当社のジャパン ファーマ ビジネス ユニット希少疾患事業部長である百合田剛史は「先天性プロテインC欠乏症は重篤な場合、長期的な後遺症が残ったり、最悪の場合、死に至ることもある疾患です。世界46カ国で承認されているセプーロチンを日本における新たな治療選択肢として提供し、もし自分の子どもが先天性プロテインC欠乏症だったらという気持ちを従業員一同が持ちながら、患者さんやご家族の皆さんに少しでも貢献できるよう尽力してまいります」と述べています。- Congenital protein C deficiency is a rare condition caused by the congenital lack of protein C, which inhibits blood coagulation and can lead to thrombosis.
- To provide a new treatment option for patients with congenital protein C deficiency, with the indications being the treatment of venous thromboembolism and disseminated intravascular coagulation caused by congenital protein C deficiency, as well as the suppression of thrombus formation tendency.
Today, we would like to inform you that we have released "Sepurochin for intravenous use 1000 units" (dried concentrated human protein C, hereinafter referred to as "Sepurochin") for the treatment of venous thromboembolism and disseminated intravascular coagulation caused by congenital protein C deficiency, as well as the suppression of thrombus formation tendency.
Sepurochin is based on the evidence of efficacy and safety obtained from a domestic phase 1/2 clinical trial (NCT04984889) targeting 5 cases of congenital protein C deficiency patients aged 4-27 years old mainly among Japanese, as well as two overseas phase 2/3 clinical trials (IMAG-098 trial, 400101 trial) targeting non-Japanese congenital protein C deficiency patients. We submitted a manufacturing and marketing approval application to the Ministry of Health, Labour and Welfare on April 28, 2023, and obtained manufacturing and marketing approval on March 26, 2024.
Yurihata Takeshi, the head of our Japan Pharma Business Unit's Rare Disease Division, stated, "Congenital protein C deficiency is a serious condition that can leave long-term sequelae and, in the worst cases, result in death. We will strive to provide Sepurochin, which is approved in 46 countries around the world, as a new treatment option in Japan, and with the thought of what if my own child had congenital protein C deficiency, all of our employees will make every effort to contribute as much as possible to the patients and their families."
Congenital protein C deficiency is a rare condition that results in a state of hypercoagulability due to the congenital deficiency of protein C, which is produced in the liver and inhibits blood coagulation, sometimes leading to life-threatening thrombosis. Especially in neonates and infants, serious intracranial lesions such as cerebral infarction and cerebral venous sinus thrombosis often occur, and severe symptoms such as fulminant purpura and vitreous hemorrhage can rapidly develop, leaving long-term sequelae in many cases.
<"Seplochin intravenous injection 1000 units" (Dry concentrated human protein C)>
Seplochin is a dry concentrated human protein C preparation for intravenous injection that is fractionated and purified from human plasma, and subjected to virus removal and inactivation. The active ingredient, protein C, exerts anticoagulant effects by being converted to activated protein C by the thrombin/thrombomodulin complex on the surface of endothelial cells, similar to endogenous protein C. Therefore, Seplochin is considered useful for the treatment of venous thromboembolism and fulminant purpura in patients with congenital protein C deficiency, as well as for the suppression of thrombus formation tendency. It has been approved in 46 countries worldwide as of July 2024 as a therapeutic drug for severe congenital protein C deficiency, obtaining manufacturing and marketing approval in Europe in July 2001 and in the United States in March 2007.
Product overview of Seplochin in Japan
| Product Name | Seplochin intravenous injection 1000 units |
| Generic name: | Dry concentrated human protein C |
| Indications or effects: | Treatment of the following diseases resulting from congenital protein C deficiency and inhibition of thrombosis tendency. Venous thromboembolism. Purpura fulminans. |
| Usage and dosage: | Dissolve this drug in the attached injection water and administer it slowly intravenously. Usually, the initial dose is 100-120 international units/kg, followed by 60-80 international units/kg every 6 hours for 3 times, and then 45-60 international units/kg every 6 or 12 hours. The dosage and frequency of administration may be adjusted according to the patient's condition. Long-term supplementation in the inhibition of thrombosis. Usually, it is administered at a dosage of 45-60 international units per kg every 12 hours, but it can also be initiated from the dosage and administration used for short-term supplementation. The dosage and frequency of administration should be adjusted as appropriate according to the patient's condition. |
| Drug price | 558,108 yen |
About Takeda Pharmaceutical
Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) aims to contribute to the health of people worldwide and a brighter future. The company is committed to creating innovative pharmaceuticals in areas and businesses such as digestive diseases and inflammatory diseases, rare diseases, plasma-derived therapies, oncology (cancer), neuroscience (mental disorders), and vaccines. In partnership with others, Takeda is working to create a strong and diverse pipeline of new treatment options to improve the quality of life of patients. Takeda Pharmaceutical Company, based in Japan, is a research and development-oriented bio-pharmaceutical-leading company that is at the forefront of its focus on patient-centric values, based on the corporate philosophy that has been shaped for over two centuries. It is active in about 80 countries and regions to fulfill its value (values) based on those values. Please see for more details.
In this disclaimer, 'press release' refers to this document, including any oral presentations, Q&A sessions, written or oral materials provided or distributed by Takeda Pharmaceutical Company Limited ('Takeda') in relation to this press release. This press release (including any oral explanations and Q&A sessions related to this press release) does not constitute a part of, and does not constitute an offer to sell or the solicitation of an offer to purchase or subscribe for, any securities, nor a proposal, guidance or solicitation for any purchase, acquisition, sale, disposal or any other transaction in respect thereof. No public offering of securities is made by means of this press release. Unless prepared in accordance with the registration or an exemption from the registration requirements of the U.S. Securities Act of 1933, offers and sales of securities are not being made in the United States. This press release is provided (together with any additional information provided to the recipient) on the condition that it is used by the recipient for information purposes only (and not for consideration of investment, acquisition, disposal or other transactions) and provided that any such use complies with the applicable securities laws. Failure to comply with such restrictions may constitute a violation of the securities laws applicable to such recipient. Companies directly or indirectly owned by Takeda are separate entities. The term 'Takeda' in this press release may, depending on the context, refer only to Takeda Pharmaceutical Company Limited and may be used as a convenient catch-all term for the Takeda group of companies. Similarly, the term 'we,' 'us,' and 'our' may refer to the Takeda group of companies or its employees. These terms may also be used where identifying a specific company is not meaningful or does not serve a useful purpose. The product names mentioned in this press release are trademarks or registered trademarks of Takeda Pharmaceutical Company Limited or their respective owners.
This news release and the accompanying materials contain forward-looking statements, beliefs or judgments regarding Takeda's future business, future position and performance, and future outlook information based on Takeda's estimates, forecasts, targets and plans, or views. Forward-looking statements often contain terms, expressions, or negative expressions such as "targeted," "planned," "believed," "hoped," "continued," "expected," "aimed," "intended," "ensured," "will," "may," "should," "would," "could," "anticipated," "estimated," "projected," "forecast," "outlook," but are not limited to them. These forward-looking statements are based on many important assumptions about key factors and actual results may differ significantly from the future results explicitly or implicitly expected in the forward-looking information. These important factors include the general economic conditions of Takeda's global operations including Japan and the USA, the emergence and development of competitive products, changes in related laws and regulations, difficulties inherent in new product development including clinical success and the uncertainty of regulatory decisions and timing, uncertainties in the commercial success of new and existing products, difficulties or delays in the manufacturing process, fluctuations in interest rates and foreign exchange rates, claims or concerns about the safety or efficacy of products sold or candidate products in the market, health crises such as the COVID-19 pandemic, success of Takeda's environmental sustainability efforts to achieve reduction of greenhouse gas emissions or other environmental goals, and Takeda's initiatives toward business efficiency, productivity improvement or cost reduction including the integration of digital technologies including artificial intelligence (AI), and other business restructuring efforts identified in Takeda's latest annual report submitted to the US Securities and Exchange Commission on Form 20-F, which can be viewed on the Takeda website (www.takeda.com) or www.sec.gov, and other reports of Takeda. Takeda is not obligated to update any forward-looking statements contained in this news release or any forward-looking information provided by Takeda unless required by law or the rules of the securities exchange. Past performance is not indicative of future results, and information on Takeda's business results and information in this news release do not represent future business results of Takeda, nor are they predictions, expectations, warranties or estimates.
Regarding 'Medical Information'
This press release contains information about products, but those products are not available in all countries and may be sold under different trademarks, efficacy, and dosage depending on the country. The information contained herein is not intended to solicit, advertise, or promote any medical pharmaceutical product, including investigational products.
