The European Medicines Agency (EMA) has officially qualified PolTREG to submit an application for marketing authorisation in the European Union for the PolTREG-T1D cell preparation.
PolTREG S.A. (WSE: PTG) announced that it has achieved a significant milestone in the registration process of a cell therapy for the treatment of symptomatic type 1 diabetes within the European Union.
The European Medicines Agency (EMA) officially confirmed that the Committee for Medicinal Products for Human Use (CHMP), based on the studies and documentation submitted by the Company, qualified the product Tregs cells preparation – PolTREG-T1D (PTG-007) for submission of an application for marketing authorisation under the centralised procedure in the European Union.
The decision of the Committee for Medicinal Products for Human Use (CHMP) was made after analysing the clinical trial results presented by PolTREG to date. The Committee highly assessed the substantive quality of the documentation, which enables the Company to seek market authorisation that also includes a paediatric indication. This is important because type 1 diabetes (T1D) most often begins in childhood. In the youngest patients, the autoimmune process is the most aggressive and, as a consequence, the pancreatic cells responsible for insulin secretion are completely destroyed, and the patient is subjected for life to a regimen of daily glycaemic monitoring and insulin supplementation.
PolTREG submitted for the registration application unique worldwide data from 7–12 years of patient follow-up (clinical trials conducted by PolTREG), which confirmed that Tregs therapy not only has an unprecedented safety profile but, above all, allows the progression of the disease to be halted and enables long-term preservation of the patient’s own (endogenous) insulin secretion. EMA originally expected 5-year data—PolTREG delivered scientific evidence covering more than a decade, which places the PTG-007 project in a “best-in-class” position.
“EMA’s decision confirms the highest quality of our therapy and is a huge step toward commercialisation. The fact that CHMP has qualified our therapy for the centralised procedure means that our clinical data—including the unique 12-year follow-up results—have been recognised as a solid foundation for registering the therapy on the European market. We are the first company in the world to provide such long-term evidence of the efficacy and safety of Tregs. We see that regulators appreciate our contribution to the development of targeted medicine which, unlike insulin therapy, targets the very cause of the disease rather than only its symptoms. The registration process at EMA concerns the treatment of type 1 diabetes in the early symptomatic phase (stage 3). In the presymptomatic phase of type 1 diabetes (stages 1 and 2), we are currently conducting clinical trials in children in Poland and we also plan them in the USA.” – says Prof. Piotr Trzonkowski, MD, PhD, co-founder and CEO of PolTREG S.A.
Positive qualification by EMA places PolTREG at the centre of attention of the global pharmaceutical industry. The history of the biotechnology market shows that the registration of an innovative therapy in type 1 diabetes is a direct factor leading to multi-billion-dollar transactions.
An example of strategic importance is teplizumab (Tzield)—the first therapy that inhibits the progression of type 1 diabetes. Tzield is used in children from the age of 8 when diabetes symptoms have not yet appeared, although signs such as fluctuations in blood sugar levels are already present, i.e. patients are entering stage 2 of diabetes development. After obtaining positive data and registration, Provention Bio, which developed this therapy, was acquired in 2023 by Sanofi for approximately USD 2.9 billion. Studies have shown that PolTREG’s therapy demonstrates an advantage over teplizumab in terms of a longer period of inhibiting disease progression, no need for hospitalisation, and a significantly better safety profile (no systemic immunosuppression), making it an even more attractive asset for Big Pharma.
Further steps at EMA. Confirmation of eligibility launches the formal registration timetable:
- designation at EMA, at the Company’s request, of a dedicated administrative and scientific team handling the registration process;
- submission of the full dossier (MAA): the Company is finalising preparation of the complete technical and clinical documentation;
- after submission, the substantive assessment phase begins (usually taking approx. 210 days of active assessment time, excluding so-called clock-stops for agency questions);
- after a positive CHMP opinion, the European Commission issues a centralised marketing authorisation valid in all EU and EEA countries.
PolTREG estimates that the full schedule of meetings and assessments (the so-called EMA calendar) will be specified in more detail within the next two months.
