Swapnroop Drugs & Pharmaceuticals is a pharmaceutical and biopharmaceutical API manufacturer and supplier supporting requirements for Tofersen, a synthetic antisense oligonucleotide (ASO) drug substance.
Tofersen is a 20-nucleotide antisense oligonucleotide designed to target superoxide dismutase 1 (SOD1) mRNA. It reduces production of SOD1 protein through sequence-specific binding to SOD1 mRNA. Tofersen is marketed as QALSODY for adults with amyotrophic lateral sclerosis (ALS) associated with a mutation in the SOD1 gene. The FDA approved QALSODY on April 25, 2023.
Tofersen is identified by CAS 2088232-70-4 in the FDA Substance Registration System. Its sodium salt is separately identified as CAS 1898254-60-8.
Tofersen is a chemically synthesized antisense oligonucleotide drug substance rather than a conventional small-molecule API or monoclonal antibody. It is a 20-mer 5-10-5 MOE gapmer containing modified nucleotides and phosphorothioate linkages. The molecule contains five 2'-O-(2-methoxyethyl) nucleosides at each end surrounding a central ten-nucleotide DNA gap.
The molecular formula of tofersen free acid is C₂₃₀H₃₁₇N₇₂O₁₂₃P₁₉S₁₅, with a molar mass of 7,127.86 Da. EMA's assessment report confirms that tofersen contains 15 chiral centres and is therefore manufactured as a mixture of diastereoisomers.
| Parameter | Details |
|---|---|
| Product Name | Tofersen |
| CAS Number | 2088232-70-4 |
| Sodium Salt CAS | 1898254-60-8 |
| UNII | 2NU6F9601K |
| Development Code | BIIB-067 |
| Brand | QALSODY |
| Molecular Formula | C₂₃₀H₃₁₇N₇₂O₁₂₃P₁₉S₁₅ |
| Molecular Weight | 7,127.86 Da |
| Molecule Type | Antisense Oligonucleotide |
| Length | 20 nucleotides |
| Chemical Class | 5-10-5 MOE Gapmer |
| Target | SOD1 mRNA |
| Therapeutic Area | Neurology |
| Indication | SOD1-mutation-associated ALS |
FDA identifies tofersen as an antisense oligonucleotide and confirms its molecular formula and molecular weight.
Tofersen is designed to bind to SOD1 mRNA through complementary base pairing. This promotes degradation of the target RNA through RNase H-mediated mechanisms, thereby reducing synthesis of SOD1 protein.
Tofersen is used in the development and manufacture of pharmaceutical products intended for adults with SOD1 mutation-associated amyotrophic lateral sclerosis (SOD1-ALS).
FDA-approved QALSODY is administered by a healthcare professional through intrathecal injection. The FDA-recommended dosage is 100 mg per administration, with three initial doses at 14-day intervals followed by maintenance doses every 28 days.
EMA also lists QALSODY as a medicine for adults with SOD1 mutation-associated ALS and identifies the active substance as tofersen.
Swapnroop Drugs & Pharmaceuticals supports pharmaceutical and biopharmaceutical organizations seeking Tofersen API / antisense oligonucleotide drug substance for qualified development, manufacturing and procurement requirements.
As a Tofersen API supplier in India, Swapnroop can support requirements involving synthetic oligonucleotide drug substances, subject to applicable specifications, manufacturing capabilities, regulatory requirements and customer qualification.
Tofersen can be positioned for qualified requirements involving:
Oligonucleotide drug substances require specialized manufacturing and analytical controls that differ from conventional small-molecule APIs. EMA specifically addresses manufacturing, characterization, specifications and analytical control for synthetic oligonucleotides.
Tofersen is manufactured through solid-phase oligonucleotide synthesis, followed by cleavage/deprotection and purification steps. EMA reports that the active substance is ultimately sterilized by filtration and stored protected from light.
Quality characterization includes sequence confirmation, mass determination, purity-related analysis, oligonucleotide-related impurities, stereochemical characteristics and other product-specific critical quality attributes.
Conventional small-molecule tests such as melting point, residue on ignition and a generic 98–102% HPLC assay are not appropriate universal release specifications for Tofersen.
EMA reports that the tofersen active substance is formulated as a liquid and protected from light during storage. The active substance is formulated with sodium phosphate monobasic dihydrate, sodium phosphate dibasic anhydrous and sodium chloride.
Final storage conditions, container-closure systems and handling requirements should follow the applicable approved specification and stability data.
Documentation for Tofersen or an applicable oligonucleotide drug substance may include:
Tofersen is a specialized SOD1-targeting antisense oligonucleotide used in the treatment of SOD1 mutation-associated ALS. Swapnroop Drugs & Pharmaceuticals supports organizations seeking Tofersen API in India for qualified pharmaceutical and biopharmaceutical development, procurement and manufacturing requirements.
| Parameter | Specification |
|---|---|
| Appearance | white to off-white crystalline powder |
| Identification | IR & HPLC compliant |
| Assay (HPLC) | 98.0% – 102.0% |