Amniotics receives positive opinion on orphan designation for PulmoStem® from the European Medicines Agency for the Treatment of Primary Graft Dysfunction following Lung Transplantation


November 20, 2023

Amniotics AB (publ) (Nasdaq Stockholm: AMNI) today announced that the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending PulmoStem (AmnioPul-02) designation as orphan medicinal product for the treatment of Primary Graft Dysfunction (PGD) following lung transplantation.

PulmoStem is an advanced therapy medicinal product (ATMP), consisting of allogeneic mesenchymal stem cells (MSC) extracted from term amniotic fluid and selected with antibodies against a lung-relevant cell surface marker, developed for the treatment of PGD following lung transplantation. Amniotics expects to start a clinical study evaluating PulmoStem in this indication during 2024.
Marcus Larsson, CEO, Amniotics AB, commented: “We are very happy to receive a positive opinion for orphan designation from COMP/EMA for PulmoStem for the prevention of PGD. There is a substantial medical need for treatment options for the significant number of patients succumbing to this severe complication after lung transplantations. The positive opinion adopted by the EMA gives increased credibility to our research and our clinical program. We look forward to initiating our Phase I study in PGD with PulmoStem in 2024.”
PGD after lung transplantation represents a multifactorial parenchymal injury and dysfunction to the transplanted lung that develops in the first 72 hours after transplantation in the absence of identifiable secondary causes. PGD of the lung occurs in approximately 20% to 30% of lung transplant recipients and is characterized by poor oxygenation, low pulmonary compliance and is a significant risk factor for death in short and long term. There is no current treatment for PGD except for prolonged hospitalization and advanced intensive care. Per the opinion of EMA there is a need for more efficacious treatment options. Amniotics background data indicate that PulmoStem has the potential to significantly reduce lung damage and increase survival in this patient population.
In the EU, orphan drug status is given to products that treat, prevent, or diagnose a disease which is life-threatening or chronically debilitating and affects less than 5 in 10,000 people across the EU. Sponsors who obtain orphan designation in the EU benefit from protocol assistance, a type of scientific advice specific for designated orphan medicines, waivers or reductions of certain fees as well as a ten-year market exclusivity once the medicine is on the market. The European Commission is responsible for granting the orphan designation based on the positive opinion from EMA. For more information about orphan designation in the EU, please see

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