2023年4月28日

同種異體細胞療法批準為未來鋪平道路療法在這門課上同種異體SCT廣泛用於許多血液學的惡性腫瘤。美國食品和藥物管理局已批準Gamida細胞的幹細胞移植(SCT)治療Omisirge (omidubicel)治療血液癌症患者接受SCT。關鍵的第三階段試驗發現Omisirge優於標準臍帶血中性粒細胞移植的平均時間(12天與22天),年級2/3的發生率3年級細菌或真菌感染(39%和60%),血小板恢複(55%比35%恢複了42天),和時間的醫院在第一個100天(61年值,對48)。這個療法代表第一個同種異體細胞療法產品表示SCT FDA批準的和將地址一個真正的未滿足的需要病人找不到匹配的捐獻者。同種異體SCT被廣泛使用在許多血液學的惡性腫瘤,尤其是急性髓係白血病,急性淋巴細胞白血病、骨髓增生異常綜合征,在較小程度上,慢性粒細胞白血病。盡管SCT提供治愈的機會符合條件的患者,這個過程相當大的風險。成功的可能性是與如何相配的供體幹細胞是收件人,相關匹配,捐贈者提供最好的結果。對於那些沒有相關的捐助者,比賽可能會發現幹細胞捐獻者登記。然而,寄存器通常缺乏多樣性,導致非白種患者不太可能找到一個匹配,因此SCT後可能麵臨貧窮的結果。捐贈者不可用,可以使用臍帶血,這是病人人口Gamida Omisirge細胞最初將瞄準。 However, the company will also hope to expand into additional patient populations, particularly those for whom SCTs carry risks, such as where the donor source is mismatched, or where the patient’s minimal residual disease status would typically render them ineligible for transplant. The approval marks the end of a turbulent time for Gamida Cell, following two separate rounds of layoffs at the company. In November 2022 the FDA announced it required an additional three months to assess Gamida Cell’s application as additional information was considered a ‘major amendment’ to the application, while the application had also previously been delayed when the FDA requested information regarding the manufacturing of the product. Omisirge now has the potential to become the standard of care in the SCT setting for patients lacking a matched donor. Gamida Cell is not likely to be troubled by competitors in this space until 2025 when GlobalData anticipates the approval of Orca Biosystems’s TRGFT-201, which is currently in a Phase III trial. Other similar therapies in development include Marker Therapeutics’s zelenoleucel, and Nohla Therapeutics’s dilanubicel, both in Phase II. Omisirge represents an important advance in the use of allogeneic cell therapies and will be particularly important for improving outcomes in diverse patient groups who typically struggle to find appropriately matched donors. Importantly, by increasing access to SCT, this product can offer the possibility of a cure to increasing numbers of patients suffering from several serious haematological malignancies. Share this article