More interestng view from ET....
Another pointer to likelihood of FDA approval for Meso's aGvHD.The Prof has indicated that we will have FDA meetings this quarter for aGvHD (OTAT manufacturing, potency assays and consistency meeting, with expectations Meso may just be able to refile most of the original BLA with no need for an additional trial) and also for Chronic Low Back Pain and Chronic Heart Failure (both pathway to approval meetings with expectations of another trial required).These meeting dates have not yet been disclosed to the market, and I assume that the company will announce them when they are set by the FDA. Assuming the FDA will give Meso a month's notice, that would mean we might start expecting them any time from mid-November, however that is a guess and they could be earlier in the quarter or later.Remember, over a year ago, the Adcomm voted 9:1 in favour of Mesoblast, and then the FDA issued a Complete Response Letter requesting more information.By way of comparison, after a tight advisory committee vote back in May that tilted just slightly in favor of approving ChemoCentryx’s avacopan, the FDA has just officially cleared the drug to treat a rare and serious disease known as anti-neutrophil cytoplasmic autoantibody (ANCA)-vasculitis. Avacopan is sold as Tavneos and its price will fall somewhere between $150,000 to $200,000 per patient per year. Some of the avacopan adcomm members raised concerns about relying on the single trial as evidence, the insufficient amount of safety data, and questions on whether the trial was statistically robust enough.Mesoblast's treatment for aGvHD in children is also addressing rare and serious diseases (up to 90% mortality in the severe forms involving multiple organs).Mesoblast is often questioned about the cost of its treatment - it is expected to cost around $300,000 for 4 weeks of treatment, with 2 doses per week and no ongoing annual cost as in the case of avacopan.Mesoblast was criticised by some investors for running a single arm trial even though the trial doctors said they considered it unethical to run a placebo control group of children who were at a very high risk of death and who would receive no treatment. Well the avacopan trial shows yet again that it is possible for the FDA to approve a drug based on a single arm trial.The Mesoblast adcom voted 9:1 that the mesenchymal stromal cell (MSC) product showed evidence of efficacy as a treatment for steroid-refractory acute graft-vs.-host disease (GVHD) in children.In contrast to the clear vote in favour of Mesoblast by the ODAC, in the case of the avacopan decision the FDA questioned if the other drugs used in the study may have contributed to the efficacy seen for avacopan, saying: “In this case, both treatment arms received background therapy in the form of cyclophosphamide or rituximab. The benefit of glucocorticoids on top of cyclophosphamide or rituximab is not well understood,” FDA statistical reviewer Yura Kim said back in May. “As a result, it is difficult to determine if similar remission rates observed on both arms can support a conclusion that avacopan is effective or if similarities can be primarily attributed to both arms receiving rituximab or cyclophosphamide.” The avocapan committee ended up voting 10-8 to recommend approving the drug; 9-9 on whether the efficacy data support approval; and 10-8 that the safety profile of avacopan is adequate enough to support approval.In the light of this approval given to avocapan, I believe Mesoblast is highly likely to receive an approval for aGvHD without the need for an extra trial, once it can address the FDA's questions on potency assays and manufacturing consistency of the relevant factors as shown by the biomarker data.In comparison with avocapan, Mesoblast's cells reduction in mortality was not disputed, they similarly treat a rare and deadly condition with no other treatment, they are in a similar price range (and cheaper if the ANCA-vasculitis disease is treated for more than 18 months to two years) and Meso had a much better adcom vote than avocapan; furthermore there are no doubts abouts Mesoblast's safety data and the statistical benefit is robust.The more you look at other drugs being approved by the FDA, the more the Mesoblast CRL looks like an anomaly, and one which is likely to be fixed by adequately addressing the FDA's questions in the upcoming OTAT meeting.
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