qanastek/EMEA-V3 huggingface dataset:
How does Naglazyme work?
Kif jaħdem Naglazyme?
Naglazyme is an enzyme replacement therapy.
Naglazyme hu terapija enzimatika sostituttiva.
Enzyme replacement therapy provides patients with the enzyme they are lacking.
Terapija enzimatika sostituttiva tipprovdi pazjenti bl - enzima li m’ għandhomx.
The active substance in Naglazyme, galsulfase, is a copy of the human enzyme arylsulfatase B.
Is- sustanza attiva ta ’ Naglazyme, galsufase, hija kopja tal- enzima umana, arylsulfatase B.
Naglazyme helps to break down the GAGs and stop them building up in the cells.
Naglazyme jgħin biex ikisser il- GAGs u jwaqqafhom milli jakkumulaw fiċ- ċelluli.
This can improve the symptoms of MPS VI, including how far people with the disease can walk.
Dan jista ’ jtejjeb is- sintomu ta ’ MPS VI, inkluż id- distanza sa fejn jistgħu jimxu n- nies li jbatu minn din il- marda.
(44-20) 74 18 84 00 Fax (44-20) 74 18 86 13 E-mail: mail@emea. europa. eu http: / /www. emea. europa. eu
(44- 20) 74 18 84 00 Fax (44- 20) 74 18 86 13 E- mail: mail@ emea. eu. int http: // www. emea. eu. int
Reproduction is authorised provided the source is acknowledged.
Reproduction is authorised provided the source is acknowledged Galsulfase tiġi prodotta b’ metodu magħruf bħala ‘ teknoloġija tad- DNA rikombinanti ’: tiġi prodotta minn ċellula li tkun ingħatat ġene (DNA), li jagħmel il- produzzjoni tal- enzima possibbli.
How has Naglazyme been studied?
Kif ġie studjat Naglazyme?
Naglazyme has been compared with placebo (a dummy treatment) in one main study involving 39 patients with MPS VI aged between five and 29 years.
Naglazyme tqabbel ma ’ plaċebo (trattament finta) fi studju ewlieni li kien jinvolvi 39 pazjent b’ MPS VI ta ’ bejn ħames snin u 29 sena.
The main measure of effectiveness was how far the patients could walk after 24 weeks of treatment.
Il- kejl ewlieni tal- effikaċja kien id- distanza sa fejn setgħu jimxu l- pazjenti wara 24 ġimgħa ta ’ trattament.
What benefit has Naglazyme shown during the studies?
X’ benefiċċji wera Naglazyme matul l- istudji mwettqa?
Naglazyme was more effective than placebo.
Naglazyme kien iktar effikaċi mill- plaċebo.
After 24 weeks, the average distance walked over 12 minutes increased by 109 metres in the patients treated with Naglazyme and by 18 metres in those receiving placebo.
Wara 24 ġimgħa, id- distanza medja li setgħu jimxu l - pazjenti fi 12- il minuta żdiedet għal 109 metri f’ dawk ittrattati b’ Naglazyme u 18- il metru f’ dawk ittrattati bi plaċebo.
What is the risk associated with Naglazyme?
X’ inhu r- riskju assoċjat ma ’ Naglazyme?
In studies, the most common side effects with Naglazyme (seen in more than 1 patient in 10) were ear pain, dyspnoea (difficulty breathing), abdominal (tummy) pain, general pain and reactions to the infusion (such as fever, chills, rash and hives).
Fl- istudji, l- effetti sekondarji l- iktar komuni b’ Naglazyme (li dehru f’ pazjent wieħed minn kull 10) kienu wġigħ fil- widnejn, dispnea (diffikultà fin- nifs), uġigħ addominali (ta ’ żaqq), uġigħ ġenerali u reazzjonijiet għall- infużjoni (bħal deni, tertir tal- bard, raxx u ħorriqija).
For the full list of all side effects reported with Naglazyme, see the Package Leaflet.
Għal- lista sħiħa tal- effetti sekondarji kollha rrapportati ma ’ Naglazyme, ara l- Fuljett ta ’ Tagħrif.
Naglazyme should not be used in people who may be hypersensitive (allergic) to galsulfase or any of the other ingredients.
Naglazyme m’ għandux jintuża minn persuni li jistgħu jkunu ipersensittivi (allerġiċi) għal galsulfase jew għal xi ingredjenti oħra tiegħu.
Why has Naglazyme been approved?
Għaliex ġie approvat Naglazyme?
The Committee for Medicinal Products for Human Use (CHMP) decided that for patients with MPS VI, treatment with Naglazyme provides benefits such as improved mobility when compared with patients given placebo.
Il- Kumitat għall- Prodotti Mediċinali għall- Użu mill- Bniedem (CHMP) iddeċieda li, għal pazjenti b’ MPS VI, it- trattament b’ Naglazyme jipprovdi benefiċċji bħal titjib tal- mobilità meta mqabbla mal - pazjenti li ngħataw plaċebo.
Naglazyme only slightly improves the symptoms of MPS VI, but because this disease is severe and there are no other treatments for most patients with the disease, the CHMP decided that Naglazyme’ s benefits are greater than its risks for long-term enzyme replacement therapy in patients with a confirmed diagnosis of MPS VI.
Nagalzyme jtejjeb is- sintomi ta ’ MPS VI sa punt limitat, iżda minħabba li din il- marda hija waħda gravi u l- fatt li ma hemm l- ebda trattament ieħor għal bosta mill- pazjenti li jbatu minn din il- marda, is- CHMP iddeċieda li l- benefiċċji ta ’ Naglazyme huma ikbar mir- riskji għal terapija enzimatika sostituttiva fuq tul ta ’ żmien twil f’ pazjenti b’ dijanjożi kkonfermata ta ’ MPS VI.
The Committee recommended that Naglazyme be given marketing authorisation.
Il- Kumitat irrakkomanda li Naglazyme jingħata awtorizzazzjoni għat- tqegħid fis- suq.
Naglazyme has been authorised under ‘ Exceptional Circumstances’.
Naglazyme ġie awtorizzat taħt ‘ Ċirkostanzi Eċċezzjonali ’.
This means that because the disease is rare, it has not been possible to obtain complete information about Naglazyme.
Dan ifisser li minħabba li l- marda hija rari, ma kienx possibbli li jinġabar it- tagħrif kollu dwar Naglazyme.
Every year, the European Medicines Agency (EMEA) will review any new information that may become available and this summary will be updated as necessary.
Kull sena, l- Aġenzija Ewropea għall - Mediċini (EMEA) tirrevedi kwalunkwe tagħrif ġdid li jista 'jinkiseb u dan is- sommarju se jiġi aġġornat kif meħtieġ.
What information is still awaited for Naglazyme?
X’ tagħrif għadu mistenni dwar Naglazyme?
The company that makes Naglazyme will carry out studies to look at the long-term safety and effectiveness of Naglazyme in pregnant and breast-feeding women and in children under the age of five years, to see if they develop antibodies (proteins that are produced in the body in response to Naglazyme that could affect the response to treatment) and to look at the medicine’ s side effects.
Il- kumpanija li tipproduċi Naglazyme se twettaq studji biex teżamina s- sigurtà u l- effikaċja ta ’ Naglazyme fuq tul ta ’ żmien twil f’ nisa tqal u li qegħdin ireddgħu u fi tfal taħt il- ħames snin, sabiex tara jekk dawn jiżviluppawx antikorpi (proteini prodotti fil- ġisem bħala rispons għal Naglazyme li jistgħu jaffettwaw ir- rispons għat- trattament) u sabiex jiġu osservati l- effetti sekondarji tal- mediċina.
It will also carry out studies to determine the best dose to give to patients on a regular long-term basis.
Se twettaq ukoll studji biex tiddetermina l- aqwa doża li għandha tingħata lil pazjenti regolarment għal tul ta ’ żmien twil.
What are the measures to minimise the risk associated with Naglazyme?
X’ inhuma l- miżuri għat- tnaqqis tar- riskju assoċjat ma ’ Naglazyme?
The company that makes Naglazyme has put a plan in place to monitor the long-term safety and effectiveness of the medicine, collecting all the necessary data from patients treated with it.
Il- kumpanija li tipproduċi Naglazyme stabbiliet pjan għall- monitoraġġ tas- sigurtà u l- effikaċja tal - mediċina fuq tul ta ’ żmien twil, bil- ġbir tad- dejta kollha meħtieġa minn pazjenti ttrattati b’ din il - mediċina.
Other information about Naglazyme:
Tagħrif ieħor dwar Naglazyme:
The European Commission granted a marketing authorisation valid throughout the European Union for Naglazyme to BioMarin Europe Limited on 24 January 2006.
Il- Kummissjoni Ewropea tat awtorizzazzjoni għat- tqegħid fis- suq valida fl- Unjoni Ewropea kollha għal Naglazyme lil BioMarin Europe Limited fl- 24 ta 'Jannar 2006.
The summary of opinion of the Committee for Orphan Medicinal Products is available here.
Is- sommarju tal- opinjoni tal- Kumitat għall- Prodotti Mediċinali Orfni huwa disponibbli hawn.
The full EPAR for Naglazyme can be found here.
L- EPAR sħiħ għal Naglazyme jista ’ jiġi kkonsultat hawn.
EU Number
Numru ta ’ l- EU EU/ 1/ 05/ 324/ 001
Invented name
Isem inventat Naglazyme
Strength
Qawwa 1 mg/ ml
Pharmaceutical Form
Għamla farmaċewtika Konċentrat għal soluzzjoni għall - infużjoni
Route of Administration
Mnejn jingħata Użu għal ġol - vini
Packaging
Il- pakkett Kunjett (ħġieġ)
Content
Content 5 ml (1mg/ ml)
Package size
Daqs tal- pakkett 1 kunjett
Concentrate for solution for infusion Concentrate for solution for infusion
Konċentrat għal soluzzjoni għall - infużjoni
Intravenous use Intravenous use
Użu għal ġol - vini
vial (glass) vial (glass)
Kunjett (ħġieġ)
1 vial 6 vial
6 kunjett
Naglazyme 1 mg/ ml concentrate for solution for infusion.
Naglazyme 1 mg/ ml konċentrat għal soluzzjoni għall- infużjoni.
Each ml of solution contains 1 mg galsulfase.
Kull ml ta ’ soluzzjoni fih 1 mg galsulfase.
One vial of 5 ml contains 5 mg galsulfase.
Kunjett wieħed ta ’ 5 ml fih 5 mg galsulfase.
Galsulfase is a recombinant form of human N-acetylgalactosamine 4-sulfatase and is produced by recombinant DNA technology using mammalian Chinese Hamster Ovary (CHO) cell culture.
Galsulfase huwa forma rikombinanti ta ’ N- acetylgalactosamine 4- sulfatase uman u huwa magħmul b’ teknoloġija ta ’ DNA rikombinanti permezz ta ’ koltura ta ’ ċelluli mammiferi tal- Ovarju ta ’ Ħamster Ċiniż (CHO).
A clear to slightly opalescent, and colourless to pale yellow solution.
Soluzzjoni ċara għal kemmxejn tkanġi, u bla kulur għal safra ċara.
Naglazyme is indicated for long-term enzyme replacement therapy in patients with a confirmed diagnosis of Mucopolysaccharidosis VI (MPS VI; N-acetylgalactosamine 4-sulfatase deficiency; Maroteaux-Lamy syndrome) (see section 5.1).
Naglazyme huwa ndikat għal terapija ta ’ sostituzzjoni ta ’ enzima fit- tul f’ pazjenti b’ dijanjosi kkonfermata ta ’ Mukoplisakkaridosi VI (MPS VI; defiċjenza ta ’ N- acetylgalactosamine 4- sulfatase; sindromu Maroteaux- Lamy) (ara sezzjoni 5. 1).
As for all lysosomal genetic disorders, it is of primary importance, especially in severe forms, to initiate treatment as early as possible, before appearance of non-reversible clinical manifestations of the disease.
Bħal f’ kull mard liżosomali ġenetiku ieħor, huwa importanti ħafna, speċjalment fil- forom severi, li tinbeda l- kura malajr kemm jista ’ jkun, qabel jibdew jidhru manifestazzjonijiet kliniċi mhux riversibli tal- marda.
A key issue is to treat young patients aged < 5 years suffering from a severe form of the disease, even though patients < 5 years were not included in the pivotal phase 3 study.
Punt kruċjali huwa li tikkura pazjenti żgħar b’ età ta ’ < 5 snin li qed isofru minn forma severa tal - marda, anke jekk pazjenti b’ età ta ’ < 5 snin ma kienux inklużi fl- istudju pivitali ta ’ fażi 3.
Naglazyme treatment should be supervised by a physician experienced in the management of patients with MPS VI or other inherited metabolic diseases.
Il- Kura b’ Naglazyme għandha tiġi sorveljata minn tabib b’ esperjenza fl- immaniġġar ta ’ pazjenti b’ MPS VI jew mard metaboliku ieħor li jintiret.
Administration of Naglazyme should be carried out in an appropriate clinical setting where resuscitation equipment to manage medical emergencies would be readily available.
L- għoti ta ’ Naglazyme għandu jingħata f’ ambjent kliniku xieraq fejn ikun hemm disponibli apparat ta ’ risuxxitazzjoni għall- immaniġġar ta ’ emerġenza medika.
The recommended dosage regimen for galsulfase is 1 mg/ kg body weight administered once every week as an intravenous infusion over 4 hours.
Il- kors ta ’ dożaġġ ta ’ galsulfase rakkomandat huwa ta ’ 1 mg/ kg piż tal- ġisem mogħti darba fil- ġimgħa bħala infużjoni fil- vina fuq medda ta ’ 4 sigħat.
The initial infusion rate is adjusted so that approximately 2.5% of the total solution is infused during the first hour, with infusion of the remaining volume (approximately 97.5%) over the next 3 hours.
Ir- rata tal- bidu tal- infużjoni tiġi aġġustata biex madwar 2. 5% tas- soluzzjoni totali tkun infuża fl- ewwel siegħa, u l- bqija tal- volum (madwar 97. 5%) jiġi infuż fuq it- 3 sigħat ta ’ wara.
For information on pre-treatment see section 4.4 and for further instructions see section 6.6.
Għall- informazzjoni dwar x’ għandu jsir qabel il- kura ara sezzjoni 4. 4 u għall - aktar struzzjonijiet ara sezzjoni 6. 6.
Caution must be exercised in the management and treatment of patients with compromised airways by limitation or careful monitoring of antihistamine and other sedative medication use.
Għandha tingħata attenzjoni speċjali fl- immaniġġjar u l- kura ta ’ pazjenti bi problemi fil- passaġġ respiratorju, permezz ta ’ limitazzjoni jew sorveljanza mill- qrib ta ’ l- użu ta ’ antistamini jew ta ’ mediċini sedattivi oħrajn.
Institution of positive– airway pressure during sleep as well as potential tracheostomy in clinically appropriate situations should also be considered.
Għandhom jiġu kkunsidrati wkoll, applikazzjoni ta ’ pressjoni pożittiva fil - passaġġi respiratorji waqt l- irqad, kif ukoll il- possibiltà ta ’ trakeotomija f’ sitwazzjonijiet kliniċi xierqa.
Consider delaying Naglazyme infusions in patients who present with an acute febrile or respiratory illness.
Ikkunsidra li titardja l- infużjonijiet b’ Naglazyme, f’ pazjenti b’ mard respiratorju jew deni akut.
The safety and efficacy of Naglazyme in children below the age of 5 years and in patients older than 65 years have not been established.
Is- sigurtà u l- effikaċja ta ’ Naglazyme fit- tfal taħt l- età ta ’ 5 snin u f’ pazjenti ta ’ età akbar minn 65 sena ma ġewx stabbiliti.
The safety and efficacy of Naglazyme in patients with renal or hepatic insufficiency have not been evaluated (see section 5.2).
Is- sigurtà u l- effikaċja ta ’ Naglazyme f’ pazjenti b’ insuffiċjenza renali jew epatika ma ġewx evalwati (ara sezzjoni 5. 2).
Patients treated with Naglazyme have developed infusion-associated reactions (IARs), defined as any drug-related adverse event occurring during the infusion or until the end of the infusion day (see section 4.8).
Pazjenti kkurati b’ Naglazyme żviluppaw reazzjonijiet assoċjati ma ’ l- infużjoni (IARs), definiti bħala kull reazzjoni avversa relatata mal- mediċina li sseħħ waqt l- infużjoni jew sa l- aħħar tal- ġurnata li fiha tkun ġiet mogħtija l- infużjoni (ara sezzjoni 4. 8).
Based on data obtained during Naglazyme clinical trials, the majority of patients are expected to develop IgG antibodies to galsulfase within 4-8 weeks of treatment initiation.
Ibbażat fuq tagħrif miġbur waqt provi kliniċi b’ Naglazyme, l- maġġoranza tal- pazjenti huma mistennija li jiżviluppaw antikorpi IgG għal galsulfase minn 4- 8 ġimgħat mill- bidu tal- kura.
In the Naglazyme clinical trials, IARs were usually manageable by interrupting or slowing the rate of infusion and by (pre-) treating the patient with antihistamines and/ or antipyretics (paracetamol), thus enabling the patient to continue treatment.
Fil- provi kliniċi b’ Naglazyme, IARs ġeneralment kienu maniġġabbli bl- interruzzjoni jew tnaqqis fir- rata ta ’ l- infużjoni u b’ kura minn qabel tal- pazjent b’ antistamini u/ jew sustanzi kontra d- deni (paracetamol), u b’ hekk ikun possibbli għall- pazjent li jkompli l- kura.
As there is little experience on resumption of treatment following prolonged interruption, caution is to be used due to the theoretical increased risk of hypersensitivity reaction.
Minħabba li l- esperjenza dwar bidu mill- ġdid tal- kura wara nterruzzjoni fit- tul hija limitata, għandha tintuża kawtela minħabba żieda teoretika fir- riskju ta ’ reazzjonijiet ta ’ sensittività eċċessiva.
With administration of Naglazyme it is recommended that patients be administered pre-treatment medications (antihistamines with or without antipyretics) approximately 30-60 minutes prior to the start of the infusion, to minimize the potential occurrence of IARs.
Biex jitnaqqas il- potenzjal li jseħħu IARs, huwa rakkomandat li meta jkun se jingħata Naglazyme pazjenti jingħataw mediċini minn qabel (antistamini flimkien ma ’, jew mingħajr sustanzi kontra d - deni), madwar 30- 60 minuta qabel il- bidu ta ’ l- infużjoni.
In case of a mild or moderate IAR, treatment with antihistamines and paracetamol should be considered and/ or a reduction in the infusion rate to half the rate at which the reaction occurred.
F’ każ ta ’ IAR ħafifa jew moderata, kura b’ antistamini u paracetamol għandha tiġi kkunsidrata u/ jew titnaqqas ir- rata tal- infużjoni għal nofs ir- rata li fiha tkun seħħet ir- reazzjoni.
In case of a single severe IAR, the infusion should be stopped until the symptoms are resolved and treatment with antihistamines and paracetamol should be considered.
F’ każ ta ’ IAR severa waħda, l- infużjoni għandha titwaqqaf sakemm is- sintomi jgħaddu u kura b’ antistamini u paracetamol għandha tiġi kkunsidrata.
The infusion can be restarted with a reduction of the infusion rate to 50% – 25% of the rate at which the reaction occurred.
L- infużjoni tista ’ terġa tinbeda bi tnaqqis fir - rata tal- infużjoni għal 50% - 25% tar- rata li fiha tkun seħħet ir- reazzjoni.
In case of a recurrent moderate IAR or re-challenge after a single severe IAR, pre-treatment should be considered (antihistamines and paracetamol and/ or corticosteroids) and a reduction of the infusion rate to 50% – 25% of the rate at which the previous reaction occurred.
F’ każ ta ’ IAR moderata rikorrenti jew f’ każ li tkun reġgħet nbdiet il- kura wara IAR severa waħda, kura minn qabel għandha tkun ikkunsidrata (antistamini u paracetamol u/ jew kortikosterojdi) u tnaqqis fir- rata tal- infużjoni għal 50% - 25% tar- rata li fiha tkun seħħet ir- reazzjoni ta ’ qabel.
As with any intravenous protein product, severe allergic-type hypersensitivity reactions are possible.
Bħal f’ kull prodott mill- proteina li jingħata fil- vina, reazzjonijiet allerġiċi severi ta ’ sensittività eċċessiva huma possibbli.
If these reactions occur, immediate discontinuation of Naglazyme is recommended and appropriate medical treatment should be initiated.
Jekk iseħħu dawn ir- reazzjonijiet, huwa rakkomandat li jitwaqqaf Naglazyme minnufih u li tinbeda kura medika xierqa.
The current medical standards for emergency treatment are to be observed.
Għandhom ikunu osservati l- istàndards mediċi kurrenti għall- kura ta ’ emerġenza.
Animal studies do not indicate direct or indirect harmful effects with respect to pregnancy or embryo-foetal development (see section 5.3).
Studji fuq il- bhejjem ma jurux effetti ħżiena diretti jew indiretti fuq it- tqala jew l- iżvilupp ta ’ l- embriju/ fetu (ara sezzjoni 5. 3).
Naglazyme should not be used during pregnancy unless clearly necessary.
Naglazyme m’ għandux jingħata waqt it- tqala ħlief meta hemm bżonn speċifiku.
It is not known whether galsulfase is excreted in milk, therefore breast-feeding should be stopped during Naglazyme treatment.
Mhux magħruf jekk galsulfase joħroġx fil- ħalib, għalhekk treddigħ għandu jitwaqqf waqt kura b’ Naglazyme.
All patients in the Phase 3 clinical study experienced Adverse Drug Reactions (ADRs), regardless of whether they were receiving Naglazyme or placebo.
Il- pazjenti kollha fl- istudju kliniku ta ’ Fażi 3 esperjenzaw Effetti Avversi għall- Mediċina (ADRs), kemm jekk kienu qed jingħataw Naglazyme kif ukoll bil- plaċebo.
ADRs reported during the Phase 3 study, in 39 patients treated up to 6 months, are listed in the table below by Body System.
L- ADRs irrappurtati waqt l- istudju ta ’ Fażi 3, f’ 39 pazjent ikkurati sa 6 xhur, huma mniżżla fit- tabella ta ’ hawn taħt skond is- sistema tal- ġisem.
The ADRs are listed following the recommended frequency convention.
L- ADRs huma mniżżla skond il- konvenzjoni ta ’ frekwenza rakkomandata.
Very common adverse events are those with a frequency of more than 1 in 10.
Effetti avversi komuni ħafna huma dawk bi frekwenza ta ’ aktar minn 1 minn kull 10.
Common events have a frequency of more than 1 in 100.
Effetti komuni għandhom frekwenza ta ’ aktar minn 1 minn kull 100.
One case of sleep apnoea was experienced during this study.
Waqt l- istudju kien hemm esperjenza ta ’ każ wieħed ta ’ apneja waqt l- irqad.
Within each frequency grouping, undesirable effects are presented in order of decreasing seriousness.
F’ kull sezzjoni ta ’ frekwenza, l- effetti mhux mixtieqa huma mniżżlin skond is- serjetata ’ tagħhom.
Number and percentage of patients with selected adverse events in the placebo- controlled study
Numru u persentaġġ ta ’ pazjenti b’ reazzjonijiet avversi magħżula fl- istudju kkontrollat bil- plaċebo.
Preferred
Terminu
Naglazyme Placebo%
Naglazyme Plaċebo%
Term
Ppreferut
% (pts)
% (pts)
Infections & Infestations
Infezzjonijiet u infestazzjonijiet
Gastroenteritis
Gastro- enterite
Nervous system disorders Eye Disorders
Disturbi fis- sistema nervuża Disturbi fl- għajnejn
Areflexia Conjunctivitis Corneal opacity
Areflexja Konġuntivite
Common Common Common Very
Komuni Komuni
Ear pain Hypertension
Opaċità tal - korneja
common Common
Komuni
Respiratory, Thoracic, & Mediastinal
Disturbi fil- widnejn u fis- sistema labirintika
Very
Komuni ħafna
Disorders
Disturbi vaskulari Disturbi respiratorji, toraċiċi u medjastinali
Dyspnoea Apnoea Nasal
Komuni Komuni ħafna
21 (4) 5 (1)
5 (1)
congestion
Imnieħer miżdud
Common Very
Komuni
Gastrointestinal Disorders
Disturbi gastro- intestinali
Abdominal pain Umbilical
Uġigħ addominali
common
Komuni ħafna
hernia
Ernja ta ’ l - umbeliku
Skin & Subcutaneous Tissue Disorders
Disturbi fil- ġilda u fit- tessuti ta ’ taħt il - ġilda
Face oedema
Edima fil- wiċċ
General Disorders & Administration Site
Disturbi ġenerali u kondizzjonijiet ta 'mnejn jingħata
Very
Uġigħ
Conditions
Komuni ħafna
Chest pain Rigors Malaise
Uġigħ fis- sider
Infusion reactions, characterized by a recurring pattern of symptoms occurring during Naglazyme infusion, were observed in 30 (56%) of the 54 patients treated with Naglazyme across all clinical studies.
Reazzjonijiet tal- infużjoni, kkarratterizzati minn sekwenza rikorrenti ta ’ sintomi li jseħħu waqt l - infużjoni ta ’ Naglazyme, kienu osservati fi 30 (56%) minn 54 pazjent ikkurati b’ Naglazyme fl- istudji kliniċi kollha.
Reactions began as early as Week 6 and as late as Week 55 of study drug treatment, and occurred during multiple infusions though not always in consecutive weeks.
Ir- reazzjonijiet bdew l- aktar kmieni mis- 6 Ġimgħa sal- 55 Ġimgħa tal- kura bil- mediċina li kienet qed tiġi studjata, u seħħew waqt infużjonijiet multipli għalkemm mhux dejjem f’ ġimgħat konsekuttivi.
The most common symptoms of these infusion reactions included fever, chills/ rigors, rash, and urticaria, although hypotension, nausea, vomiting, dyspnoea, bronchospasm, retrosternal pain, abdominal pain, headache, malaise, respiratory stress, angioneurotic oedema and joint pain were also reported.
L- aktar sintomi komuni ta ’ reazzjonijiet għall- infużjoni kienu jinkludu deni, rogħda ta ’ bard/ tertir, raxx, u urtikarja, għalkemm kienu rrapportati wkoll pressjoni baxxa, tqalligħ, rimettar, qtugħ ta ’ nifs, bronkospażmu, uġigħ retrosternali, uġigħ addominali, uġigħ ta ’ ras, telqa, stress respiratorju, edima anġjonewrotika u uġigħ fil- ġogi.
52/ 54 patients (96%) treated with Naglazyme across studies were positive for antibodies to galsulfase.
Fl- istudji kollha 52 pazjent minn 54 (96%) ikkurati b’ Naglazyme kienu pożittivi għall- antikorpi għal galsulfase.
The clinical significance of antibodies to galsulfase is not known.
Is- sinifikattiv kliniku ta ’ antikorpi għal galsulfase mhux magħruf.
No case of overdose with Naglazyme has been reported.
L- ebda każ ta ’ doża eċċessiva b’ Naglazyme ma ġie rrapurtat.
Several patients have received their total dose of Naglazyme at approximately twice the recommended infusion rate without apparent adverse events.
Diversi pazjenti rċevew id- doża totali tagħhom ta ’ Naglazyme b’ rata ta ’ l- infużjoni ta ’ madwar id- doppju ta ’ dik rakkomandata mingħajr id - dehra ta ’ episodji avversi.
Alimentary tract and metabolism products - enzymes, ATC code:
Prodotti tal- metaboliżmu u tal- kanal alimentari - enzimi, Kodiċi ATC:
This medicinal product has been authorised under "Exceptional Circumstances".
Dan il- prodott mediċinali kien awtorizzata taħt ‘ Ċirkustanzi ta ’ Eċċezzjoni ’.
This means that due to the rarity of the disease it has not been possible to obtain complete information on this medicinal product.
Dan ifisser li minħabba li l- marda hija rari ma ’ kienx possibbli li tinkiseb informazzjoni kompleta dwar dan il- prodott mediċinali.
The European Medicines Agency (EMEA) will review any new information which may become available every year and this SPC will be updated as necessary.
L- Aġenzija Ewropeja dwar il- Mediċini (EMEA) ser tirrevedi kull tip ta ’ informazzjoni ġdida li toħroġ kull sena u ser taġġorna dan is- Sommarju tal- Karatteristiċi tal- Prodott, skond il- bżonn.
Mucopolysaccharide storage disorders are caused by the deficiency of specific lysosomal enzymes required for the catabolism of glycosaminoglycans (GAGs).
Disturbi fil- ħażna ta ’ mukopolisakkarajd huwa kkawżat minn defiċjenza ta ’ enzimi liżosomali speċifiċi li huma meħtieġa għall- kataboliżmu ta ’ glycosaminoglycans (GAGs).
MPS VI is a heterogeneous and multisystemic disorder characterized by the deficiency of N-acetylgalactoasamine 4-sulfatase, a lysosomal hydrolase which catalyses the hydrolysis of sulfate moiety of the glycosaminoglycan, dermatan sulfate.
MPS VI hija marda eteroġena li tidher f’ diversi sistemi tal- ġisem, ikkaratterizzata minn defiċjenza ta ’ N - acetylgalactoasamine 4- sulfatase, hydrolase liżosomali li tikkatalizza l- idroliżi tal- porzjon ta ’ sulfate ta ’ glycosaminoglycan, dermatan sulfate.
Reduced or absent N-acetyl galactosamine 4-sulfatase activity results in the accumulation of dermatan sulfate in many cell types and tissues.
Attività ta ’ N- acetyl galactosamine 4- sulfatase imnaqqsa jew assenti tirriżulta fl- akkumulazzjoni ta ’ dermatan sulfate f’ diversi tipi ta ’ ċelluli u tessuti.
The rationale for enzyme replacement therapy is to restore a level of enzymatic activity sufficient to hydrolyze the accumulated substrate and to prevent further accumulation.
Ir- raġuni tat- terapija ta ’ sostituzzjoni ta ’ enzimi hija biex jerġa jinkiseb livell ta ’ attività enzimatika għoli biżżejjed biex ikun idrolizzat is- substrat akkumulat u tipprevjeni aktar akkumulazzjoni.
Purified galsulfase, a recombinant form of human N-acetylgalactosamine 4-sulfatase, is a glycoprotein with a molecular weight of approximately 56 kD.
Galsulfase ppurifikat, forma rikombinanti ta ’ N- acetylgalactosamine 4- sulfatase uman, huwa glikoproteina b’ piż molekulari ta ’ madwar 56 kD.
Galsulfase is comprised of 495 amino acids after cleavage of the N-terminus.
Galsulfase jikkonsisti minn 495 aċidu amminiku wara l- qtuħ ta ’ N- terminus.
The molecule contains 6 N-linked oligosaccharide modification sites.
Il- molekola fiha 6 siti ta ’ modifikazzjoni ta ’ N- linked oligosaccharide.
After intravenous infusion, galsulfase is rapidly removed from the circulation and taken up by cells into lysosomes, most likely via mannose-6 phosphate receptors.
Wara infużjoni fil- vina, galsulfase jitneħħa malajr miċ- ċirkolazzjoni u jittieħed miċ- ċelluli għal ġewwa l- liżosomi, x’ aktarx permezz ta ’ riċetturi mannose- 6 phosphate.
The three clinical studies performed with Naglazyme focused on assessing the systemic manifestations of MPS VI such as endurance, joint mobility, joint pain and stiffness, upper airway obstruction, manual dexterity and visual acuity.
It- tliet studji kliniċi li saru b’ Naglazyme iffukaw fuq il- valutazzjoni tal- manifestazzjonijiet sistemiċi ta ’ MPS VI bħal reżistenza, mobilità tal- ġogi, uġigħ fil- ġogi u ebusija, ostruzzjoni tal- passaġġ respiratorju ta ’ fuq, ħeffa manwali u akutezza fil- vista.
The safety and efficacy of Naglazyme was assessed in a randomised, double blind, placebo controlled, phase 3 study of 39 MPS VI patients, ranging in age from 5 to 29 years.
Is- sigurtà u l- effikaċja ta ’ Naglazyme kienu vvalutati fi studju randomised, double blind, u kkontrollat bil- plaċebo, ta ’ fażi 3 ta ’ 39 pazjent bi MPS VI, b’ età minn 5 sa 29 sena.
The majority of the patients presented with short stature, impaired endurance, and musculoskeletal symptoms.
Il- maġġoranza tal- pazjenti kellhom statura qasira, reżistenza mnaqqsa u sintomi muskolu- skeletari.
Patients who could walk more than 5 meters (m) but less than 250 m in 6 minutes of a 12 Minute Walk test or less than 400 m at the 12 minute time point at baseline were enrolled in the study.
Pazjenti li setgħu jimxu aktar minn 5 metri (m) iżda anqas minn 250 m f’ 6 minuti ta ’ eżami ta ’ Mixja ta ’ 12- il Minuta jew anqas minn 400 m fi 12- il minuta fil- linja bażi ddaħħlu fl- istudju.
Patients received either 1 mg/ kg of galsulfase or placebo every week for a total of 24 weeks.
Il- pazjenti rċevew 1 mg/ kg ta ’ galsulfase jew plaċebo kull ġimgħa għal total ta ’ 24 ġimgħa.
The primary efficacy endpoint was the numbers of meters walked in 12 minutes at Week 24 compared to the number of meters walked at baseline.
Il- mira prinċipali ta ’ l- effikaċja kienet in- numru ta ’ metri li mxew fi 12- il minuta fl- 24 Ġimgħa mqabbla man - numru ta ’ metri li mxew fil- linja bażi.
The secondary efficacy endpoints were the rate of stairs climbed in three minutes and the urinary glycosaminoglycan excretion of treated patients compared to placebo at Week 24.
Il- miri sekondarji ta ’ l- effikaċja kienu n- numru ta ’ taraġ li telgħu fi tliet minuti u t- tneħħija ta ’ glycosaminoglycan fl- awrina ta ’ pazjenti kkurati mqabbla mal - plaċebo fl- 24 Ġimgħa.
Thirty-eight patients subsequently enrolled in an Open Label extension study where they received 1 mg/ kg of galsulfase every week.
Wara, tminja u tletin pazjent iddaħħlu fi studju Open Label mtawwal, fejn irċevew 1 mg/ kg ta ’ galsulfase kull ġimgħa.
Following 24 weeks of therapy, Naglazyme-treated patients experienced a 92 ± 40 m improvement in the distance walked in 12 minutes relative to placebo-treated patients (p = 0.025).
Wara 24 ġimgħa ta ’ terapija, pazjenti kkurati b’ Naglazyme esperjenzaw titjib ta ’ 92 ± 40 m fid - distanza li mxew fi 12- il minuta meta mqabbla mal- pazjenti kkurati bil- plaċebo (p = 0. 025).
Treated patients experienced a 5.7 stair per minute improvement in the 3 Minute Stair Climb relative to placebo-treated patients.
Pazjenti kkurati wrew titjib ta ’ 5. 7 tarġiet kull minuta fil- prova ta ’ Tlugħ ta ’ Taraġ ta ’ 3 Minuti, meta mqabbla mal- pazjenti kkurati bil- plaċebo.
Treated patients also experienced a mean decrease in urinary glycosaminoglycan excretion of 238 ± 17.8 μ g/ mg creatinine ± SE following 24 weeks of treatment relative to placebo-treated patients.
Pazjenti kkurati esperjenzaw ukoll tnaqqis medju fit- tneħħija ta ’ glycosaminoglycan fl- awrina ta ’ 238 ± 17. 8 μg/ mg krejatinina ± SE wara 24 ġimgħa ta ’ kura meta mqabbla mal- pazjenti kkurati bil- plaċebo.
GAG results approached the normal range for age in the Naglazyme treatment group.
Ir- riżultati GAG qarrbu il- firxa normali għall- età fil- grupp ta’ kura b’ Naglazyme.
The pharmacokinetics of galsulfase were evaluated in 13 patients with MPS VI who received 1 mg/ kg of galsulfase as a 4 hour infusion.
Il- farmakokinetiċi ta ’ galsulfase kienu evalwati fi 13- il pazjent b‘ MPS VI li rċevew 1 mg/ kg ta ’ galsulfase bħala infużjoni fuq medda ta ’ 4 sigħat.
After 24 weeks of treatment the mean (± Standard Deviation [SD]) maximum plasma concentration (Cmax) was 2,357 (± 1,560) ng/ ml and the mean (± SD) area under
Wara 24 ġimgħa ta ’ kura l- medja (± Devjazzjoni Stàndard [SD]) ta ’ konċentrazzjoni massima fil- plazma (Cmax) kienet ta ’ 2, 357 (± 1, 560) ng/ ml u l - medja ta ’ l- erja (± SD) taħt il- kurva ta ’ konċentrazzjoni fil- plażma- ħin (AUC 0- t) kienet 5, 860 (± 4, 184) h × ng/ ml.
The mean (± SD) volume of distribution (Vz) was 316 (± 752) ml/ kg and the mean (± SD) plasma clearance (CL) was 7.9 (± 14.7) ml/ min/ kg.
Il- volum ta ’ distribuzzjoni medju fil- plażma (Vz) (± SD) kien 316 (± 752) ml/ kg u l- medja tat- tneħħija mill- plażma (CL) (± SD) kienet 7. 9 (± 14. 7) ml/ min/ kg.
The mean (± SD) elimination half-life (t1/ 2) was 22.8 (± 10.7) minutes at Week 24.
Il - half- life (t1/ 2) ta ’ eliminazzjoni medja (± SD) kienet 22. 8 (± 10. 7) minuti fl- 24 ġimgħa.
Pharmacokinetic parameters in Phase 1 patients have remained stable over the long term (through at least 194 weeks).
Il- parametri farmakokinetiċi fil- pazjenti ta ’ Fażi 1 baqgħu stabbli għal tul ta ’ żmien twil (ta ’ mill - anqas 194 ġimgħa).
Galsulfase is a protein and is expected to be metabolically degraded through peptide hydrolysis.
Galsulfase huwa proteina u huwa mistenni li jiġi degradat metabolikament permezz ta ’ idrolisi ta ’ peptide.
Consequently, impaired liver function is not expected to affect the pharmacokinetics of galsulfase in a clinically significant way.
Konsegwentament, funzjoni tal- fwied indebbolita mhix mistennija li taffettwa l - farmakokinetiċi ta ’ galsulfase b’ mod sinifikanti klinikament.
Renal elimination of galsulfase is considered a minor pathway for clearance (see section 4.2).
L- eliminazzjoni renali ta ’ galsulfase hija kkunsidrata bħala passaġġ minuri għat- tneħħija (ara sezzjoni 4. 2).
Non-clinical data revealed no special hazard for humans based on conventional studies of safety pharmacology, single-dose toxicity, repeated-dose toxicity or on general reproductive performance or embryo-foetal development in rats or rabbits.
Informazzjoni li mhix klinika, ma ’ turi l- ebda periklu speċjali għall- bnedmin ibbażata fuq studji konvenzjonali ta ’ sigurtà farmakoloġika, effett tossiku minn doża waħda, effett tossiku minn dożi ripetuti, jew fuq il- funzjoni riproduttiva ġenerali, jew l- iżvilupp ta ’ l- embriju/ fetu fil- firien jew fil - fniek.
Peri- and post-natal toxicity has not been investigated.
Tossiċità ta ’ waqt u wara t- twelid ma ġietx investigata.
Genotoxic and carcinogenic potential are not expected.
Mhux mistenni potenzjal ġenotossiku jew karċinoġeniku.
The cause of clinical relevance of the hepatic toxicity (bile duct hyperplasia / periportal inflammation) seen at clinically relevant doses in the repeated dose monkey toxicity study is not known.
Il- kawża ta ’ rilevanza klinika tat- tossiċità epatika (iperplasija tal- kanal biljari / infjammazzjoni periportali) li deheret f’ dożi ta ’ rilevanza klinika fl- istudju ta ’ dożi ripetuti fix- xadini mhix magħrufa.
Sodium chloride Sodium phosphate monobasic, monohydrate Sodium phosphate dibasic, heptahydrate Polysorbate 80 Water for injections
Sodium chloride Sodium phosphate monobasic, monohydrate Sodium phosphate dibasic, heptahydrate Polysorbate 80 Ilma għall- injezzjoni
36 months.
36 xhar
Chemical and physical in-use stability has been demonstrated for up to 4 days at room temperature (23°C - 27°C).
Stabbiltà kimika u fiżika waqt l- użu kienet demostrata sa 4 ijiem f’ temperatura tal- kamra (23 °C - 27 °C).
From a microbiological safety point of view, Naglazyme should be used immediately.
Mill- aspett ta ’ sigurta ’ mikrobijoloġika, Naglazyme għandu jintuża ’ immedjatament.
If not used immediately, in-use storage times and conditions are the responsibility of the user and should normally not be longer than 24 hours at 2°C - 8°C followed by up to 24 hours at room temperature (23°C - 27°C) during administration.
Jekk ma jintużax immedjatament, il- ħin u l- kondizzjonijiet ta ’ ħażna waqt l- użu huma r- responsabbiltà ta ’ minn qed juża ’ l- prodott, u normalment m’ għandhomx ikunu itwal minn 24 siegħa f’ temperatura ta ’ 2 °C – 8 °C, segwit minn massimu ta ’ 24 siegħa f’ temperatura tal- kamra (23 °C – 27 °C) waqt l- amministrazzjoni.
Store in a refrigerator (2°C to 8°C).
Aħżen fi friġġ (2°C – 8°C).
7 For storage conditions of the diluted medicinal product, see section 6.3.
Għall- kundizzjonijiet ta ’ ħażna tal- prodott mediċinali dilwit, ara sezzjoni 6. 3.
Vial (type I glass) with a stopper (siliconized chlorobutyl rubber) and a seal (aluminium) with a flip- off cap (polypropylene).
Kunjett (ħġieġ tat- tip I) b’ tapp (lasktu siliconized tal- chlorobutyl) u siġill (aluminju) u għatu li jinfetaħ b’ daqqa ta ’ saba ’ (polypropylene).
Package sizes:
Daqsijiet tal- pakketti:
Not all package sizes may be marketed.
1 u 6 kunjett Jista ’ jkun li mhux il- pakketti tad- daqsijiet kollha jkunu għall- skop kummerċjali.
Each vial of Naglazyme is intended for single use only.
Kull kunjett ta ’ Naglazyme huwa maħsub għall- użu ta ’ darba biss.
The concentrate for solution for infusion has to be diluted with sodium chloride 9 mg/ ml (0.9%) solution for infusion using aseptic technique.
Il- konċentrat għal soluzzjoni għall - infużjoni jrid jiġi dilwit b’ soluzzjoni għall- infużjoni ta ’ sodium chloride 9 mg/ ml (0. 9%) permezz ta ’ teknika asettika.
It is recommended that the diluted Naglazyme solution be administered to patients using an infusion set equipped with a 0.2 µm in-line filter.
Huwa rakkomandat li s- soluzzjoni dilwita ta ’ Naglazyme tingħata lill- pazjenti permezz ta ’ sett ta ’ l- infużjoni mgħammar b’ filter in- line ta ’ 0. 2 µm.
Any unused product or waste material is to be disposed of in accordance with local requirements.
Kull fdal tal- prodott li ma jkunx intuża jew skart li jibqa wara l- użu tal- prodott għandu jintrema kif jitolbu l- liġijiet lokali.
6.7 Preparation of the Naglazyme Infusion (Aseptic Technique is to be used)
6. 7 Preparazzjoni ta ’ l- Infużjoni ta ’ Naglazyme (Teknika Asettika li għandha tintuża)
The number of vials to be diluted based on the individual patient's weight must be determined and removed from the refrigerator approximately 20 minutes in advance in order to allow them to reach room temperature.
In- numru ta ’ kunjetti li jridu jiġu dilwiti għandu jiġi determinat skond il- piż individwali tal- pazjent u għandhom jinħarġu mill- friġġ madwar 20 minuta minn qabel biex b’ hekk ikunu jistgħu jilħqu temperatura tal- kamra.
Before dilution, each vial is to be inspected for particulate matter and discolouration.
Qabel id- dilwazzjoni, kull kunjett għandu jiġi spezzjonat għall- frak jew telf ta ’ kulur.
The clear to slightly opalescent and colourless to pale yellow solution must be free of visible particles.
Is- soluzzjoni ċara għal kemmxejn tkanġi, u bla kulur għal safra ċara m’ għandux ikollha frak viżibbli viżibbli.
A volume of the sodium chloride 9 mg/ ml (0.9%) solution for infusion is to be withdrawn and discarded from a 250 ml infusion bag equal to the total volume of Naglazyme to be added.
Volum ta ’ soluzzjoni għall- infużjoni ta ’ sodium chloride 9 mg/ ml (0. 9%) ugwali għall- volum totali ta ’ Naglazyme li jrid jiġi miżjud, għandu jittiħed mill- borża ta ’ l- infużjoni ta ’ 250 ml u jintrema.
100 ml infusion bags should be considered for patients who are susceptible to fluid volume overload and weigh less than 20 kg; in this case the infusion rate (ml/ min) should be decreased so that the total duration remains no less than 4 hours.
Boroż ta ’ l- infużjoni ta ’ 100 ml għandhom jiġu kkunsidrati għall- pazjenti li huma suxxettibli għall- ammont eċċessiv ta ’ volum ta ’ fluwidi u jiżnu anqas minn 20 kg; f’ dan il- każ ir- rata ta ’ l- infużjoni (ml/ min) għandha titnaqqas biex b’ hekk l- infużjoni tingħata fuq medda ta ’ ħin totali ta ’ mill- anqas 4 sigħat.
When using 100 ml bags, the volume of Naglazyme may be added directly to the infusion bag.
Meta jintużaw boroż ta ’ 100 ml, il- volum ta ’ Naglazyme jista ’ jiġi miżjud direttament fil- borża ta ’ l - infużjoni.
The volume of Naglazyme is to be slowly added to the sodium chloride 9 mg/ ml (0.9%) solution for infusion.
Il- volum ta ’ Naglazyme għandu jiġi miżjud bil- mod mas- soluzzjoni għall- infużjoni ta ’ sodium chloride 9 mg/ ml (0. 9%).
The solution is to be mixed gently before infusion.
Is- soluzzjoni trid titħallat bil- mod qabel l- infużjoni.
The solution is to be visually inspected for particulate matter prior to use.
Is- soluzzjoni għandha tiġi spezzjonata viżwalment biex jiġi ċċekkjat jekk hemmx xi frak qabel l- użu.
Only clear and colourless solutions without visible particles should be used.
Għandhom jintużaw biss soluzzjonijiet ċari, bla kulur u mingħajr frak viżibbli.
BioMarin Europe Limited, 164 Shaftesbury Avenue London, WC2H 8HL United Kingdom
BioMarin Europe Limited, 164 Shaftesbury Avenue London, WC2H 8HL
24 January 2006
24 ta ’ Jannar 2006
10 A MANUFACTURER OF THE BIOLOGICAL ACTIVE SUBSTANCE AND MANUFACTURING AUTHORISATION HOLDER RESPONSIBLE FOR BATCH RELEASE
MANIFATTUR TAS- SUSTANZA BIJOLOĠIKA ATTIVA U DETENTUR TA ’ L - AWTORIZZAZZJONI TAL- MANIFATTURA RESPONSABBLI GĦALL- HRUĠ TAL - LOTT
BioMarin Pharmaceutical Inc., 46 Galli Drive, Novato, CA 94949 United States of America
BioMarin Pharmaceutical Inc., 46 Galli Drive, Novato, CA 94949 L- Istati Uniti ta ’ l- Amerika
Catalent UK Packaging Ltd Wingates Industrial Park, Westhoughton, Bolton, Lancs, BL5 3XX United Kingdom
Catalent UK Packaging Ltd Wingates Industrial Park, Westhoughton, Bolton, Lancs, BL5 3XX Ir- Renju Unit
The MAH commits to performing the studies and additional pharmacovigilance activities detailed in the Pharmacovigilance Plan.
Id- Detentur ta ’ l- Awtorizzazzjoni għat- tqegħid fis- Suq jikkommetti ruħu li jwettaq l- studji u attivitajiet oħra ta ’ farmakoviġilanza li huma dettaljati fil- Pjan ta ’ Farmakoviġilanza.
An updated Risk Management Plan should be provided as per the CHMP Guideline on Risk Management System for medicinal products for human use.
Skond il- Linji Gwida tas- CHMP dwar is- Sistema ta ’ l- Immanniġjar ta ’ Riskji għall- prodotti mediċinali għall- użu uman għandu jiġi pprovdut Pjan għall- Immanniġjar ta ’ Riskji aġġornat.
The Marketing Authorisation Holder shall complete the following programme of studies within the specified time frame, the results of which shall form the basis of the annual reassessment of the benefit/ risk profile.
Id- Detentur ta ’ l- Awtorizzazzjoni għat- tqegħid fis- Suq għandu jtemm il- programm ta 'studji li ġejjin fiż- żmien speċifikat, u r- riżultati ta' dawn l- istudji għandhom iservu ta 'bażi tal- valutazzjoni mill- ġdid ta ’ kull sena tal- profil tal- benefiċċju/ riskju.
11 Specific Obligations:
11 Obligazzjonijiet speċifiċi:
Description:
Deskrizzjoni:
Due Date
Data ta ’ l- għeluq
Module 5 – Clinical
Modulu 5 – Kliniku
SO2 001.3 To evaluate the long-term safety and efficacy data from Naglazyme treatment a Clinical Surveillance Program (CSP) will be conducted.
SO2 001. 3 Se jsir Programm ta ’ Sorveljanza Klinika (CSP) biex ikun evalwat tagħrif ta ’ sigurtà u effikaċja fit- tul minn kura b’ Naglazyme.
Substudies will be conducted within the CSP that will:
Fis- CSP ser jitwettqu sotto- studji li:
Evaluate the effect of Naglazyme on pregnancy and lactation.
Jevalwaw l- effett ta ’ Naglazyme fuq it- tqala u t- treddigħ.
Interim results will be provided in the CSP Annual Reports.
Riżultati parzjali se jiġu pprovduti fir - Rapporti Annwali tas- CSP.
Evaluate the safety and efficacy of Naglazyme in 10 children less than 5 years of age will be treated with the 1 mg/ kg dose for at least one year.
Jevalwaw is- sigurtà u l- effikaċja ta ’ Naglazyme f’ għaxart itfal ta ’ età taħt il- ħames snin, ikkurati b’ doża ta ’ 1 mg/ kg għal mill- inqas sena.
Information will also be collected on clinical status, adverse events, assessments of immunogenicity and potential effects on antibody formation The CSP data will be analysed at yearly intervals and results will be submitted as annual reports.
It- tagħrif mis- CSP se jiġi analizzat f’ intervalli ta ’ sena u r- riżultati se jingħataw bħala rapporti annwali.
Detailed clinical status information will be collected at study entry and on an annual basis for at least 15 years.
Tagħrif dettaljat ta ’ l- istat kliniku se jinġabar mad- dħul fl- istudju u fuq bażi annwali għal mill- inqas 15- il sena.
Other measures (urinary GAG, antibodies) are assessed more frequently.
Miżuri oħra (GAG fl- awrina, antikorpi) jiġu stmati aktar ta ’ spiss.
Severe and serious liver toxicities will be assessed through the PSUR but also through analysis of these events in the CSP database.
Tossiċitajiet severi u serji tal- fwied se jiġu stmati permezz tal- PSUR iżda wkoll permezz ta ’ l- analiżi ta ’ dawn l- avvenimenti fid- database tas- CSP.
Subjects enrolled in the program will have urinary glycosaminoglycan and samples for IgG antibodies routinely collected as specified in the Schedule of Activities.
Individwi li se jieħdu sehem fil- programm se jkollhom il- glycosaminoglycan fl- awrina u kampjuni għall- antikorpi IgG miġbura b’ mod regolari kif speċifikat fl- Iskeda ta ’ l- Attivitajiet.
Higher antibody levels (> 10OD/ uL) will be compared with the subject ’ s urinary GAG values with a view to assess potential impacts on efficacy.
Livelli ta ’ antikorpi ogħla (> 10OD/ uL) se jiġu mqabbla mal- valuri ta ’ GAG fl- awrina ta ’ l- individwu bi ħsieb li jiġu stmati l- impatti potenzjali fuq l- effikaċja.
Samples from those subjects who have a consistent increase in urinary GAG values together with high antibody levels will have their antibody samples assessed for evidence of neutralizing activities.
Kampjuni minn dawk l- individwi li għandhom żjieda konsistenti fil- figuri ta ’ GAG fl- awrina flimkien ma ’ livelli ta ’ l- antikorpi għoljin se jkollhom il- kampjuni ta ’ l- antikorpi tagħhom analizzati għall- evidenza ta ’ attivitajiet newtralizzanti.
Samples for IgG antibody will be collected at specific intervals.
Kampjuni għall- antikorp IgG se jinġabru f’ intervalli speċifiċi.
If a physician suspects an IgE mediated reaction they have been advised in the protocol to request IgE antibody presence to be conducted by the MAH.
Jekk tabib jissuspetta reazzjoni medjata minn IgE, dawn ġew irrakkomandati fil- protokoll sabiex jitolbu li ssir tfittxija għall- preżenza ta ’ l- antikorp IgE mid- Detentur ta ’ l- Awtorizzazzjoni għat- Tqegħid fis- Suq.
This protocol has been initiated in the United States and will be submitted to Ethics Committees in several EU countries (UK, Germany, France, Portugal, Italy) with known MPS VI patients at the end of September 2005.
Dan il- protokoll inbeda fl- Istati Uniti u se jingħata lill- Kumitati ta ’ l- Etika f’ diversi pajjiżi ta ’ l- EU (Ir- Renju Unit, il- Ġermanja, Franza, il- Portugal, l - Italja) fejn hu magħruf li hemm pazjenti b’ MPS VI, fl- aħħar ta ’ Settembru 2005.
The final study report under this CSP will be submitted by 31 July 2020.
Ir- rapport finali ta ’ l- istudju taħt din is- CSP ser jingħata sal- 31 ta ’ Lulju 2020.
A brief update will be submitted as part of the Annual Reassessments.
Aġġornament qasir se jingħata bħala parti mill - Valutazzjoni mill - Ġdid ta ’ Kull Sena.
SOB 002 To evaluate the long-term safety and efficacy data from Naglazyme treatment a Clinical Surveillance Program (CSP) will be conducted.
SOB 002 Biex jiġi evalwat tagħrif dwar is- sigurtà u l- effikaċja fit- tul minn kura b’ Naglazyme ser isir Programm ta ’ Sorveljanza Klinika (CSP).
Subjects enrolled in the program will have urinary glycosaminoglycan and samples for IgG antibodies routinely collected as specified in the Schedule of Activities.
Individwi li se jieħdu sehem fil- programm se jkollhom il- glycosaminoglycan
Final CSP Study Report:
Rapport Finali ta ’ l- Istudju tas- CSP:
31 July 2020
31 ta ’ Lulju 2020
12 urinary GAG values together with high antibody levels will have their antibody samples assessed for evidence of neutralizing activities.
12 fl- awrina u kampjuni għall- antikorpi IgG miġbura b’ mod regolari kif speċifikat fl- Iskeda ta ’ l- Attivitajiet.
Samples for IgG antibody will be collected at specific intervals.
Livelli ta ’ antikorpi ogħla (> 10OD/ uL) se jiġu mqabbla mal- valuri ta ’ GAG fl- awrina ta ’ l- individwu bi ħsieb li jiġu stmati l- impatti potenzjali fuq l- effikaċja.
SO2 003.2 Several measures will be implemented to assess the Naglazyme dose.
SO2 003. 2 Diversi miżuri se jiġu mplimentati biex tiġi stmata d- doża ta ’ Naglazyme.
Data collected in the post-marketing phase will also be examined to determine if a suitable Naglazyme maintenance dose can be recommended relative to the efficacy endpoints used in the clinical studies.
Se jiġi eżaminat ukoll tagħrif miġbur fil- fażi ta ’ wara t- tqegħid fis- suq biex jiġi determinat jekk hux possibli li tiġi rrakkomandata doża xierqa ta ’ manteniment ta ’ Naglazyme relattiva għall- miri tal- effikaċja użati fil- provi kliniċi.
SO2 004.2 All clinical trial patients are currently receiving the product manufactured by the commercial process.
31 ta ’ Mejju 2008 SO2 004. 2 Il- pazjenti kollha ta ’ l- istudju kliniku qed jingħataw il- prodott manifatturat bil - proċess kummerċjali.
GAG and antibody samples continue to be collected at protocol-specified intervals.
Kampjuni ta ’ GAG u ta ’ antikorpi se jibqgħu jinġabru f’ intervalli speċifikati fil- protokoll.