Four Imported Drugs Nominated for Breakthrough Therapy Designation in China, Including Two from Takeda

On January 20, the Center for Drug Evaluation (CDE) updated its Breakthrough Therapy Designation list, proposing four imported drugs for inclusion: Takeda’s TAK-935 and Maribavir, Harbour BioMed’s batoclimab (HBM9161), and AstraZeneca’s Nirsevimab.

From the CDE Official Website

Takeda’s Two New Drugs Proposed for Breakthrough Therapy Designation

TAK-935 (Soticlestat) is a selective CH24H inhibitor co-developed by Takeda Pharmaceutical and Ovid Therapeutics. By inhibiting cholesterol 24-hydroxylase (CH24H) expressed in the brain, this drug reduces the frequency and severity of seizures. The indications proposed for inclusion in the Breakthrough Therapy designation are Dravet syndrome and Lennox-Gastaut syndrome, both of which are epileptic encephalopathies.

From the CDE Official Website

Insight database shows that TAK-935 was first submitted for clinical trial application in China in June 2018, approved for clinical trials in October of the same year, and its first clinical trial was publicly announced in January 2019. Currently, there are two ongoing clinical trials for this drug in China, with the highest progress reaching Phase II clinical trials.

From the Insight database (http://db.dxy.cn/v5/home/)

Maribavir Tablets (TAK-620) are a UL97 protein kinase inhibitor acquired by Takeda through its acquisition of Shire Pharmaceuticals, and it had previously been granted Breakthrough Therapy Designation by the FDA. The indication for which Breakthrough Therapy Designation is now being sought is: treatment of cytomegalovirus (CMV) infection or disease occurring post-transplantation.

From the official website of CDE

The development of this drug has been quite tortuous: Maribavir was initially developed by ViroPharma, but the company abandoned its development in 2009 after a trial failure. In 2013, Shire acquired the drug for $4.2 billion. In May 2018, Takeda acquired Shire, and ownership of the drug subsequently transferred to Takeda.

Harbour BioMed’s “Batoclimab” Proposed for Breakthrough Therapy Designation

Batoclimab (HBM9161) is a fully human monoclonal antibody targeting the neonatal Fc receptor (FcRn). It is a first-in-class novel drug licensed by Harbin Pharmaceutical Group from HanAll Biopharma, with Harbin Pharmaceutical Group holding the rights for development, manufacturing, and commercialization in the Greater China region (including Hong Kong, Macao, and Taiwan). The proposed indication for breakthrough therapy designation is generalized myasthenia gravis.

From the official website of CDE

Insight database shows that four clinical trials of this drug have been initiated in China, with indications under development including immune thrombocytopenia (Phase III), myasthenia gravis (Phase II), neuromyelitis optica (Phase I), and diffuse goiter with hyperthyroidism (approved for clinical trial).

From the Insight database (http://db.dxy.cn/v5/home/)

AstraZeneca’s Nirsevimab Proposed for Breakthrough Therapy Designation

Nirsevimab is a next-generation respiratory syncytial virus (RSV) antibody medication designed to prevent RSV-associated lower respiratory tract disease. A single injection provides sustained protection throughout the RSV season, which can last up to five months. Compared with the first-generation RSV antibody palivizumab, nirsevimab demonstrates more than a 50-fold increase in RSV neutralizing activity and has a half-life of 85–117 days, which is 4–6 times longer than that of palivizumab.

From the CDE Official Website

In July 2020, the phase 2b clinical trial results were published in NEJM. The results showed that for healthy preterm infants aged 29 to 35 weeks, the Nirsevimab treatment group had a 70.1% reduction in the proportion of outpatient visits due to RSV-induced lower respiratory tract infections compared to the control group, and a 78.4% reduction in hospitalization rates. Additionally, there was a significant decrease in the number of patients requiring ICU care, assisted ventilation, or supplemental oxygen.

According to the Insight database, Nirsevimab was approved for clinical trials in China on October 30, 2020, but domestic clinical trials have not yet been initiated.