Takeda Submits New Drug Application in Japan for Lanadelumab as a Preventive Treatment for Hereditary Angioedema Attacks

- Lanadelumab is an investigational monoclonal antibody for the preventive treatment of hereditary angioedema (HAE) in patients 12 years and older¹
- Takeda is committed to bringing important therapies to patients with high unmet medical needs around the world

OSAKA, Japan March 12, 2021 – Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) (“Takeda”) today announced that it has submitted a New Drug Application (NDA) to the Ministry of Health, Labour and Welfare (MHLW) in Japan for lanadelumab subcutaneous injection, a monoclonal antibody therapy for prophylaxis against attacks of hereditary angioedema (HAE).

HAE is a rare genetic disorder that results in recurrent attacks of oedema – swelling – in various parts of the body, including the abdomen, face, feet, genitals, hands and throat.^2,3,4 HAE affects an estimated 1 in 50,000 people worldwide.⁵ In Japan, it is estimated that between 2,000 and 3,000 people are living with HAE, but only approximately 450 have been diagnosed due to low awareness of the disorder in the country.⁶

“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,” said Naoyoshi Hirota, General Manager, Takeda Development Center, Japan. “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.”

Lanadelumab, under the tradename TAKHZYRO^®, received its first approval for the prevention of HAE attacks in patients 12 years and older in 2018 and is now available in more than 20 countries with additional regulatory submissions ongoing worldwide.⁷

The submission of the New Drug Application in Japan is primarily based on results of the global Phase 3 HELP (Hereditary Angioedema Long-term Prophylaxis) Study™ and the Phase 3 HELP Study Open-label Extension (OLE), in addition to interim results of a Phase 3 study evaluating the efficacy and safety of lanadelumab in Japanese subjects.^1,8 Combined, these studies have demonstrated the efficacy and safety profile of lanadelumab as a preventive treatment for HAE attacks. If approved, lanadelumab will be available to patients in Japan as a pre-filled syringe presentation.

In the randomized, double-blind, placebo-controlled HELP study, which included 125 people with HAE, lanadelumab reduced the mean number of monthly HAE attacks by 87% relative to placebo when administered at 300 mg every two weeks and 73% relative to placebo when administered at 300 mg every four weeks (adjusted P<0.001).¹ A prespecified, exploratory analysis showed that over the entire 26-week study (days 0-182), 44% (n=12/27) of patients taking lanadelumab 300 mg every two weeks were attack-free vs. 2% (n=1/41) of patients taking placebo.¹ A post-hoc sensitivity analysis showed that 77% (n=20/26) of the patients receiving lanadelumab 300 mg every two weeks were attack-free during a steady-state (day 70-182) vs. 3% of patients on placebo (n=1/37).¹ The most commonly reported treatment-emergent adverse events (excluding HAE attacks) in patients treated with lanadelumab (n=84) during the entire treatment period were injection site pain (42.9%), viral upper respiratory tract infection (23.8%), headache (20.2%), injection site erythema (9.5%), injection site bruising (7.1%), and dizziness (6.0%).¹ Most treatment-emergent adverse events (98.5%) were mild to moderate in severity.¹ The HELP Study^™ is the largest randomized, controlled clinical prevention study conducted to date in HAE.

The regulatory submission in Japan will be evaluated by the Japanese Pharmaceuticals and Medical Devices Agency (PMDA). Following its review, the PMDA will issue a report to the Ministry of Health, Labour and Welfare (MHLW) of Japan for a final decision.