Novartis' Breakthrough Oral Therapy Fabhalta® (Iptacopan) Approved in China for Paroxysmal Nocturnal Hemoglobinuria

May 06, 2024 11:42 CST Updated 11:42

Novartis

Drug Development and Manufacturing

Recently, Novartis China's innovative drug Fyhidra® (Iptacopan Hydrochloride Capsules) has been approved by the China National Medical Products Administration for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH) who have not previously received complement inhibitor therapy. Just four months after receiving approval from the U.S. Food and Drug Administration (FDA), Iptacopan Hydrochloride Capsules successfully gained approval in China, marking another example of Novartis accelerating the introduction of global innovative drugs into China at "China speed." This also reflects Novartis' commitment to addressing the needs of Chinese patients and supporting the Healthy China initiative.

PNH is a chronic, progressive, life-threatening rare blood system disease, with main clinical manifestations including anemia, paroxysmal hemoglobinuria, bone marrow hematopoietic failure, and thrombosis. PNH can occur at any age but is more common in people aged 30-40, imposing a lifelong heavy burden on young and middle-aged adults. Currently, the standard treatment for PNH is anti-complement C5 therapy, but after receiving treatment, a significant number of patients still experience residual anemia, fatigue, and transfusion dependency.

Jiajia, the founder of the PNH Patient Support Group, stated: "PNH patients are mainly young and middle-aged adults. The disease leaves them struggling to seek medical treatment during the prime of their lives, while also facing the risk of life-threatening complications such as thrombosis and renal failure. We hope that more and more innovative treatments and medications can help PNH patients better control their condition, free themselves from the burden of illness, and regain a healthy life just like ordinary people."

Ferrhad® (Iptacopan Hydrochloride Capsules) is the world's first specific complement B factor oral inhibitor, acting on the proximal pathway of the complement alternative pathway in the immune system. It can comprehensively control intravascular hemolysis and extravascular hemolysis,弥补抗C5抗体治疗的缺陷,开创了PNH靶向治疗的全新时代.

Professor Han Bing from the Department of Hematology at Peking Union Medical College Hospital emphasized: "The development of PNH diagnosis and treatment in China has undergone significant exploration and progress. Although existing anti-complement C5 therapies can inhibit intravascular hemolysis, they cannot effectively suppress extravascular hemolysis, leading to persistent anemia issues for some patients who remain dependent on blood transfusions. The method of administration via injection also forces patients to frequently travel to medical institutions, making it difficult to restore a normal pace of life. Therefore, we are very pleased to see Fesid® (Iptacopan Hydrochloride Capsules) being approved so quickly in China. This innovative oral medication not only provides PNH patients with a new option but also has the potential to transform the overall landscape of PNH treatment in China."

As the first oral monotherapy for the treatment of PNH in adults, Iptacopan hydrochloride capsules can improve treatment adherence, enhance medication experience, and improve quality of life. This approval in China is based on the results of the pivotal Phase III APPOINT-PNH global and China subgroup clinical study (with 50% of participants enrolled from China). The study demonstrated the efficacy, safety, and tolerability of Iptacopan hydrochloride capsules in adult PNH patients who had not previously received complement inhibitor therapy. After 24 weeks of treatment, most patients achieved hemoglobin levels of 12 g/dL or higher, nearly all patients avoided the need for transfusions, and patient-reported fatigue also improved.

Ms. Zhang Ying, President and Managing Director of Novartis China, stated: "Novartis has long been deeply engaged in the field of blood diseases, continuously developing and introducing globally leading high-value innovative drugs. Thanks to the national support for the prioritized review of innovative drugs, Fihada® (Iptacopan Hydrochloride Capsules) was quickly approved just 10 months after the submission of its marketing authorization application, significantly narrowing the gap for synchronized approval with the rest of the world. In the future, we will continue to adhere to the concept of 'patient-centeredness,' accelerating the nationwide rollout of Fihada® to meet the most urgent health needs of Chinese PNH patients."

Source: Pudong Release, Novartis Group

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