Antisense Oligonucleotide

Muscular Dystrophy, Duchenne

<section class="Section" data-sectioncode="34089-3"><a name="s22"></a><a name="section-8"></a><p></p><h1>11        DESCRIPTION</h1><p class="First">VYONDYS 53 (golodirsen) injection is a sterile, aqueous, preservative-free, concentrated solution for dilution prior to intravenous administration.  VYONDYS 53 is a clear to slightly opalescent, colorless liquid, and may contain trace amounts of small, white to off-white amorphous particles.  VYONDYS 53 is supplied in single-dose vials containing 100 mg golodirsen (50 mg/mL).  VYONDYS 53 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 VYONDYS 53 contains: 50 mg golodirsen; 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>Golodirsen 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).   Golodirsen contains 25 linked subunits.  The sequence of bases from the 5' end to 3' end is GTTGCCTCCGGTTCTGAAGGTGTTC. The molecular formula of golodirsen is C<span class="Sub">305</span>H<span class="Sub">481</span>N<span class="Sub">138</span>O<span class="Sub">112</span>P<span class="Sub">25</span> and the molecular weight is 8647.28 daltons.</p><p>The structure of golodirsen is:</p><div class="Figure"><a name="f01"></a><img alt="Structure of Golodirsen
" src="exo03-0013-01.jpg"/></div></section>

Vyondys 53

golodirsen

2 ML golodirsen 50 MG/ML Injection

2 ML golodirsen 50 MG/ML Injection [Vyondys 53]

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 

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Coverage Authorization Appeals 

Enroll in Patient Savings Program 

Patient Assistance Program (PAP)

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About Vyondys 53 

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Instructions For Use: Duchenne Muscular Dystrophy (DMD)