Sanofi's efdoralprin alfa gets orphan designation in EU for AAT deficiency emphysema treatment.
Sanofi has received orphan designation in the EU for efdoralprin alfa in treating emphysema related to alpha-1 antitrypsin deficiency. This designation provides incentives to develop treatments for rare diseases affecting fewer than 5 in 10,000 people in the EU. Efdoralprin alfa aims to address the needs of patients suffering from this specific type of emphysema, which is a debilitating lung condition. This designation will grant Sanofi market exclusivity for efdoralprin alfa for 10 years in the EU, upon its approval for this indication.
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