I saw a recent news article about the FDA granting accelerated approval for a liver disease drug. Here are some excerpts:
https://www.reuters.com/business/healthcare-p...024-06-10/
A few questions/comments:
1. Is this a candidate for ohm’s list? Since it is an autoimmune disease with inflammation, looks to a layman like a good candidate.
2. When a MASH/NASH trial is undertaken, might participants with diseases like PBC be included (or at least not excluded) so that this disease could possibly be incorporated into the subsequent FDA approval? Perhaps a special call-out is not required after LL is shown to improve MASH.
3. $11,500 for a one month’s supply – I don’t remember what ohm’s estimate was for Leronlimab, but this seems to be a pretty significant price for a drug that presumably is less efficacious than LL.
4. Since the FDA appears to be willing to give accelerated approvals (and with only 161 patients in this trial) in liver disease trials, what is the likelihood of that for Leronlimab? It should at least encourage the company to include an interim analysis during the trial. I wonder if they will include interim analyses in all trials.
Quote:US FDA grants accelerated approval to Genfit and Ipsen's liver disease drug
The U.S. Food and Drug Administration granted accelerated approval to French drugmakers Ipsen and Genfit's drug for a chronic inflammatory liver disease, Iqirvo, the companies said on Monday.
Primary biliary cholangitis (PBC) causes inflammation of the small bile ducts in the liver and eventually destroys them. It primarily affects women aged 30 to 60, impacting 75,000 in the United States.
Iqirvo's estimated price is $11,500 for a month's supply, and it is already available for healthcare providers to prescribe in the U.S., Ipsen said.
Iqirvo's approval was based on data from a 161-patient late-stage trial, in which it reduced levels of an enzyme called alkaline phosphatase that can lead to liver damage. It works by activating certain receptors, which reduce inflammation, increases transport of bile acids outside the liver and promotes their detoxification, said Christelle Huguet, Executive Vice President at Ipsen.
Current treatments do not address inflammation or scarring of the liver and do not promote detoxification of bile acids, Huguet added.
https://www.reuters.com/business/healthcare-p...024-06-10/
A few questions/comments:
1. Is this a candidate for ohm’s list? Since it is an autoimmune disease with inflammation, looks to a layman like a good candidate.
2. When a MASH/NASH trial is undertaken, might participants with diseases like PBC be included (or at least not excluded) so that this disease could possibly be incorporated into the subsequent FDA approval? Perhaps a special call-out is not required after LL is shown to improve MASH.
3. $11,500 for a one month’s supply – I don’t remember what ohm’s estimate was for Leronlimab, but this seems to be a pretty significant price for a drug that presumably is less efficacious than LL.
4. Since the FDA appears to be willing to give accelerated approvals (and with only 161 patients in this trial) in liver disease trials, what is the likelihood of that for Leronlimab? It should at least encourage the company to include an interim analysis during the trial. I wonder if they will include interim analyses in all trials.
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