Nusinersen Intrathecal Injection 12 mg/5ml Suppliers & Bulk Manufacturers

Available Forms: Intrathecal Injection

Available Strengths: 12 mg/5ml

Reference Brands: Spinraza(US)

Category: Orphan Drugs

Nusinersen 12 mg/5 mL intrathecal injection, an antisense oligonucleotide for 5q spinal muscular atrophy, for hospital pharmacies, specialist importers and national rare-disease programmes.

Product Description:

Nusinersen is an antisense oligonucleotide, a short modified strand of nucleic acid. In spinal muscular atrophy the SMN1 gene is lost or faulty, and the backup SMN2 gene makes mostly a short, unstable protein. Nusinersen binds SMN2 pre-messenger RNA and alters its splicing, so more full-length survival motor neuron protein is made. It does not cross the blood-brain barrier, so it is injected intrathecally. Each single-use vial holds 12 mg in 5 mL of preservative-free solution, used undiluted.

It is used for 5q spinal muscular atrophy in babies, children and adults. Four loading doses come first, three at two-week intervals and a fourth about five weeks later, then one dose every four months. Experienced staff perform the lumbar puncture, sometimes with sedation or imaging if there is scoliosis, removing about 5 mL of cerebrospinal fluid before injecting over one to three minutes. Platelets, clotting and urine protein are checked before each dose. Post-puncture headache and back pain are common.

Sourcing notes. Nusinersen is a prescription-only orphan medicine, often bought through hospital, named-patient or national programmes. Vials are stored at 2-8 °C in the carton, protected from light and never frozen, and travel in a validated cold chain. The label permits a limited time at room temperature; check the exact limit. Ask for the CTD dossier, a batch CoA, the sterile-site GMP certificate and a CoPP, and confirm the import route allowed. Give patient numbers, dosing phase and required shelf life in your enquiry.