FDA Grants Orphan Drug Designation for VAL001
Respiratorius AB has been granted Orphan Drug Designation by US Food and Drug Administration (FDA) for valproic acid for the treatment of Diffuse Large B-cell Lymphoma.
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, and constitute 30% of patients diagnosed with this type of cancer. Annually diagnosed approximately 20 000 people in the US with DLBCL. The forecast for the 5-year survival with standard treatment is estimated at 60-70%.
A clinical phase I/IIa study of VAL001 is underway and expected to be reported in first half of 2018. The study provided VAL001 as pretreatment prior to standard chemotherapy, chemotherapy plus rituximab (R-CHOP), in the treatment of DLBCL. Results of the interim analysis of the Phase I/IIa study showing that VAL001 combined with R-CHOP significantly better than the peer 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
The FDA, through the Office of Orphan Products Development (OOPD), grants Orphan Drug Designation (ODD - Orphan Drug Designation) to drugs and biological products intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 persons in the USA. ODD gives certain benefits and incentives, including market exclusivity for 7 years after marketing approval for the designated indication.
This information is information which Respiratorius AB (publ) is obliged to publish under the EU Market Abuse Regulation. The information was provided by the above contact person's auspices, for publication on April 3, 2017.
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Respiratorius AB (publ) is developing drug candidates with the goal to launch drugs for common diseases cancer, chronic obstructive pulmonary disease (COPD) and severe asthma. In addition, the project portfolio is a project for improved diagnosis of certain cardiovascular diseases.