Respiratorius AB (publ.) has received Orphan Drug Designation for its drug candidate VAL001 following a decision by the European Commission.
VAL001 will at product launch be included in the EU Community Register of Orphan Medicinal Products.VAL001 is a combination of a HDAC inhibitor (valproic acid) and a steroid (prednisone) and referred to as a pretreatment before chemotherapy (R-CHOP), for the treatment of diffuse large B-cell lymphoma (DLBCL). DLBCL is an aggressive form of lymphoma, and the most common type of non-Hodgkin lymphoma, which constitute 30% of the patients diagnosed with this type of cancer. DLBCL annually affects about 60 000 people in the United States and Europe. The forecast for the 5-year survival with standard treatment is estimated at 60-70%.
A clinical phase I/IIa study of VAL001 is on-going, in which all included patients have been treated, at Skåne Oncology Clinic, SUS Lund/Malmö, Oncology Clinic at the University Hospital in Uppsala and at the Cancer Centre, University Hospital in Umeå. In the study, VAL001 is given as pretreatment prior to chemotherapy with the addition of rituximab (R-CHOP), in the treatment of DLBCL. Results of the interim analysis of the phase I/IIa study is showing that VAL001 combined with R-CHOP is significantly better than the reference group which consisted of a matched population of the Swedish lymphoma registry, which is only treated with R-CHOP, for both 1-year survival (95% significance) and 2-year survival (90% significance).
About Orphan Drug Designation
To obtain orphan drug designation in the EU, the new investigational drug product drug must be intended for the diagnosis, prevention or treatment of a life-threatening or chronically debilitating condition affecting no more than 5 in 10 000 persons in the EU and for which no satisfactory treatments exist or, where they do exist, where the new medicine will be of great benefit to those suffering from this condition. It can also be granted if a new product is not expected to provide a sufficient return to justify the investment in the development of a new drug.
In short, by obtaining orphan drug designation Respiratorius will obtain market exclusivity for ten years in the EU, from the date of marketing approval. In addition, Respiratorius will receive free consultation and scientific advice on contact with the European Medicinal Agency, which among other things includes support for designing clinical trial protocols and reduced costs for regulatory applications for the commercialization.
For further information, please contact:
+46 709-22 41 40
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About Respiratorius AB
Respiratorius AB (publ) is developing drug candidates with the goal to launch drugs for common diseases like cancer, chronic obstructive pulmonary disease (COPD) and severe asthma. In addition, the company portfolio also holds a project for improved diagnosis of certain cardiovascular diseases.