European Medicines Agency Pre-authorisation Evaluation of Medicines for Human Use
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E-mail: orphandrugs@emea.eu.int a.eu.int
EMEA 2005 Reproduction and/or distribution of this document is authorised for non-commercial purposes only provided the EMEA is acknowledged
Document Date: London, 1 July 2005
能量隔离Doc.Ref.: EMEA/COMP/198295/2004 COMMITTEE FOR ORPHAN MEDICINAL PRODUCTS
PUBLIC SUMMARY OF
POSITIVE OPINION FOR ORPHAN DESIGNATION
OF
pirfenidone
for the treatment of idiopathic pulmonary fibrosis
电插头
On 16 November 2004, orphan designation (EU/3/04/241) was granted by the European Commission to Uppsala Medical Information System AB, Sweden, for pirfenidone for the treatment of idiopathic pulmonary fibrosis.
What is idiopathic pulmonary fibrosis?
Fibrosis is the formation of scar tissue as part of the natural repair process of the body following tissue damage. Idiopathic pulmonary fibrosis consists of a chronic inflammation (a response of the body to the injury caused to the tissue) and progressive formation of fibrous tissue in the walls of the small chambers containing air in the lungs. Since the injury causing these changes is unknown, it is called idiopathic. The progressive formation of scars impairs the normal functions of lung tissue, which are to enable exchange of oxygen and carbon dioxide between air and blood. The symptoms
developed are persistent cough and progressive severe shortness of breath.
Idiopathic pulmonary fibrosis is a chronically debilitating and life threatening disease due to the progression of symptoms, severe respiratory complications and short life expectancy.
What are the methods of treatment available?
No satisfactory methods exist that were authorised at the time of application. Only symptomatic treatments to reduce the inflammation were used (corticosteroids and medicinal products that suppress the immune system) or for some patients lung transplantation was performed.
What is the estimated number of patients affected by the condition*?
According to the information provided by the sponsor, idiopathic pulmonary fibrosis was considered to affect about 138,000 persons in the European Union.
How is this medicinal product expected to act?
Scar formation (fibrosis) is regulated by several substances produced in the body during the inflammation that precedes fibrosis. These substances stimulate the so-called fibroblasts (the main c
ells responsible for formation of the fibrosis). Although it is not yet fully understood how pirfenidone acts in idiopathic pulmonary fibrosis, it could reduce the action on these substances and thereby reduce the scar formation.
永磁电机设计What is the stage of development of this medicinal product? The effects of pirfenidone were evaluated in experimental models.
At the time of submission of the application for orphan designation, several clinical trials in patients with idiopathic pulmonary fibrosis were completed.
Pirfenidone was not marketed anywhere worldwide for idiopathic pulmonary fibrosis, at the time of submission. Orphan designation of pirfenidone was granted in the United States for idiopathic pulmonary fibrosis.
According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted on 7 October 2004 a positive opinion recommending the grant of the above-mentioned designation.
__________________________
Opinions on orphan medicinal products designations are based on the following cumulative criteria: (i) the seriousness of the condition, (ii) the existence or not of alternative methods of diagnosis, prevention or treatment and (iii) either the rarity of the condition (considered to affect not more than five in ten thousand persons in the Community) or the insufficient return of development investments. Designated orphan medicinal products are still investigational products which were considered for designation on the basis of potential activity. An orphan designation is not a marketing authorisation. As a consequence, demonstration of the quality, safety and efficacy will be necessary before this product can be granted a marketing authorisation.
For more information:
Sponsor’s contact details:
Uppsala Medical Information System AB
Döbelnsgatan 32C
752 37 Uppsala
Sweden
Telephone: +46 70 565 81 35
Telefax: +46 18 50 15 00
E-mail: info@uppsalamedical
Patients’ associations contact points: Not available
主轴加工*Disclaimer: For the purpose of the designation, the number of patients affected by the condition is estimated and assessed based on data from the European Union (EU 25), Norway, Iceland and Lichtenstein. This represents a population of 459,700,000 (Eurostat 2004). This estimate is based on available information and calculations presented by the sponsor at the time of the application. EMEA 2005
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EMEA 2005
Translations of the active ingredient and indication in all EU languages
and Norwegian and Icelandic
Language Active Ingredient Indication
English Pirfenidone Treatment of idiopathic pulmonary fibrosis Czech Pirfenidon Léčba idiopatické plicní fibrózy
Danish Pirfenidon Behandling af idiopatisk lungefibrose
Dutch Pirfenidon Behandeling van idiopathische longfibrose Estonian Pirfenidoon Idiopaatilise kopsufibroosi ravi
Finnish Pirfenidoni Idiopaattisen keuhkofibroosin hoito
French Pirfenidone Traitement de la fibrose pulmonaire idiopathique German Pirfenidon Behandlung von Idiopathischer Pulmonaler Fibrose Greek Pirfenidone Θεραπεία της ιδιοπαθούς πνευµονικής ίνωσης Hungarian Pirfenidon Idiopathiás tüd őfibrózis kezelése
Italian Pirfenidone Trattamento della fibrosi polmonare idiopatica Latvian Pirfenidons Idiop ātisk ās plaušu fibrozes ārst ēšana
自动垃圾桶Lithuanian Pirfenidonas Idiopatin ės plau či ų fibroz ės gydymas Maltese Pirfenidone Treatment of idiopathic pulmonary fibrosis Polish Pirfenidon Leczenie idiopatycznego zwłóknienia płuc Portuguese Pirfenidona Tratamento da fibrose pulmonar idiopática Slovak Pirfenidon Lie čba idiopatickej p ľúcnej fibrózy
Slovenian Pirfenidon Zdravljenje idiopatske plju čne fibroze
Spanish Pirfenidona Tratamiento de la fibrosis pulmonar idiopática Swedish Pirfenidone Behandling av idiopatisk lungfibros
Norwegian Pirfenidon Behandling av idiopatisk lungefibrose
挂包钩Icelandic Pírfenídón Meðferð sjálfvakinnar bandvefsmyndunar í lungum.
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