Abstract: most drugs are used for rare diseases.

To implement the policies set forth in the State Council executive meetings and speed up the entry of overseas new drugs urgently needed in clinical practice to China, according to the Announcement on Issues Pertaining to the Review and Approval of Overseas New Drugs Urgently Needed in Clinical Settings (No. 79 of 2018), NMPA and the National Health Commission organized relevant experts to study, demonstrate the second batch of overseas new drug with disputes and to preliminary screen of new drugs, in order to select the third batch of overseas new drugs urgently needed in clinical settings.

The list of the third batch of Overseas New Drugs Urgently Needed in Clinical Settings is now published in accordance with the procedures. All objections must be filed with CDE in writing with reasons and the electronic version of objection data should be submitted through the mailbox.

Publicity period: 2020/10/29 – 2020/11/04(5 working days)

Mailbox: linchuangjixu@cde.org.cn

National Medical Products Administration

Drug Evaluation Center

2020/10/29

 

Third Batch of Overseas New Drugs Urgently Needed in Clinical Settings

Serial No.	Drug Name (active ingredient)	Company Name (certificate holder)	Place of first approval	Frist approved date in Europe, America, and Japan	Therapeutic areas	Therapeutic target	Adaptation disease	Reason to be listed as needed in clinical settings
1	Cablivi（Caplacizumab）	Ablynx NV	EU	2018/09/03	Blood system disease	Von Willebrand Factor (Vwf) A1 Domain	Acquired Thrombotic Thrombocytopenic Purpura (aTTP)	aTTP is a rare disease. As the only approved drug currently for this indication, the drug is urgently needed in clinical settings.
2	Xospata（Gilteritinib fumarate）	Astellas Pharma Inc.	Japan	2018/09/21	Malignant blood disease	FLT3/AXL	For the treatment of recurrent or refractory acute myeloid leukemia with FLT3 mutation positive.	With clinical advantage, the is urgently needed in clinical settings for refractory recurrent population.
3	Tibsovo (ivosidenib)	Agios Pharmaceuticals, Inc.	United States	2018/07/20	Malignant blood disease	IDH1	Acute myelogenous leukemia	With clinical advantage, the is urgently needed in clinical settings for refractory recurrent population.
4	Brineura (cerliponase alfa) Injection	BioMarin Pharmaceutical Inc.	United States	2017/04/27	Hereditary metabolic diseases	Enzyme replacement therapy	Late infantile neuronal Wax lipofuscin deposition (CLN2)	Drugs for rare diseases.
5	Tecfidera (dimethyl fumarate) Delayed-Release Capsules	Biogen Idec, Inc.	United States	2013/03/27	nervous system	Nrf2	Disseminated sclerosis	Drugs for rare diseases.
6	Xofluza(Baloxavir marboxil)	(Japan）Shionogi & Co., Ltd.	Japan	2018/2/23	Infectious diseases	Cap-dependent endonuclease inhibitor	Treatment of influenza A and B	1. It involves public health; 2. Severe influenza is life-threatening; 3. The drug has therapeutic advantages over the marketed product.
7	Verkazia(ciclosporin)	Santen OY	EU	2018/7/6	Eye disease	N/A	Severe vernal keratoconjunctivitis (VKC) in children and adolescents over 4 years old	Severe vernal keratoconjunctivitis can cause damage to corneal function and even loss of vision. The drug can provide new effective treatment methods for patients who are intolerant or ineffective to existing therapies.