Novartis’ investigational oral monotherapy shows positive results against rare blood disease | The Financial Express

Novartis’ investigational oral monotherapy shows positive results against rare blood disease

The pharma major also claimed that Iptacopan was well tolerated with a favorable safety profile consistent with previously reported data.

Novartis’ investigational oral monotherapy shows positive results against rare blood disease
India is also a leading exporter of paracetamol and industry estimates this at over 45,000 tons while China exports about 40,000 tons. (File)

Novartis on Monday announced that its investigational oral monotherapy iptacopan was superior to anti-C5 therapies (eculizumab or ravulizumab) in adults with paroxysmal nocturnal hemoglobinuria (PNH). The positive outcomes come from the pivotal Phase III APPLY-PNH trial which analysed the patients experiencing residual anemia despite prior treatment with anti-C5s1.

PNH is a rare, acquired, life-threatening disease of the blood and is characterized by the destruction of red blood cells, blood clots, and impaired bone marrow function.

“Topline results showed a statistically significant and clinically meaningful increase in the proportion of patients treated with iptacopan (200 mg twice daily) achieving hemoglobin-level increases of 2 g/dL or more from baseline without the need for blood transfusions at 24 weeks, compared to anti-C5 therapies, a primary endpoint of the study. Additionally, there was a statistically significant and clinically meaningful increase in the proportion of patients in the iptacopan arm achieving hemoglobin levels of 12 g/dL or more without the need for blood transfusions at 24 weeks, compared to anti-C5 therapies, also a primary endpoint of the study,” the pharma company stated on Monday.

The pharma major also claimed that Iptacopan was well tolerated with a favorable safety profile consistent with previously reported data.

“These positive topline Phase III results highlight the practice-changing potential of iptacopan for patients suffering from debilitating anemia and the burden of lifelong blood transfusions as a result of PNH. We look forward to discussing the data with regulators so we can bring this first-in-class alternative complement pathway inhibitor as the first oral monotherapy to people living with PNH,” said Shreeram Aradhye, M.D., President, Global Drug Development and Chief Medical Officer, Novartis in a statement.

According to the company, Iptacopan is also being studied in complement-inhibitor-naïve patients with PNH in the ongoing Phase III APPOINT-PNH trial (NCT04820530), expected to read out in the coming months6.

Additionally, iptacopan is being studied in Phase III trials for complement-mediated kidney diseases (CMKDs) C3 glomerulopathy (APPEAR-C3G [NCT04817618]), IgA nephropathy (APPLAUSE-IgAN [NCT04578834]), and atypical hemolytic uremic syndrome (APPELHUS [NCT04889430]), as well as in a number of additional indications in Phase II7-9, it stated.

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First published on: 24-10-2022 at 11:56 IST