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news.The European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP) has adopted a positive opinion recommending Adaptimmune Therapeutics' SPEAR T-cell therapy targeting NY-ESO for designation as an orphan medicinal product for the treatment of soft tissue sarcoma, a solid tumor cancer.
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Adaptimmune previously received orphan drug destination from the U.S. Food and Drug Administration for its NY-ESO SPEAR T-cell therapy in this indication.
"While unresectable or metastatic soft tissue sarcomas are rare, they are associated with a high mortality rate," said Dr. Rafael Amado, Adaptimmune’s Chief Medical Officer.
"We are pleased to have received an opinion from the Committee for Orphan Medicinal Products which recognizes the unmet medical need that soft-tissue sarcomas represent. We look forward to working with them to advance our NY-ESO SPEAR T-cell therapeutic candidate through clinical evaluation, with the goal of one day bringing it to patients throughout Europe suffering from this disease."
The COMP adopts an opinion on the granting of orphan drug designation, after which the opinion is submitted to the European Commission for endorsement.
Orphan drug designation by the European Commission provides certain regulatory and financial incentives for companies to develop and market therapies that treat a life-threatening or chronically debilitating condition affecting no more than five in 10,000 persons in the European Union, and where no satisfactory treatment is available.
Orphan drug designation provides incentives for companies seeking protocol assistance and scientific advice from the EMA during the product development phase and a 10-year period of marketing exclusivity in the EU following product approval.
Data from recent published epidemiological studies estimate the prevalence of soft tissue sarcoma in the European Union to be 2.86 per 10,000 which corresponds to approximately 146,918 people based on the total population of 513.7 million people in the EU, Norway, Iceland, and Liechtenstein as of January 1, 2015 [EUROSTAT 2015].
Adaptimmune is developing its NY-ESO SPEAR T-cell therapy in certain soft tissue sarcomas. The company expects to initiate pivotal studies in synovial sarcoma in 4Q16/1Q17, and will explore development in myxoid round cell liposarcoma. Adaptimmune’s SPEAR T-cell candidates are novel cancer immunotherapies that have been engineered to target and destroy cancer cells by strengthening a patient’s natural T-cell response. T-cells are a type of white blood cell that play a central role in a person’s immune response.
Adaptimmune’s goal is to harness the power of the T-cell and, through its multiple therapeutic candidate, significantly impact cancer treatment and clinical outcomes of patients with solid and hematologic cancers.
About Soft Tissue Sarcoma
Soft tissue sarcomas can develop from soft tissues including fat, muscle, nerves, fibrous tissues, blood vessels, or deep skin tissues. There are approximately 50 types of soft tissue sarcomas, including synovial sarcoma, a cancer of the connective tissue around the joints. Soft tissue sarcomas can develop at almost any anatomic site, such as the extremities, trunk or thorax, abdomen and retroperitoneum, pelvis and the head and neck region.
The more common soft tissue sarcomas originate from muscle, nerve tissue, fat, or deep skin tissue. For a number of sarcomas, such as synovial sarcoma, the tissue origin is not well characterized. Surgical resection is the standard therapy for localized disease and radiation therapy (preoperative or postoperative) and/or chemotherapy is added in selected cases.