Novartis Announces NMPA Approval of Iptacopan Capsules for Treatment-Naïve Adult PNH Patients in China

Apr 26, 2024 22:07 CST Updated 22:07

Novartis

Drug Development and Manufacturing

▎Edited by the WuXi AppTec content team

Just now, Novartis announcedIts innovative drug, Ipkapzide Hydrochloride Capsules, has been approved by the National Medical Products Administration (NMPA) of China.For the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH) who have not previously received complement inhibitor therapy。

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. The current 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 dependence.

Iptacopan hydrochloride capsules are a specific oral inhibitor of complement factor B, acting on the proximal pathway of the alternative complement pathway in the immune system. By controlling intravascular and extravascular hemolysis, it compensates for the shortcomings of anti-C5 antibody therapy, providing a novel targeted treatment option for PNH.

According to a Novartis press release, 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.The approval in China was based on the results of the pivotal Phase 3 APPOINT-PNH global and China subgroup clinical study (50% of participants were enrolled in China).）. This study confirmed the efficacy, safety, and tolerability of iptacopan hydrochloride capsules in adult PNH patients who had not previously received complement inhibitor treatment.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.。

References:

[1] Novartis' Innovative PNH Oral Monotherapy Fehada® (Iptacopan Hydrochloride Capsules) Approved in China — Ushering in a New Era of Targeted PNH Treatment, "Racing to the Market" to Benefit Chinese Patients. Retrieved Apr 26, 2024. From https://mp.weixin.qq.com/s/TgdgxR9slP2ySWL_pv1n5A

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