These 13 clinically urgently needed overseas new drugs are expected to be launched within this year

Since the National Medical Products Administration (NMPA) issued the "Clinical Urgently Needed Overseas New Drug Review and Approval Work Procedures" in 2018, a total of 81 drugs have been included in three batches of the "Clinical Urgently Needed Overseas New Drug List".

1.

Drug mechanism: IL-6 inhibitor

Indications: Multicenter Casterman's disease

Stuximab is used to block the activity of the elevated multifunctional cytokine interleukin-6 (IL-6) detected in patients with Castman’s disease.

In 2018, Stuximab was included in the list of China's "First Batch of New Drugs Urgently Needed Overseas".

2.

Drug mechanism: GD2 targeting monoclonal antibody

Indications: Neuroblastoma

The drug was approved by the European Commission in 2017 to treat high-risk neuroblastoma patients over 1 year old who have received induction chemotherapy and achieved partial remission.

In January 2020, BeiGene and EUSA Pharma reached an exclusive development and commercialization agreement for dituximab.

3.

Drug mechanism: bradykinin B2 receptor antagonist

Indications: Acute attacks of hereditary angioedema (HAE)

Iatibant is a potent and selective bradykinin B2 receptor and antagonist.

In June 2020, Takeda submitted an application for the listing of icatibant acetate injection in China and was accepted.

4.

Drug mechanism: PDE4 inhibitor

Indications: plaque psoriasis, psoriatic arthritis

Apster is a new oral small molecule phosphodiesterase 4 (PDE4) inhibitor.

In February of this year, Amgen and Xinji jointly submitted three new drug listing applications for aprost tablets in China and were accepted by the CDE.
According to the CDE announcement, the three listing applications are planned to be included in the priority review, the corresponding specifications are 10mg, 20mg, 30mg, and the proposed indications are all psoriasis.

5.
Tebenazine

Company: Shandong Bausch & Lomb Freda Pharmaceuticals

Drug mechanism: VMAT 2 inhibitor

Indications: Huntington's disease (HD)

Tebenazine is the first drug approved in the United States for the treatment of Huntington's disease.
In August 2020, the tetrabenazine tablets submitted by Shandong Bausch & Lomb Freda Pharmaceuticals Co.
, Ltd.
for the registration of a category 5.
1 new drug were included in the priority review and approval procedure by the CDE for the clinically urgently needed rare disease drugs that have been marketed overseas and have not been marketed in the country.
.
In addition, the tetrabenazine product of CMS has also been in the stage of applying for the market.

6.
Veracilase Alpha for Injection

Company: Takeda

Medication: Enzyme Replacement Therapy

Indications: Gaucher disease

Veracilase α for injection was developed by Shire, a subsidiary of Takeda, and was approved by the FDA in February 2010 for the long-term enzyme replacement treatment of type 1 Gaucher disease in children and adults.

In China, the new drug listing application of Verazilase Alpha for injection by Baishen Biotech was submitted in China in February 2019 and was accepted by the CDE.
It is worth mentioning that Baishen Biotech was acquired by Shire in 2016, and the latter was acquired by Takeda for US$62 billion in 2018.

7.
Dimethyl fumarate

Company: Bojian

Drug mechanism: Nrf2 signaling pathway

Indications: Multiple Sclerosis (MS)

Dimethyl fumarate was approved by the FDA in 2013 as a first-line drug for the treatment of patients with relapsed MS.
It was approved by the European EMA in 2014 and approved in Japan in 2016.

In China, the marketing application submitted by Bojian in February 2020 was accepted by the CDE, and the drug was previously included in the priority review by the CDE as a rare disease drug.
In addition, related products such as Hausen, Benro and Qingfeng Pharmaceutical have entered the clinical stage.

8.
Mabaloxavir tablets

Company: Roche/Shionoyoshi Pharmaceutical

Drug mechanism: Cap structure-dependent endonuclease inhibitor

Indications: Influenza A and B

Mabaloxavir tablets are a "first-in-class" innovative anti-influenza drug jointly developed by Shiono Yoshida and Roche.
It was approved for marketing by the FDA in October 2018 and was also approved by the FDA in the past 20 years.
The first single-dose oral anti-influenza drug with a new mechanism of action, which can treat oseltamivir-resistant virus strains and avian influenza virus strains (H7N9, H5N1).
In the first quarter of 2020, sales of Mabaloxavir tablets were 28 million Swiss francs, a year-on-year increase of 371%.

In June 2020, Roche and Genentech submitted two new drug listing applications for Mabaloxavir tablets in China and were accepted by the CDE.
In November, the application was included in the priority review by the CDE.

9.
Penatinib tablets

Company: Takeda

Drug mechanism: Bcr-Abl inhibitor

Indications: chronic myeloid leukemia, acute lymphocytic leukemia, leukemia

Ponatinib is the third-generation BCR-ABL tyrosine kinase inhibitor and the first drug approved to effectively target T315I resistance mutations.
It was originally developed by Ariad Pharmaceuticals.
In 2017, Takeda acquired Ponatinib through the acquisition of Ariad.
Ni.

In China, Penatinib is conducting phase 3 clinical trials for Philadelphia chromosome-positive acute lymphoblastic leukemia.

10.
Eliglustat

Company: Genzyme

Drug mechanism: ceramide analog inhibitor

Indications: Gaucher disease type 1

Eliglustat is a potent and highly specific ceramide analog inhibitor.
It was originally developed by the University of Michigan.
Genzyme obtained the license.
In 2011, Sanofi acquired Genzyme for US$21 billion to obtain the drug.
In August 2014, eligrostat was approved by the FDA for the treatment of Gaucher's disease, a rare genetic disease.
In 2015, the drug was approved by the EMA.

In China, eligrostat was approved by two clinical trials in October 2020.
It is intended to be developed for use in adolescents ≥12 years of age who are receiving imiglucerase injection therapy and are accompanied by neurological involvement.
Treatment of adults with Gaucher disease.
In addition, Concord Pharmaceuticals submitted a listing application for imitation of eligrostat capsules in April 2019, and it has now entered the listing stage.

11.
Inanibu

Company: Agios Corporation/Xinji

Drug mechanism: IDH2 inhibitor

Indications: Acute Myeloid Leukemia (AML)

Inanib was jointly developed by Xinji and Agios.
It promotes cell differentiation by blocking the production of the metabolite 2-hydroxyglutarate.
It was approved for marketing in the United States in 2017 and is the first IDH2 inhibitor approved by the FDA.
The world's first approved drug for adult AML with IDH2 mutations.

In China, Inanib is currently conducting a Phase 3 clinical study on the effectiveness and safety of the treatment of advanced acute myeloid leukemia.

12.
Cananumumab

Company: Novartis

Drug mechanism: anti-IL-1 monoclonal antibody

Indications: Chilling pyridine-related periodic syndrome (CAPS)

Kananumab is a humanized monoclonal antibody targeting interleukin-1β (IL-1β), which can target tumor inflammation and reduce immunosuppression.

In China, Novartis has carried out a number of phase 3 international multi-center clinical trials of cananumumab, including the prevention of gout attacks and reduction of the risk of cardiovascular events.

13.
Vernacaran Hydrochloride Injection

Company: Cardiome Corporation

Drug mechanism: sodium/potassium channel blocker

Indications: Atrial Fibrillation

Venakalan is a potassium channel blocker, which can specifically block the Ikur channel for the treatment of atrial tachyarrhythmia.
In 2010, the drug was first approved for marketing in Europe.

In China, the Vernacaran hydrochloride and injection jointly declared by Sichuan Baili and Guorui have entered the phase III clinical stage.