LuxceptarGraft versus host disease (GvHD) in patients with haematological cancers - adjuvant therapy after haematopoietic stem cell transplant
New molecular entity
Development and Regulatory status
Nov 19CHMP meeting highlights indicate that at the time of the withdrawal, the EMA considered that the data presented were not adequate to prove that the medicine was effective: the main study was small, and the way it was designed did not allow the Agency to reach a conclusion on effectiveness .
Nov 19Kiadis Pharma terminates development of ATIR101 .
Nov 19Kiadis Pharma withdraw EU MAA .
May 19Kiadis Pharma announced that it has received the EMA´s day 180 second list of outstanding issues in the fourth quarter of 2018, and have spent the past few months conducting additional analysis of existing data to support a response. With the submission complete, Kiadis aim to receive an opinion from Committee for Medicinal Products for Human Use (CHMP) in 2019. If the CHMP opinion is positive, the company plan to launch in a European country at the end of 2019.
Dec 18Kiadis intends to commercially launch ATIR101 itself in a first EU member state in H2 19, and notes there are only 64 major transplant clinics in 4 EU countries (Germany, UK, Italy & France). In the US, they will need to file PIII data (currently study is ongoing), and notes that transplant provision is very concentrated with 27 clinics doing 50% of all transplants. Kiadis is setting up its own manufacturing facility, building up its own commercial organisation .
Jan 18The EMA has previously granted an Advanced Therapy Medicinal Product certificate to ATIR 101 for manufacturing quality and non-clinical data .
Jan 18Also has orphan drug status in US for reduction of transplant related mortality caused by graft versus host disease (GvHD) and/or infections following allogeneic bone marrow transplantation and as a therapy for immune reconstitution and prevention of GvHD following allogeneic bone marrow transplantation .
Jan 18Kiadis Pharma expects to receive a positive opinion and a conditional approval from the EMA during H2 2018 and launch the product in the EU in 2019 .
Sep 17ATIR101 granted regenerative medicine advanced therapy (RMAT) designation by the US FDA for the treatment of blood cancers .
Apr 17Filed in EU under the centralized procedure as an adjunctive treatment for haematopoietic stem cell transplantation (HSCT) for the treatment of malignant disease. The application was based on results of a single dose pivotal PII clinical trial and following positive interactions with the EMA Rapporteur and Co-Rapporteur .
Jun 16Kiadis intends to gain approval of the drug in blood cancers, & expects to launch the product in 2018. Kiadis also intends to file a marketing authorisation application with the FDA and Health Canada in 2019 .
Jun 16Kiadis Pharma intends to file a MAA with the EMA in Q1 2017 .
Jun 16ATIR101 is a designated orphan drug in the EU .
ATIR101 utilises the TH 9402 compound in conjunction with its TheraluxTM light exposure technology to selectively eliminate specific alloreactive T cells ex vivo from haploidentical donor samples before infusing the donor sample in the patient.
In 2013/14, 1,060 patients in the UK were transplanted with unrelated donor stem cells. It is estimated that between 20% and 80% of patients undergoing an allogeneic HSCT will develop some form of GvHD. In 2014-15, there were 2,144 admissions for bone marrow transplant rejection (ICD-10 T86.0) in England [1-3].
Graft versus host disease (GvHD) in patients with haematological cancers - adjuvant therapy after haematopoietic stem cell transplant
To be confirmed
Trial or other data
Nov 17PIII HATCY trial to compare the safety and efficacy of a haploidentical T-cell depleted haematopoietic stem cell transplantation (HSCT) and adjunctive treatment with ATIR101 versus a haploidentical T-cell replete HSCT with post-transplant administration of high dose cyclophosphamide (Baltimore protocol) in patients with a haematologic malignancies starts (CR-AIR-009; NCT02999854). Graft-versus-host disease-free and relapse-free survival, assessed until 2 years after HSCT, are the primary composite endpoints. The will enrol 250 patients in the EU, UK and US. Collection of primary outcome data is due to complete Feb 21. Kiadis intends to use the results of this trial as a basis for the filing of a Biologics License Application (BLA) with the US FDA. Moreover, the company also intends to use data from the trial support the conversion of the anticipated conditional marketing approval into standard marketing approval in the EU [11,12].
Jul 17PII study (NCT02500550) is still recruiting. Due to complete collecting primary outcome data in Apr 18 .
Aug 16PII (NCT02500550) study is still recruiting, & data collection should complete Mar 17 .
Dec 15First patient enrolled in an open-label, PII study, which is designed to assess the safety and efficacy of a two-dose regimen of ATIR101 immunotherapy in patients with haematologic malignancies, who received a CD34-selected haematopoietic stem cell transplantation from a haploidentical donor (CR-AIR-008; NCT02500550). Recruitment of approximately 15 patients is ongoing in Belgium, Canada, and Germany, and will expand to the UK. Incidence of acute graft versus host disease (GvHD) grade III/IV will be investigated as the primary endpoint. Kiadis intends to continue the PII trial into a randomised, controlled PIII pivotal study in the H2 2016 .
Mar 13PII (NCT01794299) study to determine whether ATIR is safe and effective in reducing transplant-related mortality and improving overall survival, when infused in patients with a hematologic malignancy following a T-cell depleted stem cell graft from a related haploidentical donor begins. 31 adults will be recruited in Belgium, Canada, Germany and the UK. Primary outcome is transplant-related mortality (TRM) at 6 months post HSCT; collection of these data should complete Jun 16 .