To implement the relevant spirit of executive meeting of the State Council of China and accelerate the entry of clinically imperative new drugs marketed overseas into China, the National Medical Products Administration (NMPA) and National Health Commission of China organized relevant experts on Mar. 28, 2019 to conduct research and demonstration and select the second batch of clinically imperative overseas new drugs.

The NMPA has further optimized the review and approval of clinically imperative overseas new drugs since 2018, and the first batch of innovative drugs including Gardasil 9, Opdivo, Keytruda, and Soliris (eculizumab), etc. have been accelerated to enter China. The second batch of clinically imperative overseas new drugs released by the NMPA will be focused on in this article, and the development progress of drugs among the first batch in China will be briefly updated.

I.     Help from policies: Global innovative drugs speed up to enter China

The NMPA has introduced many important review and approval policies in recent years, such as priority review, acceptance of overseas clinical trial data of pharmaceutical products, optimization of review procedures for imported innovative drugs, and clinical trial implied permission, which provide unprecedented opportunities for global innovative drugs to speed up to enter China, wherein, rare disease drugs have been benefited more as opportunities for them to be marketed in China are rare.

The second batch of clinically imperative overseas new drugs this time has a distinct characteristic:

The proportion of rare disease drugs accounts for a half: 15 drugs in the list of second batch are rare disease drugs, such as Fabrazyme (Agalsidase Beta) for β-galactosidase A deficiency; furthermore, many drugs have been included in the list owing to "therapeutic advantages over marketed products", such as the HCV drug Maviret and HIV drug Biktarvy.

There are many noteworthy points in the second batch of clinically imperative overseas new drugs, specifically:

A. Many drugs have been marketed in China and entered the list upon single indication

For example, according to the information queried, the pharmaceutical products in the list of second batch that have been marketed in China and entered upon single indication include: insulin detemir for Noonan syndrome, ustekinumab for Crohn’s disease, adalimumab for uveitis, and ciclosporin for the new indication of keratoconjunctivitis, which is developed by Santen.

And bosentan dispersible tablets of Actelion has also entered the list. According to the information queried, bosentan tablets has been marketed in China and entered the list upon the new dosage form.

Those drugs, after they enter the second batch, will be further accelerated approval for subsequent expansion of particular new indications or new dosage forms.

B. 5 drugs in the list are at the marketing application stage

Besides the marketed drugs mentioned above, 5 drugs have been submitted marketing applications to the NMPA, including two rare disease drugs, i.e., agalsidase α/β for Fabry disease, and 3 drugs with significant therapeutic advantages which are the HCV DAA Maviret, HIV drug Biktarvy, and PCSK9 monoclonal antibody Praluent (alirocumab).

Wherein, Mavyret will further increase the treatment choices of hepatitis C patients, which has a shorter treatment cycle (the only DAA with treatment course of 8 weeks) and largely reduced treatment expenses and is the best in class.

The HIV drug: Biktarvy is a complete single-tablet regimen that combines integrase inhibitors, and is currently a first-line therapy in the U.S. The HIV drugs in China are still very behind, there is no fixed-dose compound, and integrase inhibitor product with excellent efficacy is rarely seen. The medical insurance in China has extremely poor coverage of fixed-dose compound or complete single-tablet regimen for HIV. Therefore, the marketing of this drug will greatly increase the access to HIV drugs in China and improve patients’ medication.

C. 9 drugs are at the clinical stage

The 9 drugs at the clinical stage are mostly innovative drugs approved for marketing in the world in recent 3 years, wherein, Erleada (apalutamide), Lokelma (sodium zirconium cyclosilicate), and Xofluza (Baloxavir marboxil) have recently been approved in the EU or U.S. in 2018. Those drugs will be further accelerated marketing in China after they are included in the list.

D. Drugs that have not been applied in China

The 9 drugs that have not been applied in China may directly be filed the marketing applications in China, wherein, 1 deserves special attention, which is Vigadrone (vigabatrin).

Grand Pharma is now developing the drug: vigabatrin according to Class 3 generic drug, with the acceptance No. of CYHS1800517, which is to be included in the priority review, and has not been approved when the list was released.

And the first approval time of the drug in the U.S. given by the NMPA is wrong. Vigabatrin developed by Ovation Pharmaceuticals (acquired by Lundbeck) was first approved in the U.S. on Aug. 21, 2009 upon the trade name of SABRIL, which can be verified on FDA website.

Read More:

Review progress of the first batch of clinically imperative overseas new drugs

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