Antisense Oligonucleotide

Survival Motor Neuron-2-directed RNA Interaction

Muscular Atrophy, Spinal

<section class="Section" data-sectioncode="34089-3"><a name="s35"></a><a name="section-8"></a><h1>11 DESCRIPTION</h1>Nusinersen is a modified antisense oligonucleotide, where the 2'-hydroxy groups of the ribofuranosyl rings are replaced with 2'-O-2-methoxyethyl groups and the phosphate linkages are replaced with phosphorothioate linkages. Nusinersen binds to a specific sequence in the intron downstream of exon 7 of the SMN2 transcript. The structural formula is:<a name="f01"></a><img alt="Structural Formula
" src="spi02-0018-01.jpg"/>SPINRAZA is supplied as a sterile, preservative-free, colorless solution for intrathecal use in a single-dose glass vial. Each 1 mL solution contains 2.4 mg of nusinersen (equivalent to 2.53 mg of nusinersen sodium salt). Each 1 mL also contains calcium chloride dihydrate (0.21 mg) USP, magnesium chloride hexahydrate (0.16 mg) USP, potassium chloride (0.22 mg) USP, sodium chloride (8.77 mg) USP, sodium phosphate dibasic anhydrous (0.10 mg) USP, sodium phosphate monobasic dihydrate (0.05 mg) USP, and Water for Injection USP. The product may contain hydrochloric acid or sodium hydroxide to adjust pH. The pH is ~7.2.The molecular formula of SPINRAZA is C234H323N61O128P17S17Na17 and the molecular weight is 7501.0 daltons.</section>

Spinraza

nusinersen

5 ML nusinersen 2.4 MG/ML Injection

5 ML nusinersen 2.4 MG/ML Injection [Spinraza]

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

Spinraza is a specific medication categorized as an antisense oligonucleotide. Its primary purpose is to address spinal muscular atrophy (SMA), a form of motor neuron disease affecting both children and adults. Remarkably, it became the inaugural drug sanctioned by the US Food and Drug Administration (FDA) for this particular demographic.

SMA is an inherited condition characterized by weakening and degeneration of skeletal muscles, which typically deteriorates over time. The primary culprit behind SMA is often mutations within the SMN1 gene (survival motor neuron 1). Such mutations result in insufficient functional SMN protein, crucial for maintaining healthy motor neurons in the body.

Fortunately, there exists a backup gene called SMN2, responsible for producing some SMN protein. However, a minor portion of this protein actually operates effectively due to a tendency in SMN2's gene expression process to skip over a segment called exon 7. Consequently, the resulting protein lacks essential building blocks required for proper functioning.

Spinraza intervenes by rectifying the SMN2-produced protein to ensure its proper functionality. It achieves this by ensuring exon 7's missing information is included in the final protein structure.

The FDA granted approval for Spinraza in 2016.
