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Home » ORPHAN DESIGNATION: SODIUM (4-{(E)-3-(4-FLUOROPHENYL)-3-[4-(3-MORPHOLIN-4-YL-PROP1YNYL)PHENYL]ALLYLOXY}-2-METHYLPHENOXY)ACETATE, TREATMENT OF LONG-CHAIN 3-HYDROXYACYL-COENZYME A DEHYDROGENASE DEFICIENCY, 21/08/2020, POSITIVE

ORPHAN DESIGNATION: SODIUM (4-{(E)-3-(4-FLUOROPHENYL)-3-[4-(3-MORPHOLIN-4-YL-PROP1YNYL)PHENYL]ALLYLOXY}-2-METHYLPHENOXY)ACETATE, TREATMENT OF LONG-CHAIN 3-HYDROXYACYL-COENZYME A DEHYDROGENASE DEFICIENCY, 21/08/2020, POSITIVE

(AGENPARL) – LONDON (UNITED KINGDOM), mer 25 novembre 2020

At the time of designation, LCHAD deficiency affected less than 0.5 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 26,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This is based on the information provided by the sponsor and the knowledge of the Committee for Orphan Medicinal Products (COMP).


*For the purpose of the designation, the number of patients affected by the condition is estimated and assessed on the basis of data from the European Union, Iceland, Liechtenstein, Norway and the United Kingdom. This represents a population of 519,200,000 (Eurostat 2020).

Fonte/Source: https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3202319

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