The company’s new drug application to the Japanese Ministry of Health, Labour and Welfare (MHLW) specifically asks Takhzyro be approved for the prophylactic, or preventive, treatment of swelling attacks associated with HAE.
The application will now be reviewed by the Japanese Pharmaceuticals and Medical Devices Agency, which will later issue a report to the MHLW for a final decision. If approved, Takhzyro will be supplied to patients living in the country in a pre-filled syringe.
“Takeda is committed to bringing important therapies such as lanadelumab to patients with high unmet medical needs around the world,” Naoyoshi Hirota, general manager of the Takeda Development Center in Japan, said in a press release. “Subject to approval, we are looking forward to providing lanadelumab as a new treatment option for patients in Japan living with HAE.”
It is estimated that between 2,000 and 3,000 people in Japan have HAE. Yet, due to low disease awareness in the country, less than 500 patients have been diagnosed, according to Takeda.
“Recognition of the disease remains low in Japan, meaning there are significant challenges relating to diagnosis and access to effective therapies,” Hirota said.
Takhzyro is an antibody-based therapy that prevents HAE attacks by blocking the activity of the enzyme plasma kallikrein, and preventing the overproduction of bradykinin, a peptide that controls blood pressure and inflammation and, in HAE patients, can trigger sudden bouts of swelling.
The therapy is administered by a subcutaneous (under-the-skin) injection, and has been approved to prevent HAE swelling attacks in patients, 12 and older, in the U.S., the European Union, Canada, Australia, Switzerland, and Brazil.
These prior approvals, as well as Takeda’s new request for approval in Japan, were all supported by findings from the Phase 3 HELP trial (NCT02586805). In this trial, which enrolled 125 people with HAE, treatment with Takhzyro reduced the mean average number of monthly HAE attacks by 87% compared with a placebo, when given at a dose of 300 mg every two weeks — its currently approved dosage in the U.S. and other countries.
The most common side effects observed in patients who received Takhzyro in HELP included pain, redness and/or bruising around the injection site, headache, dizziness, and viral upper respiratory tract infection. Most of them were mild or moderate in severity.
Further results from the trial’s open-label extension study (NCT02741596) demonstrated the medication could safely and effectively prevent swelling attacks in HAE patients for up to 2.5 years. Additional analyses of data from these trials also showed Takhzyro could substantially improve patients’ health-related quality of life.
The regulatory submission in Japan was also supported by interim data from an ongoing Phase 3 trial (NCT04180163) that is testing Takhzyro in Japanese patients, 12 or older, with HAE type 1 or 2. This study is currently recruiting participants at multiple locations in Japan.
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