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PARALLEL TEXTS
Il Tribunale annulla la decisione con cui la Commissione ha negato l’autorizzazione all’immissione in commercio dell’Orphacol
Inglese tratto da:
http://europa.eu/rapid/press-release_CJE-13-84_en.htm
Italiano tratto da:
http://europa.eu/rapid/press-release_CJE-13-84_it.htm
Data documento: 04-07-2013
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The General Court annuls the Commission’s decision refusing to grant a marketing authorisation for Orphacol
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Il Tribunale annulla la decisione con cui la Commissione ha negato l’autorizzazione all’immissione in commercio dell’Orphacol
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A marketing authorisation may be issued in respect of that orphan medicinal product on the basis of well-established medicinal use dating back more than 10 years
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L’autorizzazione all’immissione in commercio può essere rilasciata a tale medicinale orfano in considerazione di un impiego medico ben consolidato da oltre dieci anni
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EU law states that any marketing authorisation (MA’) application regarding a medicinal product for human use must be accompanied by the results of the pre-clinical tests and clinical trials of the medicinal product concerned.
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Il diritto dell’Unione europea prevede che ogni domanda di autorizzazione all’immissione in commercio (AIC) di un medicinale a uso umano dev’essere accompagnata da prove precliniche e cliniche dello stesso.
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However, by way of derogation, a simplified procedure is applicable to, inter alia, orphan medicinal products (that is, products intended for the treatment of very rare and serious conditions).
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Tuttavia, a titolo derogatorio, una procedura semplificata si applica ai medicinali orfani (vale a dire destinati al trattamento di malattie molto rare e gravi):
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Thus, an applicant will not be required to provide the results of pre-clinical tests or clinical trials if he can demonstrate that the active substances of the medicinal product have been in well-established medicinal use within the EU for at least 10 years, with recognised efficacy and an acceptable level of safety.
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il richiedente non è tenuto a fornire i risultati delle prove precliniche e cliniche, qualora possa dimostrare che le sostanze attive del medicinale sono di impiego medico ben consolidato da almeno dieci anni nell’Unione e presentano un’efficacia riconosciuta nonché un accettabile livello di sicurezza.
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In that event, the test and trial results are to be replaced by appropriate scientific literature.
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In tal caso, i risultati di tali prove sono sostituiti da adeguata letteratura scientifica.
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Laboratoires CTRS (Cell Therapies Research & Services) (‘CTRS’) has developed the medicinal product Orphacol, which contains cholic acid as an active substance.
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La società Laboratoires CTRS (Cell Therapies Research & Services) ha elaborato il medicinale Orphacol, la cui sostanza attiva è l’acido colico.
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That orphan medicinal product is used to treat rare but very serious liver disorders.
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Tale medicinale orfano è destinato a trattare patologie epatiche rare ma molto gravi.
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Those disorders, if not properly treated within the first weeks or months of life, can lead to death.
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Tali malattie, in assenza di trattamento adeguato nelle prime settimane o nei primi mesi di vita, possono portare alla morte.
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On 30 October 2009, CTRS applied to the European Medicines Agency (‘EMA’) for an MA for that medicinal product.
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Il 30 ottobre 2009 la CTRS ha presentato richiesta di AIC per tale medicinale presso l’agenzia europea per i medicinali (EMA).
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The Committee for Medicinal Products for Human Use (‘CMPHU’), a part of the EMA, issued a positive opinion, followed by a revised opinion, which was also positive, in December 2010 and April 2011 respectively, recommending that an MA be granted.
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Il comitato dei medicinali per uso umano (CMUU), facente parte dell’EMA, ha emanato parere favorevole ed un parere rivisto, parimenti favorevole, che raccomandava la concessione di un’AIC, rispettivamente nel dicembre 2010 e nell’aprile 2011.
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However, in July 2011 the Commission submitted to the Standing Committee on Medicinal Products for Human Use (‘the Standing Committee’) a draft decision refusing to grant CTRS an MA for Orphacol.
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Ciononostante, la Commissione ha sottoposto al comitato permanente per i medicinali per uso umano («comitato permanente»), nel luglio del 2011, un progetto di decisione recante diniego di rilascio di un’AIC alla CTRS per l’Orphacol.
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In October 2011, the Standing Committee issued a negative opinion on the Commission’s draft decision refusing to grant an MA.
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Nell’ottobre del 2011, il comitato permanente ha emanato un parere sfavorevole sul progetto di decisione della Commissione recante diniego dell’AIC.
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In the same month, the Commission submitted the draft decision refusing to grant an MA to the Appeal Committee.
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Nello stesso mese, la Commissione ha sottoposto il progetto di decisione che negava il rilascio dell’AIC al comitato d’appello.
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In November 2011, the Appeal Committee also issued a negative opinion on the Commission’s draft decision refusing to grant an MA.
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Nel novembre del 2011, il comitato d’appello ha parimenti emanato un parere sfavorevole.
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On 12 January 2012, CTRS brought an action before the General Court seeking a declaration that the Commission had failed to act in unlawfully failing to adopt a final decision in relation to its application for an MA for the medicinal product Orphacol and, in the alternative, applying for the annulment of the decision, allegedly contained in a letter of 5 December 2011 from the Commission, refusing to grant that MA.
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In data 12 gennaio 2012, la CTRS ha chiesto al Tribunale di dichiarare la carenza della Commissione in quanto l’Istituzione si sarebbe illegittimamente astenuta dall’adozione di una decisione definitiva in merito alla richiesta di AIC per il medicinale Orphacol e, in subordine, di annullare la decisione di diniego di rilascio dell’AIC medesima, asseritamente contenuta in una lettera della Commissione del 5 dicembre 2011.
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On 4 July 2012, the General Court of the European Union declared that the application for a declaration of failure to act was inadmissible and that, because the Commission had replaced the decision refusing the MA allegedly contained in the letter of 5 December 2011 with a decision of 25 May 2012 refusing to grant the MA, there was no need to adjudicate on the application for annulment of the former decision.
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In data 4 luglio 2012, il Tribunale dell’Unione europea ha dichiarato che la domanda per carenza era irricevibile e che, considerato che la Commissione, con decisione di diniego di rilascio dell’AIC del 25 maggio 2012, aveva proceduto alla sostituzione della decisione di diniego di rilascio dell’AIC, asseritamente contenuta nella menzionata lettera del 5 dicembre 2011, non occorreva più statuire sulla domanda di annullamento di quest’ultima decisione.
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On 10 July 2012, CTRS brought another action before the General Court seeking annulment of the decision of 25 May 2012.
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Il 10 luglio 2012, la CTRS ha adito nuovamente il Tribunale per l’annullamento della decisione del 25 maggio 2012.
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The Court decided to give the case priority treatment.
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Il Tribunale ha trattato la presente causa in via prioritaria.
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By its judgment delivered today, the General Court annuls the Commission’s decision of 25 May 2012 refusing to grant the MA for the medicinal product Orphacol.
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Con la sentenza odierna, il Tribunale annulla la decisione della Commissione del 25 maggio 2012, recante diniego di rilascio dell’AIC per il medicinale Orphacol.
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First of all, the Court notes that cholic acid has been used to treat patients in France between 1993 and October 2007 in the form of hospital preparations provided on medical prescription, prepared individually in accordance with the prescriptions of a pharmacopoeia and in compliance with the rules of good practice laid down in French legislation.
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Il Tribunale rileva, anzitutto, che l’acido colico è stato utilizzato per trattare pazienti in Francia, nel periodo compreso fra il 1993 e l’ottobre del 2007, quale preparato ospedaliero rilasciato su prescrizione medica, preparato individualmente secondo le indicazioni della farmacopea e conformemente alle corrette prassi della normativa francese.
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They are thus prescribed in a hospital or pharmaceutical establishment, under strict medical supervision.
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Tali preparati ospedalieri vengono prescritti sotto stretto controllo medico nell’ambito di un nosocomio o istituto farmaceutico.
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Since that date, cholic acid capsules have been authorised for use in France under the brand name Orphacol.
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Successivamente a tale data, capsule di acido colico sono state utilizzate in Francia, con il nome di Orphacol.
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Next, the Court finds that those hospital preparations were intended to fulfil ‘special needs’ as defined in EU law, that is, they were supplied in response to individual situations which were justified by medical considerations, and that they were necessary to meet patients’ needs.
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Il Tribunale afferma che tali preparati ospedalieri sono volti a rispondere a «esigenze speciali» ai sensi del diritto dell’Unione, vale a dire a situazioni individuali giustificate da considerazioni mediche e sono necessari per rispondere ai bisogni del paziente.
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Therefore, CTRS was not obliged, when applying for the MA, to provide the results of pre-clinical tests or clinical trials required by EU law
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La CTRS non era quindi tenuta, all’atto della richiesta di AIC, a fornire i risultati delle prove precliniche e cliniche richieste dal diritto dell’Unione.
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Accordingly, the General Court rejects the Commission’s arguments alleging that there was no well-established medicinal use, and finds that well-established medicinal use of cholic acid has been demonstrated by CTRS.
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Il Tribunale respinge pertanto gli argomenti della Commissione relativi all’assenza di un impiego medico ben consolidato e dichiara che l’utilizzazione dell’acido colico, in quanto impiego medico ben consolidato, risulta dimostrato dalla CTRS.
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In addition, the General Court finds that CTRS has shown that it was unable to provide comprehensive particulars on the efficacy and safety of the medicinal product concerned under normal conditions of use because of certain exceptional circumstances6 within the meaning of EU law.
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Inoltre, il Tribunale ritiene che la CTRS abbia dimostrato di non essere in grado di fornire informazioni complete sull’efficacia e l’innocuità del medicinale in condizioni normali di uso, in considerazione di determinate circostanze eccezionali6 ai sensi del diritto dell’Unione.
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The Court notes that an MA applicant must justify in the non-clinical and clinical summaries the reasons why it is not possible to provide comprehensive information on the efficacy and safety of the orphan medicinal product concerned and must justify the risk/benefit balance for that product.
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Il Tribunale ricorda che il richiedente dell’AIC deve fornire nei riassunti non clinici e clinici le ragioni per le quali non è possibile fornire informazioni complete quanto all’efficacia e alla sicurezza del medicinale e che deve fornire una giustificazione del profilo rischio/beneficio per il medicinale orfano di cui trattasi, cosa che la CTRS ha fatto nella specie.
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CTRS has done so in the present case, providing a list of bibliographical references to studies on cholic acid and demonstrating that it was unable to provide comprehensive data for objective, verifiable reasons: namely, the rareness of the disorders in question and ethical considerations.
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Essa ha effettivamente fornito una lista di riferimenti di letteratura scientifica relativa a studi attinenti all’acido colico e ha dimostrato di non essere in grado di fornire dati completi, da un lato, per ragioni obiettive e verificabili attinenti alla rarità della malattia e, dall’altro, per considerazioni deontologiche.
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As regards the first reason, at the time when the MA application was submitted (30 October 2009), only 90 patients had been diagnosed with the disorders, 19 of whom were treated in France.
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Quanto alla prima ragione, risulta che, alla data di deposito della richiesta (30 ottobre 2009), solamente 90 pazienti risultavano affetti dalla malattia; fra questi, 19 erano trattati in Francia.
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As regards the second reason, the CMPHU concluded (and its assessment was not challenged by the Commission) that, because participation in a clinical trial would expose patients to the risk of serious liver damage, or even death, it would be contrary to the principles of medical ethics to carry out a controlled study of the efficacy of cholic acid.
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Quando alla seconda ragione, il CMUU ha ritenuto, senza che tale valutazione venisse rimessa in discussione dalla Commissione, che, considerato che la partecipazione ad una prova clinica esporrebbe i pazienti al rischio di lesioni epatiche gravi, se non al rischio di decesso, sarebbe contrario ai principi di deontologia medica effettuare uno studio controllato sull’efficacia dell’acido colico.
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Accordingly, the Commission was wrong to conclude in its decision that the data submitted by CTRS should have been comprehensive, and that it could not invoke the existence of exceptional circumstances in its application made on the basis of well-established medicinal use.
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Erroneamente quindi la Commissione ha ritenuto, nella propria decisione, che i dati presentati dalla CTRS dovessero essere completi e che detta società non potesse invocare l’esistenza di circostanze eccezionali nell’ambito della propria richiesta fondata su un impiego medico ben consolidato.
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Consequently, the General Court annuls the Commission’s decision.
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Conseguentemente, il Tribunale annulla la decisione della Commissione.
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NOTE:
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IMPORTANTE:
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An appeal, limited to points of law only, may be brought before the Court of Justice against the decision of the General Court within two months of notification of the decision.
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Contro la decisione del Tribunale, entro due mesi a decorrere dalla data della sua notifica, può essere proposta un'impugnazione, limitata alle questioni di diritto, dinanzi alla Corte.
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NOTE:
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IMPORTANTE:
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An action for annulment seeks the annulment of acts of the institutions of the European Union that are contrary to European Union law.
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Il ricorso di annullamento mira a far annullare atti delle istituzioni dell’Unione contrari al diritto dell’Unione.
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The Member States, the European institutions and individuals may, under certain conditions, bring an action for annulment before the Court of Justice or the General Court.
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A determinate condizioni, gli Stati membri, le istituzioni europee e i privati possono investire la Corte di giustizia o il Tribunale di un ricorso di annullamento.
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If the action is well founded, the act is annulled.
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Se il ricorso è fondato, l'atto viene annullato.
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The institution concerned must fill any legal vacuum created by the annulment of the act.
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L'istituzione interessata deve rimediare all’eventuale lacuna giuridica creata dall’annullamento dell’atto.
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LISTEN WITH READSPEAKER
The General Court annuls the Commission’s decision refusing to grant a
marketing authorisation for Orphacol
A marketing authorisation may be issued in respect of that orphan
medicinal product on the basis of well-established medicinal use dating back
more than 10 years
EU law states that any marketing authorisation (?MA’) application
regarding a medicinal product for human use must be accompanied by the results
of the pre-clinical tests and clinical trials of the medicinal product
concerned.
However, by way of derogation, a simplified procedure is applicable to,
inter alia, orphan medicinal products (that is, products intended for the
treatment of very rare and serious conditions).
Thus, an applicant will not be required to provide the results of
pre-clinical tests or clinical trials if he can demonstrate that the active
substances of the medicinal product have been in well-established medicinal use
within the EU for at least 10 years, with recognised efficacy and an acceptable
level of safety.
In that event, the test and trial results are to be replaced by
appropriate scientific literature.
Laboratoires CTRS (Cell Therapies Research & Services) (‘CTRS’) has
developed the medicinal product Orphacol, which contains cholic acid as an
active substance.
That orphan medicinal product is used to treat rare but very serious
liver disorders.
Those disorders, if not properly treated within the first weeks or
months of life, can lead to death.
On 30 October 2009, CTRS applied to the European Medicines Agency
(‘EMA’) for an MA for that medicinal product.
The Committee for Medicinal Products for Human Use (‘CMPHU’), a part of
the EMA, issued a positive opinion, followed by a revised opinion, which was
also positive, in December 2010 and April 2011 respectively, recommending that
an MA be granted.
However, in July 2011 the Commission submitted to the Standing
Committee on Medicinal Products for Human Use (‘the Standing Committee’) a draft
decision refusing to grant CTRS an MA for Orphacol.
In October 2011, the Standing Committee issued a negative opinion on
the Commission’s draft decision refusing to grant an MA.
In the same month, the Commission submitted the draft decision refusing to grant
an MA to the Appeal Committee.
In November 2011, the Appeal Committee also issued a negative opinion
on the Commission’s draft decision refusing to grant an MA.
On 12 January 2012, CTRS brought an action before the General Court
seeking a declaration that the Commission had failed to act in unlawfully
failing to adopt a final decision in relation to its application for an MA for
the medicinal product Orphacol and, in the alternative, applying for the
annulment of the decision, allegedly contained in a letter of 5 December 2011
from the Commission, refusing to grant that MA.
On 4 July 2012, the General Court of the European Union declared that
the application for a declaration of failure to act was inadmissible and that,
because the Commission had replaced the decision refusing the MA allegedly
contained in the letter of 5 December 2011 with a decision of 25 May 2012
refusing to grant the MA, there was no need to adjudicate on the application for
annulment of the former decision.
On 10 July 2012, CTRS brought another action before the General Court
seeking annulment of the decision of 25 May 2012.
The Court decided to give the case priority treatment.
By its judgment delivered today, the General Court annuls the
Commission’s decision of 25 May 2012 refusing to grant the MA for the medicinal
product Orphacol.
First of all, the Court notes that cholic acid has been used to treat
patients in France between 1993 and October 2007 in the form of hospital
preparations provided on medical prescription, prepared individually in
accordance with the prescriptions of a pharmacopoeia and in compliance with the
rules of good practice laid down in French legislation.
They are thus prescribed in a hospital or pharmaceutical establishment,
under strict medical supervision.
Since that date, cholic acid capsules have been authorised for use in
France under the brand name Orphacol.
Next, the Court finds that those hospital preparations were intended to
fulfil ‘special needs’ as defined in EU law, that is, they were supplied in
response to individual situations which were justified by medical
considerations, and that they were necessary to meet patients’ needs.
Therefore, CTRS was not obliged, when applying for the MA, to provide
the results of pre-clinical tests or clinical trials required by EU law
Accordingly, the General Court rejects the Commission’s arguments
alleging that there was no well-established medicinal use, and finds that
well-established medicinal use of cholic acid has been demonstrated by CTRS.
In addition, the General Court finds that CTRS has shown that it was
unable to provide comprehensive particulars on the efficacy and safety of the
medicinal product concerned under normal conditions of use because of certain
exceptional circumstances6 within the meaning of EU law.
The Court notes that an MA applicant must justify in the non-clinical
and clinical summaries the reasons why it is not possible to provide
comprehensive information on the efficacy and safety of the orphan medicinal
product concerned and must justify the risk/benefit balance for that product.
CTRS has done so in the present case, providing a list of
bibliographical references to studies on cholic acid and demonstrating that it
was unable to provide comprehensive data for objective, verifiable reasons:
namely, the rareness of the disorders in question and ethical considerations.
As regards the first reason, at the time when the MA application was
submitted (30 October 2009), only 90 patients had been diagnosed with the
disorders, 19 of whom were treated in France.
As regards the second reason, the CMPHU concluded (and its assessment
was not challenged by the Commission) that, because participation in a clinical
trial would expose patients to the risk of serious liver damage, or even death,
it would be contrary to the principles of medical ethics to carry out a
controlled study of the efficacy of cholic acid.
Accordingly, the Commission was wrong to conclude in its decision that
the data submitted by CTRS should have been comprehensive, and that it could not
invoke the existence of exceptional circumstances in its application made on the
basis of well-established medicinal use.
Consequently, the General Court annuls the Commission’s decision.
NOTE:
An appeal, limited to points of law only, may be brought before the
Court of Justice against the decision of the General Court within two months of
notification of the decision.
NOTE:
An action for annulment seeks the annulment of acts of the institutions
of the European Union that are contrary to European Union law.
The Member States, the European institutions and individuals may, under
certain conditions, bring an action for annulment before the Court of Justice or
the General Court.
If the action is well founded, the act is annulled.
The institution concerned must fill any legal vacuum created by the
annulment of the act. |