DERIO, Spain.- Atlas Molecular Pharma, a private company founded in 2015 in Derio (Spain) as a spin-off from the Centre for Co-operative Research in Biosciences (CIC bioGUNE), announced today a favourable report, as Orphan Medicinal Product, from the European Medicines Agency (EMA) for its candidate drug (L2.7.D7) for the treatment of Congenital Erythropoietic Porphyria.
“The favorable report from the European Medicines Agency is a key milestone in the development of L2.7.D7 for the treatment of Congenital Erythropoietic Porphyria, as it certifies that this molecule has the appropriate pharmacological activity, both “in vitro” and “in vivo”, having shown efficacy in mice with a similar pathology than humans”, said Dr. Emilio Díez, CEO of Atlas.
Dr. Oscar Millet, Director of the Laboratory of Protein Stability and Inherited Disease of the CIC bioGUNE, said that “understanding the molecular mechanism of this disease has enabled us to design a therapy based on pharmacological chaperones, molecules that bind to the defective protein fixing its stability problem and reversing its pathogenic effects”.
The EMA´s Orphan Medicinal Product program provides orphan status to drugs and biologics that are being developed to address rare diseases or disorders that affect less than five people in every 10,000 in Europe. With the designation of L2.7.D7 as Orphan Medicinal Product, Atlas will qualify for various incentives that will facilitate the launching of clinical trials on patients in the near future. These incentives include scientific advice and high-quality clinical trial protocol assistance, leading to effective and acceptably safe medicines for the benefit of patients.
This is the first time that an Orphan Medicinal Product has received a favourable report by the European Medicines Agency in the Basque Country.
About Congenital Erythropoietic Porphyria
Congenital erythropoietic porphyria (CEP) is a very rare inherited metabolic disorder resulting from the deficient function of the enzyme uroporphyrinogen lll cosynthase (UROS), the fourth enzyme in the heme biosynthetic pathway. Due to the impaired function of this enzyme, excessive amounts of particular porphyrins accumulate, particularly in the bone marrow, plasma, red blood cells, urine, teeth, and bones. The major symptom of this disorder is hypersensitivity of the skin to sunlight and some types of artificial light, bone loss, and deformities. Available treatments only relieve the symptomathology and currently there is no curative therapy for this disease.