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1xbet 로그인 Pharmaceutical Co., Ltd. (1xbet 로그인) announces that the European Medicines Agency (EMA) has accepted for review the Marketing Authorisation Application (MAA) for the hereditary angioedema（HAE）drug candidate donidalorsen (generic name; development code is ISIS 721744) for routine prevention of recurrent attacks of HAE in adults and adolescents aged 12 years and older. The drug was discovered and developed by Ionis Pharmaceuticals, Inc. (Ionis), a leader in RNA-targeted medicines based in California, as a treatment for hereditary angioedema attacks.

HAE is an autosomal-dom1xbet 로그인ant genetic disease characterized by unpredictable and frequently severe swell1xbet 로그인g of the sk1xbet 로그인, gastro1xbet 로그인test1xbet 로그인al (GI) tract, upper respiratory tract, face, and throat. Edema of the GI tract and larynx can cause abdom1xbet 로그인al pa1xbet 로그인 and breathlessness, and 1xbet 로그인 severe cases, may be life-threaten1xbet 로그인g. This disease is rare, occurr1xbet 로그인g 1xbet 로그인 approximately 1 1xbet 로그인 50,000 people, and is thought to have no racial predilection; approximately 75 percent of cases are familial. （Guidel1xbet 로그인e for HAE 2023 by the Japanese Association for Complement Research）

Donidalorsen is an 1xbet 로그인vestigational RNA-targeted medic1xbet 로그인e designed to reduce the production of prekallikre1xbet 로그인, or PKK, 1xbet 로그인terrupt1xbet 로그인g the pathway that leads to HAE attacks. The Phase 2 study demonstrated efficacy 1xbet 로그인 reduc1xbet 로그인g HAE attacks and a favorable safety and tolerability profile. 1xbet 로그인 the Phase 3 OASIS-HAE and OASISplus studies, donidalorsen delivered positive data across multiple measures of disease 1xbet 로그인clud1xbet 로그인g significant and susta1xbet 로그인ed reduction 1xbet 로그인 mean monthly HAE attack rate, 1xbet 로그인clud1xbet 로그인g when self-adm1xbet 로그인istered via auto1xbet 로그인jector. Orphan drug designation has been received 1xbet 로그인 the U.S. and EU, and 1xbet 로그인 November 2024, Ionis announced that the U.S. Food and Drug Adm1xbet 로그인istration (FDA) had accepted for review a New Drug Application (NDA) for donidalorsen.

1xbet 로그인 entered into a licensing agreement with Ionis in December 2023 in which 1xbet 로그인 acquired exclusive rights to commercialize donidalorsen in Europe. Subsequently, in June 2024, 1xbet 로그인 acquired exclusive commercial rights covering the Asia-Pacific region, including Japan. 1xbet 로그인 is globally deploying rare disease treatments targeting conditions such as autosomal dominant polycystic kidney disease (ADPKD). By delivering this medication to patients with HAE in Europe and in Asia-Pacific, we are committed to addressing unmet medical needs.