'Too Many Tests': Expert Panel Questions Novartis Trial Protocol For Rare Kidney & Blood Disorder Drug
Novartis spokesperson told News18 that the company is in the process of reviewing the minutes of the meeting and post consultation with our global teams, it will take the necessary next steps.
The panel of experts has rejected the protocol submitted to kick start the clinical trial of Novartis Iptacopan – first-in-class oral medication to treat rare blood disorders and kidney diseases. The subject expert committee (SEC) has asked the Swiss drugmaker to “submit a revised protocol" as experts believed that the patients are subjected to “many investigations" in the clinical trial which are not relevant to the progress of the diseases and the mechanism of effect of the drug.
Novartis has presented a phase 2a clinical study of its drug Iptacopan which is used to treat paroxysmal nocturnal hemoglobinuria (PNH), a type of anaemia where an excessive breakdown of red blood cells occurs, resulting in insufficient healthy cells to deliver oxygen throughout the body.
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The drug was granted accelerated approval by the US Food and Drug Administration (FDA) as it is a first-in-class complement inhibitor for the reduction of proteinuria in adults with primary immunoglobulin. The drug reduces protein in the urine (proteinuria) in adults with chronic kidney disease.
According to the minutes of the meeting, “Committee opined that justification of exposing the patients to invasive procedure (biopsy) is not clear; moreover, it seems procedure is without any clear therapeutic advantage."
“It is not clear how to define inadequate biopsy and what action to be taken if it is inadequate. Repeat biopsy seems to be difficult in such cases. It has been observed that patients are subjected to many investigations like exploratory transcriptomics, multiparametric renal magnetic resonance imaging (MRI), many biomarker, potential predictive markers, biomarkers of tubular injury and genetics studies. etc which are not relevant to the progress of the diseases and the mechanism of action and effect of this drug," the panel noted.
“Looking to these investigations it seems the objectives and methodology are not focused…," the panel noted while concluding “Keeping in view of the above points committee opined that the firm should submit a revised protocol with detail methodology aligning with the specific objectives for further review by the committee."
Reviewing SEC Observations: Novartis
On being asked how the company plans to go ahead post SEC observations, Novartis spokesperson told News18, “As you will understand, it is quite early to comment on the next steps."
“We are in the process of reviewing the minutes by the SEC and post consultation with our global teams, we will take the necessary next steps," the spokesperson added.
It went ahead adding that the efficacy of the drug is already established in the bigger trials and in India, the company is planning only a small study.
“Having said that, we would like to point out that the said trial for LNP023 (iptacopan) is a small sample size study to investigate into deeper mechanisms that may possibly contribute to the already established efficacy of the medicine amongst patients with IgA nephropathy," the spokesperson said.
“As you understand, this molecule has demonstrated positive phase III outcomes for patients with IgA nephropathy for the primary endpoints of the trial post which Novartis has received US FDA accelerated approval for iptacopan, the first and only complement inhibitor, for the reduction of proteinuria in primary IgA nephropathy (IgAN). As we move forward, patient safety and improved outcomes will continue to remain our priority," Novartis said.
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