Jeremy Bastid, Chief Development Officer of ORPHELIA Pharma, will attend the 2018 BIO International Convention, to be held in Boston, Massachusetts, June 4-7, 2018. Please meet us there on June 6 or 7 (contact: jeremy.bastid@orphelia-pharma.eu)
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Jeremy Bastid, Chief Development Officer of ORPHELIA Pharma, will attend the 2018 ASCO annual meeting to be held in Chicago, Illinois, June 1-5, 2018. Please meet us there (contact: jeremy.bastid@orphelia-pharma.eu).
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Jeremy Bastid, Chief Development Officer of ORPHELIA Pharma, will attend the Advances in Neuroblastoma Research (ANR) 2018 conference, “Building Bridges for Neuroblastoma Research” in San Francisco, California from May 9-12, 2018. Please meet us there (contact: jeremy.bastid@orphelia-pharma.eu).
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Paris and Lyon, February 27th 2018 – ORPHELIA Pharma, a French biopharmaceutical company dedicated to the development and marketing of pediatric drugs in the fields of oncology and neurology announces today that it has submitted a European Centralized Marketing Authorization Application (MAA) for Kigabeq® in 2017. Kigabeq® is the first pediatric formulation of vigabatrin, an essential antiepileptic drug, and will be available as scored soluble tablets of 100 mg and 500mg strengths. Kigabeq® is expected to be used as first-line treatment for infantile spasms (West syndrome), an extremely severe early childhood encephalopathy. Kigabeq has been exclusively developed for use in children and is eligible to a Pediatric Use Marketing Authorization (PUMA). « We take great pride in having developed this unique formulation, which is also our first product», comments Hugues Bienaymé, Founder and General Manager of ORPHELIA Pharma. « It seemed to us that it was of paramount interest to propose a new formulation adapted to the pediatric population, which represents the vast majority of patients treated with vigabatrin in Europe». « We supported the development of Kigabeq® from the beginning.» says Pr. Rima Nabbout, Head of the Pediatric Neurology Department at Necker – Enfants Malades Hospital. « The benefits of this new formulation rely in a better dose to body weight adjustment and a greater ease of use ». An acceptability, tolerance and pharmacokinetic study was carried out in 24 French hospitals between 2014 and 2016, with 38 children suffering from infantile spasms or pharmaco-resistant focal epilepsy. The results of this study, presented at the congress of the French Pediatric Neurology Society (SFNP), which took place in Bordeaux between 31st January and 2nd February 2018, demonstrated a good adherence to treatment and an accurate dose to body weight adjustment. About West Syndrome West Syndrome, or infantile spasms, is an early childhood epileptic encephalopathy which combines axial spasms in cluster, a marked psychomotor delay and an hypsarythmic electroencephalogram. It is a rare disease with an estimated incidence of around 0.05 per 10,000. It affects infants who were previously normal or children with impaired cognitive development. About ORPHELIA Pharma Based in Paris and Lyon, ORPHELIA Pharma SAS is a French biopharmaceutical company dedicated to the development and marketing of oncology and neurology pediatric drugs. Its mission is to significantly improve essential medicines administered to children through the development of efficient, reliable and safe drugs with child-friendly formulations. ORPHELIA Pharma also leads innovative R&D projects through […]
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