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Recently, Shandong Bausch & Lomb Furuida Pharmaceutical Co.
, Ltd.
has entered the administrative stage for the import application of the 5.
1 new drug tetrabenazine tablets (the relevant acceptance number is JXHS2000123/124), and it is expected to be officially approved in the near future, becoming the second domestic approval.
Huntington's disease (HD) treatment drugs
.
Huntington's disease (HD) is an autosomal dominant neurodegenerative disease, discovered in 1872 by the American medical scientist George Huntington
.
HD patients usually get onset between the ages of 35 and 50, and the survival period after the onset is about 15-20 years.
HD is a rare disease.
It was included in the "First List of Rare Diseases" in China in 2018
.
According to expert estimates, there are currently about 30,000 HD patients in China
Tetrabenazine (Xenazine) is a vesicle monoamine transporter 2 (VMAT2) inhibitor, which reversibly inhibits VMAT2 in vivo through its dihydroxy metabolites (HTBZ) α-HTBZ and β-HTBZ.
Role
.
The drug was originally used to treat schizophrenia.
In China, tetrabenazine was included in the "List of the First Batch of Clinically Needed New Drugs Abroad" issued by CDE in 2018, and will be reported for domestic production in August 2020
.
Once officially approved in the future, tetrabenazine will become the second HD drug approved in China after deuterated tetrabenazine
In addition to the above two drugs, there are currently several HD drugs under development abroad, such as Roche's tominersen (RG6042, also called RO7234292), UniQure's AMT-130, and Voyager Therapeutics' VY-HTT01
.
RG6042 is an antisense RNA drug that works by reducing the production of toxic mutant huntingtin (mHTT).
Roche introduced it from Ionis Pharmaceuticals in 2017
.
RG6042 is the first disease-modifying therapy that targets the intrinsic cause of HD by reducing mHTT production in a critical phase III clinical trial.
AMT-130 is a gene therapy based on the adeno-associated virus vector AAV5, which produces a kind of microRNA called microRNA, which is specifically designed to target HTT mRNA.
The binding of this microRNA leads to the degradation of HTT mRNA, thereby hindering inhibition Defective HTT protein production
.
In 2017, the drug was granted orphan drug designation for the treatment of HD by the FDA
VY-HTT01 is also a gene therapy.
Its clinical trial application was previously suspended due to chemistry, manufacturing and control (CMC) issues, but the FDA lifted the clinical suspension in April this year
.
In addition, all the evaluated doses of antisense nucleic acid drugs WVE-120101 and WVE-120102 in PRECISION-HD2 and PRECISION-HD1 studies did not show statistically significant clinical efficacy.
