Project's information

Project's title Study on ataluren synthesis for produce of medicine to treat duhenne and becker muscular dystrophy
Project’s code VAST04.01/18-19
Research hosting institution Institute of Chemistry
Project leader’s name Trần Đức Quân
Job Title, The rank of a Professor, Academic degree Dr.
Project duration 01/01/2018 - 31/12/2019
Project’s budget 600 million VND
Classify Excellent
Goal and objectives of the project

Build a process of synthesis and purification of ataluren (purity  ≥ 99%) with high efficiency and stability.

Main results
+ Theoretical results:
- Ataluren has been synthesized at the laboratory scale from methyl 3-cyanobenzoate and 2-fluorobenzoyl chloride, with total efficiency of > 70% (based on methyl 3-cyanobenzoate).
- Develop a simple procedure without column chromatography to purify ataluren up to 99% purity (HPLC).
+ Applied results:
Estimated cost of the ataluren synthesized in this project is about ~ 500,000 VND / 1g, only equal to 1/20 (pure chemical form) and 1/40 ( drug form) compared to the current price of ataluren being sold on the market. Therefore, the project results can be applied in practice.
Novelty and actuality and scientific meaningfulness of the results

- Has built a process of ataluren synthesis with high efficiency, low price, environmentally friendly and in accordance with the technical conditions in Vietnam

Products of the project
+ Scientific papers in referred journals (list): 01 publication
- Study on synthesis process of intermediate compound, methyl 3-(N-hydroxycarbamimidoyl) benzoate, for produce of ataluren (Translarna) medicine to treat  duhenne and becker muscular dystrophy. Journal of Chemistry, 1-3, Volume 57 (6E12), 2019.
+ Patents (list): 01 Patent for utility solution (GPHI)
- Synthesis process of methyl 3-(N-hydroxycarbamimidoyl) benzoate. The decision to accept the valid application No. 106181 / QD-SHTT dated November 26, 2019
+ Technological products (describe in details: technical characteristics, place):
- Synthesis process of 2-flourobenzoyl chloride
- Synthesis process of methyl 3-cyanobenzoate
- Synthesis process of ataluren 
- Synthesis process of ataluren
- A set of documents proving product structure: TLC, mp, 1H, 13C-NMR, LC-HRMS, HPLC spectra
- 40g of ataluren 
Recommendations

From the achieved results, the research team respectfully recommends the Vietnam Academy of Science and Technology allow following studies:
From the achieved results, the research team respectfully suggests the Vietnam Academy of Science and Technology to allow realizing the following studies:
- continue to study the synthesis process at a larger scale,
- assess the quality and stability of synthesized ataluren product.
so that ataluren product within the framework of the project are eligible to put into practice the production of drugs for treatment of DMD patients.