Takeda submits New Drug Application for lanadelumab in Japan

Japan-based, R&D-driven Takeda Pharmaceutical Company Limited has submitted a New Drug Application (NDA) for lanadelumab to the Ministry of Health, Labour and Welfare (MHLW) in Japan.

Lanadelumab subcutaneous injection is a monoclonal antibody therapy for prophylaxis against hereditary angioedema (HAE) attacks.

The submission will be evaluated by the Japanese Pharmaceuticals and Medical Devices Agency (PMDA), following which the PMDA will issue a report to Japan’s Ministry of Health, Labour and Welfare (MHLW) for a final decision.

Upon approval, lanadelumab will be available as a pre-filled syringe presentation to patients in Japan.

The rare, highly complex genetic disorder of HAE causes recurrent attacks of oedema or swelling in parts of the body, such as throat, face, abdomen, genitals, hands and feet. 

One out of an estimated 50,000 people in the world are affected by HAE. Japan itself has about 2,000 to 3,000 people suffering from HAE. However, only around 450 people have been diagnosed until now because of the low level of awareness for HAE here.

Takeda Development Center Japan general manager Naoyoshi Hirota said: “HAE is unpredictable, debilitating and potentially life-threatening, and recognition of the disease remains low in Japan, meaning there are significant challenges relating to diagnosis and access to effective therapies.

“Lanadelumab is a fully human monoclonal antibody that specifically binds and decreases plasma kallikrein activity, with a proven efficacy and safety profile as a preventive treatment for HAE attacks. Takeda is committed to bringing important therapies such as lanadelumab to patients with high unmet medical needs around the world. Subject to approval, we are looking forward to providing lanadelumab as a new treatment option for patients in Japan living with HAE.”

Under the tradename TAKHZYRO, Lanadelumab received its first approval for the prevention of HAE in patients aged 12 and above in 2018. It is now available in over 20 countries.

HAE’s NDA submission in Japan is mainly based on the global Phase 3 HELP (Hereditary Angioedema Long-term Prophylaxis) Study and the Phase 3 HELP Study Open-label Extension (OLE) results, apart from the interim results of a Phase 3 study evaluating lanadelumab’s safety and efficacy in Japanese subjects. Currently, the HELP Study is the largest randomised, controlled clinical prevention study conducted in HAE.

Lanadelumab is a fully human monoclonal antibody, which is a subcutaneously administrated formula that is indicated for prophylaxis to prevent HAE attacks in patients 12 years and above.