Melphalan Flufenamide, also known as melflufen, is a peptide-conjugated alkylating agent and prodrug of melphalan. It is designed to enter cells efficiently and undergo enzymatic cleavage by aminopeptidases to release the active alkylating agent melphalan.
Melphalan Flufenamide is a specialized oncology pharmaceutical compound associated with multiple myeloma research and treatment. Its mechanism involves release of melphalan, which alkylates DNA and produces DNA cross-linking, thereby inhibiting DNA and RNA synthesis and promoting tumor-cell death.
Melphalan Flufenamide is a specialized oncology pharmaceutical API / drug substance requiring stringent control of identity, purity, related substances, stereochemical integrity and residual solvents.
CAS Number: 380449-51-4
Molecular Formula: C24H30Cl2FN3O3
Molecular Weight: 498.42 g/mol
Synonyms: Melflufen, Prodrug J-1, J-1
Development Name: J-1
Drug Class: Alkylating Agent
Therapeutic Class: Antineoplastic Agent
Therapeutic Area: Oncology / Hematology
Molecule Type: Peptide-Conjugated Small-Molecule Prodrug
The free-base form has a molecular weight of approximately 498.4 g/mol. The hydrochloride salt has molecular formula C24H31Cl3FN3O3 and molecular weight 534.9 g/mol.
Melphalan Flufenamide is a prodrug of melphalan. Its lipophilic structure facilitates cellular uptake, after which aminopeptidases can cleave the compound to release melphalan.
Released melphalan acts as an alkylating agent and:
Melphalan Flufenamide is associated with pharmaceutical applications involving:
Melphalan Flufenamide hydrochloride has been developed as a sterile lyophilized powder for intravenous administration. The FDA description identifies the product as a single-dose vial containing melphalan flufenamide equivalent to the hydrochloride salt.
Melphalan Flufenamide is a relatively lipophilic prodrug designed for cellular uptake. The hydrochloride form is sparsely soluble in aqueous solutions and soluble in most organic solvents, with a reported pKa of 7.13.
A controlled Melphalan Flufenamide API manufacturing process may involve:
Typical Melphalan Flufenamide API testing may include:
Store Melphalan Flufenamide under validated pharmaceutical storage conditions, protected from excessive moisture, heat and direct light. Exact storage requirements should follow the applicable approved specification and stability data.
Melphalan Flufenamide is relevant to research involving:
The distinction between Melphalan Flufenamide free base (CAS 380449-51-4) and Melphalan Flufenamide Hydrochloride (CAS 380449-54-7) is important for CMS data. The FDA labeling describes the hydrochloride salt as the drug substance used in the product.
| Parameter | Specification |
|---|---|
| Appearance | White to off-white crystalline powder |
| Identification | IR & HPLC compliant |
| Assay (HPLC) | 98.0% – 102.0% |
| Loss on Drying | NMT 1.0% |
| Residue on Ignition | NMT 0.5% |
| Heavy Metals | NMT 10 ppm |
| Total Impurities | NMT 1.0% |
| Water Content | NMT 1.0% |