Mustang Bio is seeking to conduct large clinical trials across multiple clinical sites and aims to file for a biological license application (BLA) by the end of 2021. The Investigational New Drug (IND) will be transferred back to Mustang Bio by the end of the year. Phase I/II trialsįor the Phase I/II trials, Mustang Bio had licensed MB-107 to St. This therapy has also been used in infants with severe infections, and the infections have been cleared after the infants received this gene therapy. After infusion of MB-107, the infants were able to return home with a working immune system. This allowed all of the patients to come out of isolation, which is the current method of protecting these patients from severe infections. Seven out of eight infants who received MB-107 achieved normalised level of CD3+, CD4+, naïve CD4+ T cells, and natural killer cells at the 16.4-month follow-up visit. X-SCID is caused by a mutation in the IL2RG gene, which codes for normal immune cell count. Therefore, a gene therapy with the potential to cure the disorder has the potential to seek reimbursement at a similarly high rate. Recent economic studies found that $100,000 per year of life saved was feasible for the introduction of newborn X-SCID screening. Without treatment, most patients will not survive past infancy. The costs associated with the care for X-SCID patients is extremely high the only treatment currently available for patients is a bone marrow transplant from a sibling donor, which is not a viable option for many patients. As such, about 70 children per year are born in the US with X-SCID. X-SCID is a genetic disorder that affects approximately one in 50,000 newborns. The company should also aim to compare MB-107’s cost with the total cost of currently managing patients with X-SCID. GlobalData believes that economic studies will be a vital step for MB-107 to achieve reimbursement, even after a demonstration of clinical efficacy in further trials. However, the Institute for Clinical and Economic Review (ICER) subsequently determined that the price should be 50–75% lower to be cost-effective.
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