Key Points:
- Overview
This medicine was designated as an orphan medicine for the treatment of Mucopolysaccharidosis type IIIA (Sanfilippo A syndrome) on 12 January 2024.
- Orphan designation does not mean the medicine is available or authorised for use.
- All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.
- 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:
Patient,
Committee,
EMA,
IRIS,
COMP,
European Commission,
Marketing,
Laryngeal papillomatosis,
Antigen,
Medicine,
Pharmaceutical industry Key Points:
- Overview
This medicine was designated as an orphan medicine for the treatment of recurrent respiratory papillomatosis on 12 January 2024.
- Orphan designation does not mean the medicine is available or authorised for use.
- All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.
- 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:
Patient,
Committee,
Amyloidosis,
IRIS,
EMA,
COMP,
European Commission,
Marketing,
ATTR,
Medicine,
Pharmaceutical industry Key Points:
- Overview
This medicine was designated as an orphan medicine for the treatment of ATTR amyloidosis on 19 February 2024.
- Orphan designation does not mean the medicine is available or authorised for use.
- All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.
- 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:
Patient,
Committee,
Autotransplantation,
Amyloidosis,
IRIS,
EMA,
COMP,
European Commission,
Marketing,
Medicine,
Pharmaceutical industry Key Points:
- Overview
This medicine was designated as an orphan medicine for the treatment of AL amyloidosis on 19 February 2024.
- All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.
- For information on ongoing clinical trials in the EU, see:
Key facts
- Active substance
- Autologous CD3-positive T-cells expressing a chimeric antigen receptor against B cell maturation agent
- Intended use
- Treatment of AL amyloidosis
- Orphan designation status
- Positive
- EU designation number
- EU/3/24/2900
- Date of designation
- Sponsor
Raremoon Consulting Esp S.L.
- 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:
Key Points:
- Overview
This medicine was designated as an orphan medicine for the treatment of hereditary angioedema on 19 February 2024.
- Orphan designation does not mean the medicine is available or authorised for use.
- All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.
- 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:
Key Points:
- Overview
This medicine was designated as an orphan medicine for the treatment of acute respiratory distress syndrome on 19 February 2024.
- Orphan designation does not mean the medicine is available or authorised for use.
- All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.
- 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:
Patient,
Committee,
Carboplatin,
Glioma,
EMA,
IRIS,
COMP,
European Commission,
Marketing,
Medicine,
Pharmaceutical industry Key Points:
- Overview
This medicine was designated as an orphan medicine for the treatment of glioma on 19 February 2024.
- Orphan designation does not mean the medicine is available or authorised for use.
- All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.
- 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:
Key Points:
- Overview
This medicine was designated as an orphan medicine for the treatment of small lung cell cancer on 19 February 2024.
- Orphan designation does not mean the medicine is available or authorised for use.
- All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.
- 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:
Key Points:
- Overview
This medicine was designated as an orphan medicine for the treatment of ovarian cancer on 19 February 2024.
- Orphan designation does not mean the medicine is available or authorised for use.
- All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.
- 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:
Key Points:
- Overview
This medicine was designated as an orphan medicine for the treatment of fibrodysplasia ossificans progressiva on 19 February 2024.
- All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.
- For information on ongoing clinical trials in the EU, see:
Key facts
- Active substance
- Andecaliximab
- Intended use
- Treatment of fibrodysplasia ossificans progressiva
- Orphan designation status
- Positive
- EU designation number
- EU/3/24/2901
- Date of designation
- Sponsor
Regulatory Pharma Net S.r.l.
- 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:
