Breakthrough Therapy Drugs Receive Major Policy Boost as Novartis, Innovent and Others Gain CDE Designations

　　【Pharmaceutical Network Industry DynamicsOn March 14, the website of the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) showed that Novartis' LNP023 capsule is proposed to be included in the breakthrough therapy program for C3 glomerulopathy (C3G). On March 1, Innovent Biologics Group and AnHeart Therapeutics jointly announced that Taletrectinib (Innovent's R&D code: IBI-344, AnHeart’s R&D code: AB-106) has been included by the Center for Drug Evaluation (CDE) of China's National Medical Products Administration (NMPA) in the list of breakthrough therapy drugs. The proposed indication is for ROS1 fusion-positive non-small cell lung cancer patients who have not previously received ROS1 tyrosine kinase inhibitor (TKI) treatment and those who have previously received ROS1 TKI treatment.
 

According to reports, breakthrough therapy drugs refer to those used for the prevention and treatment of diseases that severely affect the quality of life or are life-threatening without effective means, or drugs with sufficient evidence showing obvious clinical advantages over existing treatments. In August 2020, the opening of this special review channel accelerated the market entry of innovative drugs.
 

Recently, the CDE released the "CDE's Expedited Review and Approval Procedures for Innovative Drug Marketing Applications (Trial) (Draft for Comments)" (hereinafter referred to as the "Procedures"), aiming to encourage the research and development of new drugs, meet clinical medication needs, timely summarize and transform the experience from the emergency review during the pandemic, and accelerate the review process for innovative drugs.
 

Industry insiders said that the publication and clarification of the "Working Procedures" will bring significant benefits to breakthrough therapies. It is reported that the policy increases support for breakthrough therapy drugs, allowing them to directly enter the priority review process, which will accelerate their approval and market launch.
 

This "Working Procedure" applies to applicants after the completion of exploratory clinical trials and when the conditions for conducting pivotal clinical trials have been met, up until the approval for market launch. This procedure does not require an application from the applicant; innovative drug candidates included in the breakthrough therapy program can automatically follow this procedure for subsequent communication, exchange, review, and approval processes. The communication and exchange time limit is 30 days, and the review time limit for drug candidates is the same as that for priority review, which is 130 days.
 

For instance, the previous priority review arranged Type I meeting communication based on queue order. The new working procedure is that, according to specific products, after completing preliminary exploratory clinical trials, applicants can submit a communication request before and during pivotal clinical trials. They must clearly state key technical issues affecting drug submission and provide relevant supporting materials, as well as interim research data in compliance with the format requirements for registration submission; an expert panel will assist in planning the clinical strategy. Additionally, pre-New Drug Application (pre-NDA) communication has been formulated as a separate working procedure; coupled with a series of registration support measures, it undoubtedly becomes a mode akin to teachers highlighting key points before an exam.
 

According to reports, several products have been included in the list of proposed breakthrough therapies this year. In addition to Novartis' LNP023 capsule being considered for inclusion in the breakthrough therapy program, the CDE has also announced that two other products—CK-3773274 tablets introduced by Jixing Pharmaceuticals and Mavacamten capsules from LianBio—have been designated as proposed breakthrough therapies. CK-3773274 tablets are indicated for conditions involving excessive cardiac contraction, such as hypertrophic cardiomyopathy, while Mavacamten capsules are intended for the treatment of symptomatic obstructive hypertrophic cardiomyopathy.
 

Breakthrough therapy drugs can directly enter the priority review process. According to the new "Working Procedures," breakthrough therapies will receive benefits, and the spring of China's innovative drugs is also coming. However, some industry insiders have pointed out that by carefully reading the document, it can be seen that the scope of application of the "Working Procedures" is only for "innovative drugs included in the breakthrough treatment drug program." To take this shortcut, they need to be recognized as breakthrough therapies first.
 

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