Melphalan hydrochloride for injection (Applause)®) Approved in China

2018Year12Month3day,CASIAnnouncedEvomela®(Uweining, melphalan hydrochloride for injection) is approved in China for high-dose treatment before autologous stem cell transplantation in patients with multiple myeloma, or for palliative treatment in patients with multiple myeloma who are not suitable for oral melphalan.

This isCASIThe first drugs to be approved independently were previously applied through partners. This drug is also one of the rare disease drugs approved by the National Medical Products Administration of China for the first time through foreign clinical data into the new priority review. There are three reasons: (1) Innovative drugs with obvious therapeutic advantages compared with existing treatment methods; (2) for the treatment of multiple myeloma, a rare disease in China; (3) clinical shortage.

The drug belongs to the registered classification5.1Class.2016Year12month,CFDAByEvolmelaImport Registration Approval, same year4Monthly entry review,9Enter the priority review channel in January,12It took a total of money to get listing approval in China in January, from entering the approval process to formally obtaining approval.242Day.

Multiple myeloma

Multiple myeloma(multiple myeloma, MM)It is a systemic malignant tumor caused by abnormal proliferation of bone marrow plasma cells. The tumor accumulates in the bone marrow, accompanied by the secretion of monoclonal antibodies, resulting in bone marrow failure and bone destruction. Due to the slow onset of the disease, it can be asymptomatic for several months to more than ten years. It is easy to be misdiagnosed in the early stage and cannot be cured at present. It is still dominated by anti-tumor chemotherapy. Although multiple myeloma is usually sensitive to cytotoxic drug chemotherapy, most patients have a short reaction time and often relapse. Melphalan is the most commonly used high-dose pretreatment intravenous drug for multiple myeloma patients undergoing autologous stem cell transplantation.

It occurs in the elderly and can cause bone marrow failure, bone destruction, hypercalcemia, anemia, infection, kidney failure, and neurological symptoms. At present, the incidence of multiple myeloma in China is about one in 100,000, making it the second largest hematological malignancy after non-Hodgkin's lymphoma, compared with one in 100,000 in Western countries.3.6~6.3The incidence is still relatively rare, but as the society gradually ages and the elderly population continues to grow, the disease burden of multiple myeloma will increase.

Drug Introduction

Chinese name: melphalan hydrochloride

English name:Melphalan Hydrochloride

Trade name: Uweining (Evomela®)

Molecular formula:C₁₃H₁₈Cl₂N₂O₂Ho ﹒HCl

Evomela®is a kind of useCaptisoltechnology (Ligand Pharmaceuticalsdevelopment) of a new intravenous formulation of non-propylene glycol, which has beenFDAOrphan drug granted and approved for marketing. In the existing melphalan hydrochloride injection, propylene glycol is used as a solubilizer, and it is reported that propylene glycol can cause adverse reactions in kidney and heart, thus limiting the large-dose application of melphalan injection.

CaptisolThe technology improves the solubility and stability of melphalan, eliminating the need for a cosolvent containing propylene glycol, and the research data affirm the efficacy and acceptable safety of the new dosage form as a high-dose pretreatment regimen for patients with multiple myeloma during autologous stem cell transplantation, making it possible for clinicians to safely use higher doses of chemotherapy before transplantation. At the same time, this is also the following1964year of beautyThe first new formulation after the approval of Farren.

AboutCaptisolTechnology

CaptisolIs a patent-protected modified cyclodextrin drug, its structure makes the drug has good solubility and stability, originally by Kansas.HiguchiCenter for Biological SciencesValentino StellaD.'s laboratory is developed to achieve specific effects in drug development and formulation. This unique technology promotes6drugs throughFDAApprovals, includingOnyxof the pharmaceutical industryKyprolis, Baxter International'sNexteroneand Merck'sNOXAFIL IV. In addition to this, there are30The remainder is used.CaptisolTechnology's products are in the clinical development phase.

Patent Information

Evomela®It is registered in the orange peel book of the United States.4The relevant U.S. patents are all Chinese family patents, two of which are still under substantive examination.