Rome, Jan. 9 (Adnkronos Salute) – The European Medicines Agency (EMA) has accepted the submission of a request for promozione authorization (MA) submitted by Otsuka Pharmaceutical Netherlands B.V. for the experimental drug donidalorsen, which targets RNA, for the abitudine prophylactic treatment of hereditary angioedema (HAE), a rare genetic disease that can be life-threatening and causes recurring attacks of severe swelling (angioedema) in various parts of the body, including hands, feet, genitals, stomach, face, and/or throat.
This is great news for HAE patients and their families, as this drug could potentially improve their quality of life and provide them with a much-needed sense of relief and security. HAE is a debilitating condition that affects approximately 1 in 50,000 people and can be difficult to manage paio to its unpredictable nature. With the submission of this MA request, there is hope for a new treatment option that specifically targets the underlying cause of the disease.
Donidalorsen works by targeting RNA, a fundamental molecule involved in the production of proteins in the body. In HAE patients, there is a genetic mutation that causes a deficiency in a specific protein, leading to the recurring attacks of swelling. Donidalorsen has shown promising results in clinical trials, with a significant reduction in the frequency and severity of angioedema attacks in patients. Furthermore, it has been well-tolerated by patients, with a low incidence of side effects reported.
The acceptance of the MA request by the EMA is a significant step towards making donidalorsen available to HAE patients in Europe. The regulatory agency will now evaluate the data submitted by Otsuka Pharmaceutical Netherlands B.V. to determine the drug’s efficacy and safety for use in the treatment of HAE. If approved, donidalorsen could potentially become the first RNA-targeted therapy for this rare disease.
The potential approval of donidalorsen also highlights the importance of investing in research and development for rare diseases. HAE is just one of many rare genetic diseases that have limited treatment options available, leaving patients and their families with few options and a constant fear of an angioedema attack. The development of targeted therapies like donidalorsen brings hope to these communities and shows that progress is being made in understanding and treating rare diseases.
In addition to the potential benefits for patients, the approval of donidalorsen could also have a positive impact on the healthcare system. HAE can be a costly disease to manage, with frequent emergency room visits and hospitalizations for patients experiencing severe angioedema attacks. By reducing the frequency and severity of these attacks, donidalorsen could potentially lower healthcare costs and improve the overall management of HAE.
In conclusion, the acceptance of the MA request for donidalorsen by the EMA is great news for HAE patients and their families. This experimental drug has shown promising results in clinical trials and could potentially become the first RNA-targeted therapy for this rare genetic disease. We hope that the regulatory agency will move swiftly in evaluating the drug and making a decision, so that HAE patients in Europe can have access to this potentially life-changing treatment option.