LEUKOTAC® takes next step in paediatric acute steroid-resistant GvHD
ELSALYS BIOTECH plans to submit to the European Medicines Agency before summer 2018 a Paediatric Investigation Plan (PIP) proposing to conduct a prospective clinical study in the paediatric population
The management of acute steroid-resistant graft versus host disease (SR-aGvHD*), the indication for LEUKOTAC®, is even more critical in paediatric patients than in adults as the limitations of therapeutic options are even worse
Lyon, FRANCE, 05 April 2018, ELSALYS BIOTECH, a new player in immuno-oncology, met paediatric transplant physicians at the 44th EBMT congress that took place in Lisbon, Portugal, from 18 to 21 March. The Company presented the progress made on LEUKOTAC® (inolimomab), its monoclonal antibody that has demonstrated promising clinical results in the treatment of SR-aGvHD and addressed in particular the objectives and challenges of the upcoming paediatric investigation plan.
Retrospective analysis ongoing in about 150 children treated with LEUKOTAC®
In the context of the early access programme (Temporary Use Authorisation** ) granted by the French medicine agency (ANSM), over 250 children suffering from SR-aGvHD were treated with LEUKOTAC® in compassionate use, mainly between 2005 and 2015. This antibody is well known by the French paediatric physicians specialised in bone marrow transplantation.
Efficacy and safety data of LEUKOTAC® were collected retrospectively among 147 patients and are being analysed. In a heterogeneous paediatric population – in terms of age and origin of the disease conducting to bone marrow transplantation – the main objectives of this analysis are to identify the optimal dosing, the rate of response as well as the duration of the response together with the overall impact on survival after treatment of this population with LEUKOTAC®. The data may allow a more precise identification of the patient typologies who are most likely to benefit from treatment and will be used to design the prospective clinical study in the paediatric population that will later be conducted within the frame of a Paediatric Investigation Plan.
Paediatric Investigation Plan to be filed before summer
The planned study will be multicentric in Europe, even North America. It is expected to involve approximately 60 to 80 patients in a non-randomised study (without control arm, in other words all patients will receive LEUKOTAC®).
«The medical need represented by steroid-resistant aGvHD among children under the age of 18 is major and critical. The therapeutic solutions in this population are very limited, also coming from clinical trials given that treatments are always validated in adults before in children. The paediatric physicians who used LEUKOTAC® say that since the product has no longer been available they have had difficulties and they support making the product available again at the earliest opportunity,» explains Dr. David LIENS, Chief Medical Officer, ELSALYS BIOTECH.
«We are preparing a paediatric investigation plan that proposes to conduct a prospective clinical study in children with the support of several French and European clinicians. This plan, which is an obligation under European regulation, will be submitted before summer to the European Medicines Agency that is expected to welcome this proposal since it is not common to propose studies in these populations. It is an ambitious timing but it is equal to the challenges faced by young patients in the absence of any therapeutic solution,» adds Dr. Catherine MATHIS, Chief Operations Officer, ELSALYS BIOTECH.