European Medicines Grants Orphan Drug Designation to Resolaris for Girdle Muscular Dystrophy.
M2 PHARMA-March 6, 2017-European Medicines Grants Orphan Drug Designation to Resolaris for Girdle Muscular Dystrophy
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- The European Medicines Agency has granted orphan drug designation to Resolaris for the treatment of limb girdle muscular dystrophy patients, US-based biotherapeutics company aTyr Pharma, Inc. (NASDAQ: LIFE) said.
aTyr Pharma is developing Resolaris as a potential first-in-class intravenous protein therapeutic for the treatment of rare myopathies with an immune component.
Resolaris is derived from a naturally occurring protein released by human skeletal muscle cells. aTyr believes Resolaris has the potential to provide therapeutic benefit to patients with rare myopathies with an immune component characterized by excessive immune cell involvement.
EMA orphan designation is intended to encourage development of medicines for the diagnosis, treatment, or prevention of life-threatening or chronically debilitating conditions that affect no more than 5 in 10,000 people in the EU.
Medicines that receive orphan designation are eligible for a number of incentives, including assistance with development of the medicine; reduced fees for marketing-authorization applications; and extended market exclusivity once the medicine is authorized.
Orphan designation is conferred following a positive opinion by the EMA's Committee for Orphan Medicinal Products.
Limb girdle muscular dystrophy refers to a group of rare genetic myopathies, of which there are more than 20 different subtypes, none with approved therapies. LGMD affects an estimated 16,000 patients in the US, approximately 3,000 of whom have LGMD2B.
LGMD2B is a recessive genetic disease caused by a toxic loss of function in the dysferlin gene. Patients experience progressive debilitating muscle weakness and atrophy as well as immune cell invasion in the skeletal muscle.
aTyr Pharma is engaged in the discovery and clinical development of innovative medicines for patients suffering from severe, rare diseases using its knowledge of Physiocrine biology, a newly discovered set of physiological pathways.
The company's lead candidate, Resolaris, is a potential first-in-class intravenous protein therapeutic for the treatment of rare myopathies with an immune component.
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|Date:||Mar 6, 2017|
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