Medicine Rong Cloud data www.pharnexcloud.com monitoring shows: In April 2024, Novartis China announced that the innovative drug ® (Iptacopan / hydrochloride capsules) has been approved by the Chinese National Medical Products Administration (NMPA) for the treatment of adult patients with paroxysmal sleep hemoglobinuria (PNH) who have not been treated with complement inhibitors. Just four months after the approval of the US Food and Drug Administration (FDA), ipcopan hydrochloride capsules were successfully approved in China, another example of Novartis's acceleration of global innovative drugs into China at the "China speed" and a manifestation of Novartis's commitment to focus on the needs of Chinese patients and support the building of a healthy China.

Medicine Rong Cloud data www.pharnexcloud.com

 This approval in the Chinese market is based on the results of a pivotal Phase III APPOINT-PNH global and Chinese subgroup clinical study (50% of subjects enrolled in China).

 The Phase III clinical APPOINT-PNH study of Iptacopan PNH patients included 40 patients and showed that the proportion of patients with Iptacopan hemoglobin was 92.2%, above baseline to 12 g/dL, 62.8%, and 97.6%. Another pivotal phase III APPLY-PNH study showed that Iptacopan treatment of adult PNH anemia with C5 anemia was significantly better than continuation of anti-C5 therapy. The Iptacopan group had a higher proportion of patients higher than 2 g/dL from baseline than ecuzumab / Ravulizumab (82.3%vs2.0%) and a higher proportion of patients above 12g / dL (68.8%vs1.8%). There was also a gap in side effects: headache (16.1%vs2.9%), higher probability of diarrhea (14.5%vs5.7%) and lower probability of infection (38.7%vs48.6%).

 Can / Iptacopan is a new oral drug, the world's first specific complement factor B oral inhibitor (Complement Factor B inhibitor), acting on the immune system of complement bypass pathway, can fully control intravascular hemolysis and extravascular hemolysis, make up for the defects of anti-C5 antibody therapy, created a new era of PNH targeted therapy. It is worth noting that another major indication, IgA nephropathy (IgA nephropathy), has been submitted for new drug marketing application in many places.

In clinical studies, ippocopam was used at 200mg / dose twice daily, which is more convenient for patients to take compared to the injection of C5 inhibitor.

 Regarding the disease, paroxysmal sleep hemoglobinuria (PNH)

 PNH is a rare, chronic, and severe, complement-mediated blood disorder. Hematopoietic stem cells (located in the bone marrow, can grow, develop into red blood cells, white blood cells and platelets) acquired mutation, lead to the complement system premature destruction, causing blood vessel lysis (red blood cells in blood vessels) and extravascular destruction (red blood cells in the spleen and liver), as anemia (low level of circulating red blood cells), thrombosis (blood clot) and other debilitating symptoms.

It is estimated that around 10 – 20 people per million people have PNH worldwide. Although PNH can occur at any age, it is common in people aged 30 – 40 years. There is a large unmet need for PNH that is not fully resolved by anti-C5 therapy (eculizumab or ravulizumab). That is, after anti-C5 therapy, a large proportion of PNH patients still have residual anemia and transfusion dependence.

consult:

NMPA/CDE；

Medicine Rong Cloud data www.pharnexcloud.com；

FDA/EMA/PMDA；

Public disclosure of relevant companies;

Novartis Pharmaceutical; www.novartis.com；

https://mp.weixin.qq.com/s/SzzsQvgjdb9aSAPf3de2gg；

Novartis blockbuster Fabhalta® (iptacopan) is FDA approved as the first oral monotherapy for adult PNH to improve hemoglobin levels without blood transfusion;

 A.M.Risitano, et al.Oral complement factor B inhibitor iptacopan monotherapy improves hemoglobin to normal/near-normal levels in paroxysmal nocturnal hemoglobinuria patients naÏve to complement inhibitors: Phase III APPOINT-PNH trial.OS12-06; 2023 EBMT；

Cançado RD, Araújo A da S, Sandes AF, et al.Consensus statement for diagnosis and treatment of paroxysmal nocturnal haemoglobinuria.Hematol Transfus Cell Ther.2021;43(3):341-348. doi:10.1016/j.htct.2020.06.006; etc.