium phosphate monobasic, 8 mg sodium chloride, and 1.14 mg sodium phosphate dibasic, anhydrous, in water for injection. The product may contain hydrochloric acid or sodium hydroxide to adjust pH.</p><p>Eteplirsen is an antisense oligonucleotide of the phosphorodiamidate morpholino oligomer (PMO) subclass. PMOs are synthetic molecules in which the five-membered ribofuranosyl rings found in natural DNA and RNA are replaced by a six-membered morpholino ring. Each morpholino ring is linked through an uncharged phosphorodiamidate moiety rather than the negatively charged phosphate linkage that is present in natural DNA and RNA. Each phosphorodiamidate morpholino subunit contains one of the heterocyclic bases found in DNA (adenine, cytosine, guanine, or thymine). Eteplirsen contains 30 linked subunits. The molecular formula of eteplirsen is C<span class="Sub">364</span>H<span class="Sub">569</span>N<span class="Sub">177</span>O<span class="Sub">122</span>P<span class="Sub">30</span> and the molecular weight is 10305.7 daltons.</p><p>The structure and base sequence of eteplirsen are:</p><div class="Figure"><a name="f01"></a><img alt="Structure and Base Sequence of Eteplirsen
" src="exo00-0020-01.jpg"/></div></section>

Exondys 51

eteplirsen

2 ML eteplirsen 50 MG/ML Injection

Exondys

2 ML eteplirsen 50 MG/ML Injection [Exondys]

10 ML eteplirsen 50 MG/ML Injection

10 ML eteplirsen 50 MG/ML Injection [Exondys]

Psoriasis (PsO)

Psoriatic Arthritis (PsA)

Ankylosing Spondylitis (AS)

Nonradiographic Axial Spondyloarthritis

Taltz

Juvenile Idiopathic Arthritis

Hidradenitis Suppurativa (HS)

Cosentyx

Siliq

Tremfya

Crohn's Disease (CD)

Ulcerative Colitis (UC)

Stelara

Skyrizi

Ilumya

Migraine

Cluster Headache

Migraine Prevention

Emgality

Qulipta

Acute Migraine

Ubrelvy

Nurtec ODT

Vyepti

Aimovig

Ajovy

Breast Cancer: HR+ (HER2-)

Verzenio

Ibrance

Kisqali

Atopic Dermatitis (Eczema)

Cibinqo

Eucrisa

Asthma (injectable)

Asthma OCS Dependent

Nasal Polyposis

Eosinophilic Esophagitis (EoE)

Prurigo Nodularis

Dupixent

Opzelura

Adbry

NSCLC ALK+ OR ROS1+ Disease

Lorbrena

Alecensa

Alunbrig

NSCLC cMET Mutation

Xalkori

Rheumatoid Arthritis (RA)

Xeljanz

Uveitis

Graft Versus Host Disease (GVHD)

Orencia

COVID-19 Treatment

Alopecia Areata

Olumiant

Multiple Sclerosis (MS)

Secondary Progressive Multiple Sclerosis

Zeposia

Rinvoq

Entyvio

Report Adverse Event 

Prescribing Information 

Pharmacy List 

Coverage Authorization Request 

Coverage Authorization Appeals 

Enroll in Patient Savings Program 

Patient Assistance Program (PAP)

SareptAssist Start Form 

SareptAssist Start Form - Spanish 

About Exondys 51 

Side Effects 

Receiving Treatment 

About Your Infusion

FAQs: Duchenne Muscular Dystrophy (DMD)

Patient Resources 

SareptAssist Support Program 

Exondys 51 Start Guide 

Instructions For Use: Duchenne Muscular Dystrophy (DMD)