EU/3/20/2350: Orphan designation for the treatment of hypoparathyroidism

Poly(oxy-1,2-ethanediyl), alpha-hydro-omega-methoxy, ether with N-[[[2-[[6-[[1-[3-[[3-(2,3-dihydroxypropoxy)propyl]amino]-3-oxopropyl]-2,5-dioxo-3-pyrrolidinyl]thio]hexyl]amino]ethyl]amino]carbonyl]-2-methylalanyl-teriparatide (2:1)

Overview

On 19 October 2020, orphan designation EU/3/20/2350 was granted by the European Commission to Ascendis Pharma Bone Diseases A/S, Denmark, for poly(oxy-1,2-ethanediyl), alpha-hydro-omega-methoxy, ether with  N-[[[2-[[6-[[1-[3-[[3-(2,3-dihydroxypropoxy)propyl]amino]-3-oxopropyl]-2,5-dioxo-3-pyrrolidinyl]thio]hexyl]amino]ethyl]amino]carbonyl]-2-methylalanyl-teriparatide (2:1) (also known as TransConPTH) for the treatment of hypoparathyroidism.

Key facts

Active substance
Poly(oxy-1,2-ethanediyl), alpha-hydro-omega-methoxy, ether with N-[[[2-[[6-[[1-[3-[[3-(2,3-dihydroxypropoxy)propyl]amino]-3-oxopropyl]-2,5-dioxo-3-pyrrolidinyl]thio]hexyl]amino]ethyl]amino]carbonyl]-2-methylalanyl-teriparatide (2:1)
Intended use
Treatment of hypoparathyroidism
Orphan designation status
Positive
EU designation number
EU/3/20/2350
Date of designation
19/10/2020
Sponsor

Ascendis Pharma Bone Diseases A/S
Tuborg Boulevard 12
2900 Hellerup Hovedstaden
Denmark
Tel: + 4570222244
E-mail: MedicalAffairs@ascendispharma.com

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

Documents related to this orphan designation evaluation

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.

EU register of orphan medicines

The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:

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