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Ascendis Pharma A/S Receives Orphan Designation for TransCona?? CNP for the Treatment of Achondroplasia in Europe

COPENHAGEN, Denmark, Aug. 12, 2020 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND), a biopharmaceutical company that utilizes its innovative TransCon technologies to address unmet medical needs, today announced the European Commission (EC) has granted Orphan Designation to TransCon C-Type Natriuretic Peptide (CNP) for the treatment of achondroplasia, the most common form of dwarfism.1 ATransCon CNP is a investigational long-acting prodrug of CNP, designed to provide continuous exposure of CNP at safe, therapeutic levels via a single, weekly subcutaneous dose. ATransCon CNP also received orphan designation for the treatment of achondroplasia in the United States (U.S.) in February 2019. ACurrently, there is no medical therapy approved for the treatment of achondroplasia.2
a??We now have orphan designations in Europe and the U.S. for TransCon CNP in achondroplasia, which we believe signifies the importance of developing a safe and effective therapy that can address the many medical complications these children face,a?? Dana Pizzuti, M.D., Senior Vice President of Development Operations at Ascendis Pharma. Aa??With our global phase 2 ACcomplisH Trial of TransCon CNP underway in achondroplasia, we are on track with the development of TransCon CNP and our receipt of the orphan designation in Europe is a key step towards developing a new treatment option for children with achondroplasia.a??Orphan Designation in the European Union (EU) is granted to therapies aimed at the treatment, prevention or diagnosis of a disease that is life-threatening or chronically debilitating, affects no more than five in 10,000 persons and for which no satisfactory therapy is available. AThe medicine also must be expected to provide significant benefit to those affected by the condition. AOrphan medicines have 10 years of market exclusivity after they receive marketing authorization in the EU. Under certain conditions, market exclusivity for pediatric indications may be extended for an additional two years.About TransCona?? Technology TransCon refers to a??transient conjugation.a?? AThe proprietary TransCon platform is an innovative technology designed to create new therapies that optimize therapeutic effect, including efficacy, safety and dosing frequency. ATransCon molecules have three components: an unmodified parent drug, an inert carrier that protects it, and a linker that temporarily binds the two. AWhen bound, the carrier inactivates and shields the parent drug from clearance. AWhen injected into the body, physiologic conditions (e.g., pH and temperature) initiate the release of the active, unmodified parent drug in a predictable manner. ABecause the parent drug is unmodified, its original mode of action may be maintained. ATransCon technology is designed to be applied broadly to a protein, peptide or small molecule in multiple therapeutic areas, and to be used systemically or locally.
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