dm+d

Unassigned

New Medicines

NiCordAcute lymphoblastic leukemia (ALL), acute myeloid leukemia (AML), myelodysplastic syndrome (MS), chronic myeloid leukemia (CML) or lymphoma

Information

NiCord
New molecular entity
Gamida Cell
Gamida Cell

Development and Regulatory status

None
Phase III Clinical Trials
Pre-registration (Filed)
Yes
Yes
Aug 22FDA has accepted the BLA for omidubicel and has granted a priority review with a target action date of 30 Jan 2023 [25]
Jun 22Gamida announces completion of the rolling BLA to the FDA [24]
Feb 22 Gamida announces that it has initiated the BLA rolling submission process with the FDA. The company remains on track to complete the BLA submission in the second quarter of 2022 [23]
Nov 21Gamida cell announces it has conducted a pre-BLA meeting with the FDA, who have requested a revised analysis of manufacturing data of the drug, generated at the commercial manufacturing facility. Gamida Cell plans to file a full BLA in H1 22 [22].
Jul 21Company has no plans to develop this for the UK [21].
Jul 21Gamida Cell are planning for a US launch in 2022. Commercial manufacturing will be at the Lonza Netherlands facility and at their own Kiryat Gat facility (construction completed in 2020 and qualification for BLA filing underway) [18,19].
Feb 21Gamida Cell anticipate filing BLA with US FDA H2 21 [16].
Oct 20Gamida Cel anticipates initiating rolling BLA submission in Q4 20. It anticipates initial commercial supply to be produced by Lonza Technology. A scalable, Gamida Cell-owned manufacturing facility is planned which will further enable reliable, consistent supply - construction is complete and validation expected to be complete by year-end 2020. Commercial launch in US planned for H2 21. No mention of EU/UK plans [14,15].
Oct 20Gamida Cell expands its agreement with Be The Match BioTherapies® to provide a smooth omidubicel therapy supply chain, to enable access to patients in clinical and commercial settings. Under the terms of expanded agreement, Gamida Cell will work through Be The Match BioTherapies for the ordering and supply of cord blood units, which serve as the starting material for omidubicel [13].
Feb 20In its latest annual report, Gamida Cell states that it plans to seek regulatory approval for omidubicel in the US and EU. Approval and commercialisation of omidubicel may be in collaboration with strategic partners, particularly in Europe. No timeline is given for EU approval (unlike for US approval) [11].
Jan 20Gamida Cell expected milestones for 2020/21 include reporting topline data from the PIII study in H1 20, submit a biologics licence application to the FDA in H2 20, assuming positive data and launch omidubicel in 2021, contingent upon FDA approval. Plans for EU development not stated [10].
May 19Gamida Cell plans to file in 2020 [7]
Jul 18US FDA granted orphan drug designation for NiCord® for treatment of haematopoietic stem cell transplantation.[2]
Mar 17EMA´s Committee for Orphan Medicinal Products (COMP) granded orphan drug designation for NiCord® for treatment of haematopoietic stem cell transplantation.[2]
Oct 16US FDA granted Breakthrough Therapy Designation for NiCord®, for bone marrow transplantation in pts with high risk haematological malignancies such as leukaemia and lymphoma. [2]

Category

First-in-class stem cell therapy composed of umbilical cord-derived ex vivo expanded stem and progenitor cells
Annual incidence rate of all haematological malignancies in the UK is 68.2 per 100,000 people [6].
Acute lymphoblastic leukemia (ALL), acute myeloid leukemia (AML), myelodysplastic syndrome (MS), chronic myeloid leukemia (CML) or lymphoma
Intravenous infusion

Trial or other data

Jun 21PIII study results published in Blood, https://doi.org/10.1182/blood.2021011719 [20].
Feb 21Detailed results announced from PIII study showing the median time to neutrophil engraftment was 12 days with omidubicel vs. 22 days for the comparator group using cord blood units (p<0.001). Platelet engraftment was significantly accelerated with omidubicel; 55% on omidubicel achieving platelet engraftment at day 42 vs. 35% with the compator (p = 0.028). The infection rate was significantly reduced with omidubicel; the cumulative incidence of serious bacterial or invasive fungal infection with omidubicel was 37% with omidubicel vs. 57% with the comparator (p = 0.027). Hospitalization in the first 100 days after transplant was also reduced in patients on midubicel; median no of number of days alive and out of hospital with omidubicel was 60.5 daysvs. 48 days with the comparator (p = 0.005).[17]
Oct 20Gamida Cell announces PIII study (NCT02730299) met its secondary endpoints related to platelet engraftment, infections and hospitalisations [14].
May 20PIII trial topline results from NCT02730299 on the safety and efficacy of omidubicel found the median time to neutrophil engraftment was 12 days for patients randomized to omidubicel compared to 22 days for the comparator group (p<0.001) [12].
Jan 20In December 2019 the company completed patient enrollment in its Phase 3 study of the company’s lead clinical program [9].
Jan 20UK trial sites - Marsden, Birmingham, Leeds, Manchester [8]
Jan 19A PIII open-label, controlled, multicenter, international, pivotal PIII, randomised study of NiCord® vs. transplantation of 1-2 unmanipulated, unrelated cord blood units is currently recruiting ~120 pts with ALL, AML, CML or lymphoma, all with required disease features rendering them eligible for allogeneic transplantation. The primary outcome measure will be the time to neutrophil engraftment in pts on or before 42 days post transplant. Topline data are expected in early 2020.[3,5]
Jun 18In a PI/II single-arm, open-label trial study (NCT01816230; EudraCT2014-000074-19), 43 adult pts with high-risk haematological malignancies in the US, Italy, the Netherlands, Singapore and Spain undergoing transplantation with a single unit of NiCord had rapid and long-lasting engraftment of neutrophils and platelets vs. pts who received cord blood alone. The primary endpoint measure was the cumulative incidence of pts with NiCord®-derived neutrophil engraftment at 42 days following transplantation. NiCord treatment was associated with a significant reduction in the rate and severity of infections as well as significantly shorter hospital stays. Gamida presented positive results at the 52nd Annual Meeting of the American Society of Clinical Oncology showing improved engraftment, reduced morbidity and less transplant related mortality compared to controls. [1-4]