China urgently needs new drugs for overseas rare diseases: more than 10 models have been approved and multiple models have been submitted for marketing applications

Source: Guanlan Pharmaceutical

Clinically urgently needed overseas new drugs mainly refer to those that have been marketed in Europe, America and Japan in recent years but have not yet been marketed in China, and are used to treat rare diseases, severely life-threatening or severely affecting the quality of life, and there are no effective treatments or obvious clinical advantages.

Up to now, the Center for Drug Evaluation (CDE) of the National Food and Drug Administration of China has officially released three batches of the "List of New Drugs Urgently Needed in Clinics" (non-comment version), totaling 73 varieties, of which 34 are rare disease drugs, accounting for up to This is 46.

More than 10 clinically urgently needed overseas rare disease drugs have been approved

According to public information, among the 34 clinically urgently needed overseas rare disease drugs, more than 10 have been approved for marketing, covering pulmonary hypertension, Fabry disease, multiple sclerosis (MS), and high phenylalanine caused by BH4 deficiency.

Pulmonary hypertension is a common and frequently-occurring disease, with a high disability rate and fatality rate.

Fabry disease is a congenital metabolic disease caused by X-linked gene defects.

Among other drugs, there are several innovative therapies that have been approved for the treatment of corresponding rare diseases.

Some new drugs for rare diseases overseas that have been approved in China

Note: This table is incomplete statistics (new drugs that have not been officially included in the three batches of CDE "Clinical Urgently Needed Overseas New Drugs" are not in the scope of statistics).

These rare disease drugs are expected to be approved in China this year

Public information shows that in addition to the above-mentioned approved drugs, a number of clinically urgently needed rare disease drugs have entered the stage of submitting new drug marketing applications or clinical development in China.

1.

Indications: Multicenter Castleman disease (multicenter Castleman disease)

Stuximab (siltuximab) is an IL-6 inhibitor introduced by BeiGene from EUSA Pharma.

In China, the marketing application of Stuximab for injection has been accepted on January 20, 2021.

2.

Indications: Gaucher disease

Velacilase Alfa for injection is a hydrolyzable lysosomal-specific glucocerebrosidase developed by Shire under Takeda.

In China, the new drug listing application of Verazilase Alpha for injection was accepted by CDE in February 2019.

3.
Biogen: Dimethyl fumarate

Drug mechanism: Nrf2 signaling pathway

Indications: multiple sclerosis

Dimethyl fumarate (dimethyl fumarate) is an oral medication used for the treatment of multiple sclerosis.
Studies have shown that it may have antioxidant properties, which can regulate immune responses, reduce inflammation, and protect the brain and spinal cord from damage.

In China, companies such as Bojian and Janssen have jointly submitted a new drug listing application for dimethyl fumarate enteric-coated capsules in 2020, and the rare disease drug has been included in the priority review by CDE, which is suitable for relapsed multiple disease.
Treatment of adult patients with sexual sclerosis: including clinically isolated syndrome, relapsing-remitting type, and active secondary progressive type.
The application is currently being reviewed and approved.

It is hoped that more clinically needed new drugs for rare diseases abroad will be approved in China this year, bringing treatment options to patients with rare diseases.