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Levima received orphan designation from MHLW |Japan drug approvals | Japan Healthcare System

Updated: Jun 27, 2023

Mar 12. 2021

Takeda’s New Drug Application was submitted for MHLW in Japan for Hereditary Angioedema Attacks

Takeda announced that it submitted a new drug application of TAKHZYRO (lanadelumab) to the Ministry of Health, Labour and Welfare (MHLW) in Japan for hereditary angioedema.

The drug is indicated for patients who were 12 years and older.

The submission is based on the global Phase 3 HELP study, and the Phase 3 HELP open-label extension study. These studies demonstrated the efficacy of lanadelumab in preventing treatment for HAE attacks. When lanadelumab was administered as 300 mg every two weeks, mean number of monthly HAE attackes reduced by 87% versus placebo. HAE attacks reduced by 73% versus placebo when administered every four weeks. Site pain, headache, upper respiratory infections, headache, injection site erythema were the common adverse events.

The Japanese Pharmaceuticals and Medical Devices Agency (PMDA) will review the application, following which MHLW will make the final recommendation.

Hereditary angioedema is a rare genetic disorder wich is impacts 1 in 50,000 patients worldwide. In Japan, nearly 2,000-3,000 patients are suffering with hereditary angioedema.

Mar 12. 2021

Japan's MHLW provided orphan designation to Lenvima for treating uterine cancer

Eisai announced that it received an orphan designation from the Ministry of Health, Labour, and Welfare (MHLW) for Lenvima (lenvatinib mesylate) for treating patients with uterine body cancer.

The approval is based on the Phase 3 Study 309/KEYNOTE-775 study, evaluating Lenvima in combination with Keytruda for treating patients with advanced endometrial cancer. The combination has met the primary endpoint of progression-free survival (PFS) and overall survival (OS) along with the secondary endpoint of objective response rate.

There are more than 30,000 patients in Japan who have uterine body cancer. In 2020, 3,000 patients died because of uterine body cancer. 90% of uterine cancers start from the endometrium.

Eisai entered a collaboration to co-develop and co-commercialize Lenvima with Merck & Co.

May 20 2020

Gilead’s Veklury (remdesivir) approved by Japan’s MHLW for COVID-19

Gilead has announced that the Japanese Ministry of Health, Labor and Welfare approved Veklury (remdesivir) for severe COVID-19. The drug approved under an exceptional approval pathway, which was granted based on the Emergency Use Authorization of remdesivir in the United States.

The approval is based on the Phase 3 SIMPLE clinical trial; data is available under the compassionate use program. “The Japanese approval of remdesivir is in recognition of the urgent need to treat critically ill patients in Japan. It is a reflection of the exceptional circumstances of this pandemic,” said Merdad Parsey, Chief Medical Officer, Gilead Sciences.

Earlier the drug has received an Emergency Use Authorization from the Food and Drug Administration in the United States. The drug is only indicated for severe patients (patients with oxygen saturation (SpO2) <94% on room air or requiring supplemental oxygen or requiring ventilation or requiring extracorporeal membrane oxygenation (ECMO)).

The drug is indicated for both adults and pediatrics and is administered intravenously. The drug has not yet received approval from the US FDA for treating patients with COVID-19.


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