Takeda Announces Two Investigational Innovative Drugs Granted Breakthrough Therapy Designation by China's CDE

Shanghai, February 2, 2021 /PRNewswire/ -- Takeda China announced today that its investigational innovative drugs, Maribavir (TAK-620) and Soticlestat (TAK-935), have been officially designated as “Breakthrough Therapy” by the Center for Drug Evaluation of the National Medical Products Administration. With this designation, Takeda now has three investigational innovative drugs in China included in the “Breakthrough Therapy” program, including Mobocertinib (TAK-788), which was previously added.”。

Among the two latest drugs included in the “Breakthrough Therapy Drug”"Species"Among the investigational drugs, Maribavir (TAK-620) is a novel benzimidazole ribonucleoside agent approved for the treatment of post-transplant cytomegalovirus (CMV) infection or disease, including cases refractory or resistant to ganciclovir, valganciclovir, cidofovir, and foscarnet. Cytomegalovirus is a betaherpesvirus, with serological evidence of prior infection found in 40%–100% of the adult population [1]. In immunocompromised patients, such as recipients of hematopoietic stem cell transplantation (HSCT) or solid organ transplantation (SOT), CMV infection represents one of the most challenging post-transplant complications and is a significant cause of morbidity and mortality.

The primary evidence supporting the recent Breakthrough Therapy Designation for maribavir comes from two completed Phase II studies (SHP620-202 and SHP620-203). Among these, study SHP620-202 demonstrated an efficacy rate of 67% in patients with drug-resistant, refractory cytomegalovirus (CMV) infection [2]. The antiviral activity of maribavir is mediated by competitive inhibition of the protein kinase activity of the human CMV enzyme UL97, thereby inhibiting protein phosphorylation. Furthermore, since maribavir’s antiviral activity does not involve the viral UL54 gene, resistance to ganciclovir/valganciclovir and/or cidofovir caused by mutations in this gene is not expected to affect maribavir’s anti-CMV activity. Therefore, maribavir is anticipated to provide breakthrough therapeutic benefits for patients developing CMV infection or disease post-transplantation.

Another investigational drug, Soticlestat (TAK-935), is being jointly developed by Takeda Pharmaceutical Company Limited and Ovid Therapeutics. It has been granted Breakthrough Therapy designation for the indications of Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS). Dravet syndrome is a rare and severe developmental and epileptic encephalopathy, primarily caused by mutations in the SCN1A gene. It typically onset within the first year of life, with symptoms including convulsions and seizures, accompanied by developmental delays, intellectual and behavioral issues, and language impairments. These symptoms severely impact the quality of life for affected children and their families, and may even be life-threatening. Lennox-Gastaut syndrome is another rare and highly detrimental developmental and epileptic encephalopathy, usually onset between the ages of 1 and 8 years. Patients experience multiple types of refractory seizures, characteristic electroencephalogram (EEG) changes, and intellectual impairment; most patients lose their ability to perform activities of daily living by adulthood. Currently, there are no drugs approved in China for the treatment of DS, and the variety of antiepileptic drugs used off-label based on clinical experience for DS and LGS is limited, failing to adequately meet patients' clinical needs.

Soticlestat (TAK-935) is a novel antiepileptic drug with a unique mechanism of action, functioning as an oral, potent, and selective inhibitor of cholesterol 24S-hydroxylase (CH24H). By inhibiting CH24H, the drug reduces the concentration of its product, 24S-hydroxycholesterol, in the brain, thereby decreasing excitability in glutamatergic pathways, alleviating inflammation, and reducing seizure frequency. In 2017, Soticlestat was granted orphan drug designation by the U.S. Food and Drug Administration (FDA) for the treatment of Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS).

As a global innovative biopharmaceutical company that is values-based, R&D-driven, and patient-centric, Takeda is committed to translating scientific achievements into highly innovative breakthrough therapies to benefit patients worldwide. China is one of the four key regions in Takeda’s global R&D network. Leveraging its innovation capabilities and local expertise, Takeda aligns the needs of Chinese patients with its global drug development strategy, progressively achieving simultaneous drug development in China and globally since 2018. The recent designation of Maribavir (TAK-620) and Soticlestat (TAK-935) as “Breakthrough Therapy” drugs is expected to further accelerate the development and approval of innovative medicines in China, enabling earlier access for Chinese patients.

Declaration:
1. This article aims to convey cutting-edge information in the field of medicine and does not constitute a recommendation or promotion of any drug or treatment plan.
2. If you wish to learn more about diseases or obtain information related to medications, diagnosis, and treatment, please consult a healthcare professional.

[1] De La Hoz, R. E., Stephens, G. & Sherlock, C. 2002. Diagnosis and treatment approaches of CMV infections in adult patients. J Clin Virol, 25 Suppl 2, S1-12.

[2] Genovefa A Papanicolaou, Fernanda P Silveira, Amelia A Langston et al. Maribavir for Refractory or Resistant Cytomegalovirus Infections in Hematopoietic-cell or Solid-organ Transplant Recipients: A Randomized, Dose-ranging, Double-blind, Phase 2 Study. Clin Infect Dis. 2019 Apr 8;68(8):1255-1264.

About Takeda Pharmaceutical Company Limited

Takeda Pharmaceutical (Tokyo Stock Exchange Code: 4502(NYSE: TAK) is a values-based, R&D-driven global biopharmaceutical company headquartered in Japan. Takeda is committed to translating scientific discoveries into highly innovative medicines that safeguard patients’ healthy lives and promising futures. Takeda focuses on drug development in four therapeutic areas: oncology, gastroenterology, neuroscience, and rare diseases, with dedicated R&D investments in plasma-derived therapies and vaccines. We remain steadfast in our pursuit of highly innovative medicines, expanding novel treatment options and strengthening our collaborative R&D engine to build a robust and diverse product pipeline that helps improve people’s lives. Our employees operate in approximately 80 countries and regions, working hand in hand with local healthcare partners to deliver health benefits to patients worldwide.

Takeda entered China in 1994. Takeda China’s headquarters is located in Shanghai, with its main business centers on the Chinese mainland situated in Beijing, Shanghai, Tianjin, Guangzhou, and the Hong Kong Special Administrative Region. The company maintains offices in major cities across China and currently employs more than 2,000 people in the country. With China’s economic development and the growing demand for healthcare, China has become one of Takeda’s most important emerging markets globally.

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