Today, the innovative oral monotherapy for Paroxysmal Nocturnal Hemoglobinuria (PNH)Feihe Da®(The first prescription for Ipecacuan Hydrochloride Capsules was successfully issued in China,)Marks a new treatment option for adult PNH patients in China, offering comprehensive control of intravascular and extravascular hemolysis with the convenience of oral medication.
AsThe world's first and currently only oral monotherapy for adult PNH,Iptacopan Hydrochloride Capsules were approved in China this April for the treatment of adult patients who have not previously received complement inhibitor therapy. Thanks to the country's accelerated promotion of the introduction of innovative drugs for rare diseases, it took only 54 days for Iptacopan Hydrochloride Capsules to complete the entire process from approval to formal entry into clinical practice.
Potentially Fatal Complications: PNH Patients Live Under the Shadow of Disease for Life
PNH is a chronic, progressive, and life-threatening rare blood system disease, with main clinical manifestations including anemia, paroxysmal hemoglobinuria, and bone marrow hematopoietic failure.1，Patients are always at several times higher risk of thrombosis and chronic kidney disease than the general population, and thrombosis is the most common cause of death.2。PNH is common in people aged 30-403, placing a heavy burden on the health, work, social interactions, and economic conditions of young and middle-aged patients.
Professor Han Bing, the principal investigator in China for the international multi-center Phase III clinical trial of Iptacopan Hydrochloride Capsules and a hematologist at Peking Union Medical College Hospital, stated: "PNH is 'a rare disease within rare diseases,' and patients require lifelong medication to control hemolysis. Many young patients wish to contribute to their families and society but find themselves in the dilemma of being 'rich in years but not strong in ability,' and are desperately hoping for effective treatment. For many years, the pursuit of innovative therapies has been both the shared aspiration of patients and our goal as doctors."
Compensate for the shortcomings of traditional therapies, help patients regain hope for life
Currently, the standard treatment for PNH is mainly based on anti-complement C5 therapy. Although this therapy can inhibit intravascular hemolysis, it cannot effectively suppress extravascular hemolysis, leading to residual anemia, fatigue, and transfusion dependence in some patients.4According to statistics, approximately 88% of patients receiving anti-complement C5 therapy may suffer from anemia, and about one-third of these patients require at least one blood transfusion within 12 months.5Moreover, since anti-complement C5 therapy is administered through intravenous and subcutaneous injections, patients have to "frequently visit the hospital" for blood transfusions and medication, making it difficult for them to live, work, and study like ordinary people, severely impacting their quality of life.
Iptacopan Hydrochloride Capsules are the world's first and currently only specific oral inhibitor of complement factor B.Targeting the proximal pathway of the complement alternative pathway in the immune system, it can comprehensively control intravascular and extravascular hemolysis, compensate for the shortcomings of anti-complement C5 therapy, and usher in a new era of targeted treatment for PNH.
Professor Han Bing stated: "We are very excited to see that multiple clinical studies have confirmed the performance of Iptacopan Hydrochloride Capsules in improving hemoglobin levels, enhancing treatment compliance, tolerance, and safety. We look forward to this innovative drug benefiting more Chinese PNH patients as soon as possible, improving their quality of life, and reducing the disease burden and related healthcare resource consumption. We also hope that all parties will strengthen cooperation to jointly build a more comprehensive rare disease diagnosis, treatment, and support system."
According to the extended-phase results of the pivotal Phase III APPOINT-PNH clinical study recently presented at the 2024 European Society for Blood and Marrow Transplantation (EBMT) Annual Meeting, most patients who continued receiving treatment with Eculizumab Hydrochloride Capsules for 48 weeks were able to sustain an increase in hemoglobin levels to near-normal (12 g/dL or higher). Nearly all patients (97.5%) remained transfusion-free (from Week 2 to Week 48), and patient-reported fatigue also showed improvement.6。
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