qanastek/EMEA-V3 huggingface dataset:
Analyses of efficacy did not reveal meaningful differences between the 2 dose groups with respect to survival, invasive ventilator-free survival, any ventilator-free survival, decrease in LVM, gains in growth parameters and acquisition of motor milestones.
L- analiżi ta ’ l- effikaċja ma żvelatx differenzi sinifikanti bejn iż- 2 gruppi li kienu qed jieħdu d- doża fir - rigward tas- sopravivenza, sopravivenza mingħajr sapport ta ’ ventilazzjoni invażiva, kwalunkwe sopravivenza mingħajr ventilatur, tnaqqis fil- LVM, titjib fil- parametri tat- tkabbir u l- akkwist ta ’ milestones tal- moviment.
Based on these results the 20 mg/ kg qow dose is recommended.
Ibbażat fuq dawn ir- riżultati, id- doża ta ’ 20 mg/ kg kull ġimagħtejn hi rakkomandata.
Infantile-onset Pompe disease; clinical trial in patients aged 6 months to 3.5 years A second open-label clinical trial also assessed the safety and efficacy of Myozyme in 21 patients with predominantly a non-typical form of infantile-onset Pompe disease who ranged in age from 6 months to 3.5 years at initiation of treatment.
8 Pazjenti li fihom il- marda ta ’ Pompe bdiet fl- infanzja; prova klinika f’ pazjenti li kellhom 6 xhur sa 3 snin u nofs It- tieni studju kliniku tat- tip open- label evalwa wkoll is- sigurtà u effikaċja ta ’ Myozyme f’ 21 pazjent li fil- biċċa l- kbira tagħhom kellhom forma mhux tipika tal- marda ta ’ Pompe li bdiet fl- infanzja, li varjaw fl- età minn 6 xhur sa 3. 5 snin fil- bidu tal- kura.
Patients received 20 mg/ kg Myozyme every other week for 52 weeks except for 8 patients who received 40 mg/ kg after at least 26 weeks of treatment.
Il- pazjenti rċivew 20 mg/ kg ta ’ Myozyme ġimgħa iva u oħra le għal 52 ġimgħa ħlief għal 8 pazjenti li rċivew 40 mg/ kg wara mill- inqas 26 ġimgħa ta ’ kura.
After 52 weeks all patients continued treatment for a total duration of more than 3 years (168 weeks with a median of 121 weeks).
Wara 52 ġimgħa, il- pazjenti kollha komplew il- kura għal total ta ’ żmien ta ’ iktar iktar minn 3 snin (168 ġimgħa b’ medjan ta ’ 121 ġimgħa).
8 The primary endpoint of the pivotal trial was the proportion of patients who were alive.
Ir- riżultat aħħari primarju tal- prova importanti ħafna kien il- proporzjon ta ’ pazjenti li kienu ħajjin.
After 52 weeks of treatment, 16 of 21 patients (76.2%) treated with Myozyme were alive.
Wara 52 ġimgħa ta ’ kura, 16 minn 21 pazjent (76. 2%) kkurati b’ Myozyme kienu ħajjin.
After 104 weeks of treatment, 14 of 21 patients (66.7%) were alive and 1 patient was alive but had discontinued from the study.
Wara 104 ġimgħat ta ’ kura, 14 minn 21 pazjent (66. 7%) kienu ħajjin u pazjent 1 kien ħaj iżda kien waqaf jipparteċipa fl- istudju.
These proportions were maintained up to the end of the study (with individual patient treatment durations ranging from 1 to 168 weeks; mean follow-up period of 109 weeks).
Dawn il- porzjonijiet inżammu sat- tmiem tal- istudju (bit- tul tal- kura ta ’ pazjenti individwali li varjaw minn 1 sa 168 ġimgħa; il- medja tal- perjodu follow- up kienet ta ’ 109 ġimgħa).
In the untreated historical cohort 5 of 47 patients (10.6%) for whom data were available, were alive at age 30 months (2.5 years).
Fil- koorti storiku mhux ikkurat, 5 minn 47 pazjent (10. 6%) li għalihom kien hemm dejta disponibbli, kienu ħajjin fl- età ta ’ 30 xahar (sentejn u nofs).
Survival in the treated patients was compared to survival in a similar historical cohort of untreated subjects using a Cox proportional hazards regression analysis (See Table 3).
Is- sopravivenza fil- pazjenti kkurati tqabblet mas- sopravivenza f’ koorti storiku simili ta ’ pazjenti mhux ikkurati bl- użu ta ’ l- analiżi tar- rigressjoni tal- perikli proporzjonali ta ’ Cox (Ara Tabella 3).
Results for endpoint survival using the Cox regression model
Riżultati għas- sopravivienza tar- riżultat aħħari bl- użu tal- mudell tar- rigressjoni ta ’ Cox Proporzjon
Historical
Komparatur
Treatment
tal- Periklu
Treated
Pazjenti Storiku tar -
Reference
tal- Kura -
Effect Hazard Confidence
Intervall tal -
Patients
Kkurati Referenza
Comparator
Riżultat aħħari
Endpoint
Effett
Ratio Interval
Kunfidenza
N=21
N=48
N=48
N=21
Survival
Sopravivenza
0.301
0. 301
(0.112,0.804)
(0. 112, 0. 804)
0.0166
0. 0166
Subjects ranged in age from 6 months to 3.5 years at initiation of treatment.
Il- pazjenti varjaw minn età ta ’ 6 xhur sa 3. 5 snin fil- bidu tal- kura.
Subjects in the untreated historical cohort were born in 1995 or later.
Il- pazjenti fil- koorti storiku mhux ikkurat twieldu fl- 1995 jew wara.
Additional efficacy data showed that of 16 patients who were free of invasive-ventilator support at baseline, 7 remained so after 104 weeks of treatment.
Informazzjoni addizzjonali dwar l- effikaċja wriet li mis- 16- il pazjent li kienu ħielsa minn sapport ta ’ ventilazzjoni invażiva fil- linja bażi, 7 baqgħu hekk wara 104 ġimgħat ta ’ kura.
The 9 remaining patients either died (5 patients) or became invasive-ventilator dependent (4 patients).
Id- 9 pazjenti li kien baqa ’, jew mietu (5 pazjenti) jew saru dipendenti fuq sapport invażiv bil- ventilatur (4 pazjenti).
All 5 patients who were receiving invasive ventilation at baseline continued to require ventilation throughout the study (4 patients survived beyond week 104 and one patient died).
Il- 5 pazjenti kollha li kienu qed jirċievu ventilazzjoni invażiva fil- linja bażi baqa ’ jkollhom bżonn tal - ventilazzjoni matul l- istudju kollu (4 pazjenti għexu wara ġimgħa 104 u pazjent wieħed miet).
After 52 weeks of treatment, LVM decreased from baseline in all 12 patients with available data and was within normal limits in 6 of 12 patients.
Wara 52 ġimgħa ta ’ kura, l- LVM naqas mil- linja bażi fit- 12- il pazjent kollha b’ dejta disponibbli, u kienu fil- limiti normali f’ 6 minn 12- il pazjent.
After the first year (58 up to 168 weeks) of treatment LVM further decreased in 9 out of 12 patients with available data.
Wara l- ewwel sena (minn 58 sa 168 ġimgħa) ta ’ kura, l - LVM naqas b’ mod ulterjuri f’ 9 minn 12- il pazjent b’ dejta disponibbli.
At 104 weeks of treatment LVM assessments were available for 10 patients, of which 9 decreased to within normal limits.
Wara 104 ġimgħat ta ’ kura, evalwazzjonijiet tal- LVM kienu disponibbli għal 10 pazjenti, li minnhom 9 naqsu sal- limiti normali.
After 52 weeks of treatment, 3 out of 8 patients with available data made gains in motor function over baseline as measured by raw scores and age-equivalent scores from baseline in the AIMS.
Wara 52 ġimgħa ta ’ kura, 3 minn 8 pazjenti b’ dejta disponibbli kellhom titjib fil- funzjoni tal - moviment fuq il- linja bażi kif imkejjel mill- punteġġi mhux analizzati u punteġġi ekwivalenti għall- età mil- linja bażi fl- AIMS.
Six of the 11 patients with available data continued to make motor development gains beyond Week 52 (with individual patient treatment durations ranging from 58 to 168 weeks; mean follow-up period of 121 weeks), including 3 patients ambulatory and 3 patients with only functional sitting skills by the last study visit.
Sitta mill- 11- il pazjent b’ dejta disponibbli komplew jiksbu titjib fl- iżvilupp tal- moviment wara Ġimgħa 52 (bit- tul tal- kura ta ’ pazjenti individwali li varja minn 58 sa 168 ġimgħa; il- medja tal- perjodu follow- up kienet ta ’ 121 ġimgħa), li jinkludu 3 pazjenti ambulatorji u 3 pazjenti li kellhom biss ħiliet funzjonali li joqogħdu bilqiegħda sal- aħħar viżta tal- istudju.
The remaining 5 patients showed no significant change in motor development beyond Week 52 (with individual patient treatment durations ranging from 104 to 168 weeks; mean follow-up period of 140 weeks), including 4 patients with no significant motor skills in any of the positions evaluated and 1 patient with only functional sitting skills by the last study visit.
Il- 5 pazjenti l- oħra ma wrew l- ebda żvilupp sinifikanti jibdlu fl- iżvilupp tal- moviment wara minn Ġimgħa 52 (bit- tul tal- kura ta ’ pazjenti individwali li varja minn 104 sa 168 ġimgħa; il- medja tal- perjodu follow- up kienet ta ’ 140 ġimgħa), li jinkludi 4 pazjenti mingħajr l- ebda ħiliet sinifikanti tal- moviment fi kwalunkwe mill - pożizzjonijiet evalwati, u pazjent 1 li kellu biss ħiliet funzjonali li joqgħod bilqiegħda sal- aħħar viżta tal- istudju.
The vast majority of patients with infantile-onset Pompe disease treated with Myozyme demonstrate improvement in cardiac function as well as stabilisation or improvements in growth parameters.
9 Il- maġġoranza l- kbira tal- pazjenti li fihom il- marda ta ’ Pompe bdiet fl- infanzja u li kienu kkurati b’ Myozyme, juru titjib fil- funzjoni kardijaka kif ukoll stabbilizzazzjoni jew titjib fil- parametri tat - tkabbir.
However, motor and respiratory responses to treatment have been more variable.
Madankollu, ir- risponsi tal- moviment u dawk respiratori għall- kura kienu aktar varjabbli.
Patients with infantile-onset Pompe disease who demonstrated motor gains, had greater preservation of motor function and lower glycogen content in the quadriceps muscle at baseline.
Pazjenti li fihom il- marda ta ’ Pompe bdiet fl- infanzja li urew titjib fil- moviment, kellhom preservazzjoni akbar tal- funzjoni tal- moviment u kontenut ta ’ glycogen aktar baxx fil- muskolu quadriceps fil- linja bażi.
It is noteworthy that a higher proportion of patients with better motor outcomes show stability or improvement in growth parameters (weight), while the large majority of patients, regardless of their motor outcomes or baseline features, show reversal of cardiomyopathy as measured by changes in LVM Z-score.
Ta ’ min jinnota li proporzjon ogħla ta ’ pazjenti b’ riżultati aħjar tal - moviment, juru stabbiltà jew titjib fil- parametri tat- tkabbir (piż), filwaqt li l- maġġoranza kbira tal - pazjenti, irrispettivament mir- riżultati tal- moviment jew tal- karatteristiċi fil- linja bażi tagħhom, juru t - treġġigħ lura tal- kardjomijopatija, hekk kif imkejla mit- tibdil fil- punteġġ Z tal- LVM.
The totality of the data suggests that early diagnosis and treatment at an early stage of disease may be critical to achieve the best outcomes in these infantile onset patients.
It- totalità ta ’ l- informazzjoni tissuġġerixxi li dijanjosi bikrija u l- kura f’ fażi bikrija tal- marda jistgħu jkunu kritiċi biex jintħaqu l- aħjar riżultati f’ dawn il- pazjenti li fihom il- marda bdiet fl- infanzja.
9 Late-onset Pompe disease An open-label clinical trial assessed the safety and efficacy of Myozyme in 5 patients with late-onset Pompe disease who ranged in age from 5 to 15 years at initiation of treatment.
Pazjenti li fihom il- marda ta ’ Pompe bdiet tard fil- ħajja Studju kliniku tat- tip open- label evalwa s- sigurtà u effikaċja ta ’ Myozyme f’ 5 pazjenti li fihom il - marda ta ’ Pompe bdiet tard fil- ħajja, li varjaw fl- età minn 5 sa 15- il sena fil- bidu tal- kura.
Patients received 20 mg/ kg Myozyme every other week for 26 weeks.
Il- pazjenti rċivew 20 mg/ kg ta ’ Myozyme ġimgħa iva u oħra le għal 26 ġimgħa.
All patients were freely ambulatory and all but one patient did not require any form of ventilator support (1 patient required nocturnal non-invasive ventilation).
Il- pazjenti kollha kienu jimxu b’ mod ħieles u kollha ħlief wieħed ma kellhomx bżonn ta ’ xi forma ta ’ sapport bil- ventilatur (pazjent wieħed kien jeħtieġ ventilazzjoni mhux invażiva matul il- lejl).
Of the 3 patients with significant pulmonary involvement at screening/ baseline (percentage predicted forced vital capacity in the sitting position ranging from 58-67%), two demonstrated clinically meaningful improvements in FVC (+11.5% and +16.0%) in the sitting position by Week 26.
Mit- 3 pazjenti b’ involviment pulmonari sinifikanti fl- iscreening/ linja bażi (il- perċentwali bassret il- ħila vitali sfurzata fil- pożizzjoni ta ’ bilqiegħda li varjat minn 58 - 67%), tnejn urew titjib klinikament sinifikanti fl- FVC (+11. 5% u +16. 0%) fil- pożizzjoni ta ’ bilqiegħda minn ġimgħa 26.
Evaluation of motor function gave disparate results.
L- evalwazzjoni tal- funzjoni tal- moviment tat riżultati differenti.
Ten patients with advanced late-onset Pompe disease (i. e. wheelchair-bound for 10/ 10 and ventilator- dependent for 9/ 10) aged 9-54 years were treated in expanded access programs with alglucosidase alfa 20-40 mg/ kg every 2 weeks for various periods of time between 6 months and 2.5 years.
Għaxar pazjenti li fihom il- marda ta ’ Pompe bdiet tard fil- ħajja, bil- marda fi stat avvanzat (i. e. li kienu f’ siġġu bir- roti għal 10/ 10 u jiddependu fuq ventilatur għal 9/ 10), li kellhom minn 9 sa 54 sena, kienu kkurati fi programmi b’ aċċess imkabbar b’ alglucosidase alfa 20- 40 mg/ kg kull ġimagħtejn għal diversi perjodi ta ’ żmien ta ’ bejn 6 xhur u 2. 5 snin.
The pulmonary benefits observed in patients included a clinically meaningful improvement in FVC of 35% in one patient, and significant reductions in the number of hours of ventilator support needed in 2 patients.
Il- benefiċċji pulmonari osservati fil- pazjenti inkludew titjib klinikament sinifikanti fl- FVC ta ’ 35% f’ pazjent wieħed, u tnaqqis sinifikanti fin- numru ta ’ sigħat ta ’ sapport tal- ventilatur meħtieġ f’ 2 pazjenti.
Benefits of treatment on motor function including the regaining of lost motor skills were observed in some patients.
Il- benefiċċji tal- kura fuq il- funzjoni moviment, li jinkludu l- ksib lura ta ’ ħiliet tal- moviment, kienu osservati f’ xi pazjenti.
Only one patient became wheelchair-free.
Pazjent wieħed biss ma baqax fis- siġġu bir- roti.
In this group of patients a variable response has also been seen with respect to motor function.
F’ dan il- grupp ta ’ pazjenti, rispons varjabbli kien osservat ukoll fir- rigward tal - funzjoni tal- moviment.
Pompe Registry Medical or healthcare professionals are encouraged to register patients who are diagnosed with Pompe disease at www. PompeRegistry. com.
Pompe Registry Persuni mediċi jew professjonisti fil- qasam tas- saħħa huma inkoraġġiti biex jirreġistraw pazjenti li huma dijanjostikati bil- marda ta ’ Pompe, fuq www. PompeRegistry. com.
Patient data will be anonymously collected in this Registry.
L- informazzjoni dwar il - pazjenti ser tinġabar b’ mod anonimu f’ din ir- Registry.
The objectives of the “ Pompe Registry” are to enhance the understanding of Pompe disease and to monitor patients and their response to enzyme replacement therapy over time, with the ultimate goal of improving clinical outcomes for these patients.
L- għanijiet tal - “ Pompe Registry ” huma li jkabbru l- għarfien dwar il- marda ta ’ Pompe u li jsir monitoraġġ tal- pazjenti u r- rispons tagħhom għat - terapija tas- sostituzzjoni ta ’ l- enzimi matul iż- żmien, bil- għan aħħari jkun li jittejbu r- riżultati kliniċi għal dawn il- pazjenti.
In a pivotal trial including 18 patients, the pharmacokinetics of Myozyme were evaluated in 15 patients with infantile-onset Pompe disease (all aged less than 6 months at treatment-onset) who received doses of 20 mg/ kg or 40 mg/ kg alglucosidase alfa as an approximate 4 to 6.5-hour infusion, respectively.
Fi prova importanti ħafna li kienet tinkludi 18- il pazjent, il- farmakokinetika ta ’ Myozyme kienet evalwata fi 15- il pazjent li fihom il- marda ta ’ Pompe bdiet fl- infanzja (kollha ta ’ età ta ’ inqas minn 6 xhur fil- bidu tal- kura) li rċivew dożi ta ’ 20 mg/ kg jew 40 mg/ kg ta ’ alglucosidase alfa bħala infużjoni ta ’ madwar 4 sa 6. 5 sigħat, rispettivament.
Pharmacokinetics were dose proportional and did not change over time.
Il- farmakokinetika kienet proporzjonali għad- doża u ma nbidlitx maż- żmien.
After the first and sixth infusion of Myozyme, mean maximum plasma concentrations (Cmax) ranged from 178.2 to 263.7 μ g/ ml for the 20 mg/ kg and 40 mg/ kg dose groups respectively.
Wara l- ewwel u s- sitt infużjoni ta ’ Myozyme, il- medja tal- konċentrazzjonijiet massimi fil- plażma (Cmax) varjat minn 178. 2 sa 263. 7 μg/ ml għall- gruppi li kienu qed jieħdu doża ta ’ 20 mg/ kg u 40 mg/ kg rispettivament.
The mean area under the plasma concentration-time curve (AUC∞) ranged from 977.5 to 1,872.5 μ g• hr/ mL for the 20 mg/ kg and 40 mg/ kg dose groups.
Il- medja taż- żona taħt il- konċentrazzjoni tal- plażma- kurva tal - ħin (AUC∞) varjat minn 977. 5 sa 1, 872. 5 μg• siegħa/ mL għall- gruppi li kienu qed jieħdu doża ta ’ 20 mg/ kg u 40 mg/ kg.
Mean plasma clearance (CL) was 21.9 mL/ hr/ kg and mean volume of distribution at steady state (Vss) was 66.2 mL/ kg for both dose groups with small between-subject variability of 15% and 11%, respectively.
Il- medja tat- tneħħija tal- plażma (CL) kienet ta ’ 21. 9 mL/ siegħa/ kg u l- medja tad - distribuzzjoni tal- volum fl- istat fiss (Vss) kienet ta ’ 66. 2 mL/ kg għaż- żewġ gruppi li kienu qed jieħdu
Mean plasma elimination half-life (t1/ 2) was 2.75 hours for the two dose groups.
Il- medja tal- half- life (t1/ 2) ta ’ l- eliminazzjoni tal- plażma kienet ta ’ 2. 75 sigħat għaż- żewġ gruppi li kienu qed jieħdu d- doża.
The pharmacokinetics of Myozyme were also evaluated in a separate trial in 21 patients with infantile- onset Pompe disease (all aged between 6 months and 3.5 years at treatment-onset) who received doses of 20 mg/ kg of alglucosidase alfa.
Il- farmakokinetika ta ’ Myozyme kienet evalwata wkoll fi prova separata f’ 21 pazjent li fihom il- marda ta ’ Pompe bdiet fl- infanzja (kollha bejn l- età ta ’ 6 xhur u 3. 5 snin fil- bidu tal- kura) li rċivew dożi ta ’ 20 mg/ kg ta ’ alglucosidase alfa.
In 12 patients with available data the AUC∞ and Cmax were approximately equivalent to those observed for the 20 mg/ kg dose group in the pivotal trial.
Fi 12- il pazjent li għalihom kien hemm informazzjoni disponibbli, l - AUC∞ u s- Cmax kienu ta ’ bejn wieħed u ieħor ekwivalenti għal dawk osservati għall- grupp li ngħata doża ta ’ 20 mg/ kg fil- prova importanti ħafna.
The t½ of approximately 2-3 hours was also similar in this group of patients.
It- t½ ta ’ madwar 2- 3 sigħat kienet simili wkoll f’ dan il - grupp ta ’ pazjenti.
The pharmacokinetics of Myozyme were evaluated in a trial in 5 patients with late-onset Pompe disease aged 6-15 years who received 20 mg/ kg alglucosidase alfa every 2 weeks.
Il- farmakokinetika ta ’ Myozyme kienet evalwata fi prova f’ 5 pazjenti li fihom il- marda ta ’ Pompe bdiet tard fil- ħajja u li kellhom minn 6 sa 15- il sena, li rċivew 20 mg/ kg ta ’ alglucosidase alfa kull ġimagħtejn.
There was no difference in the pharmacokinetic profile of Myozyme in late-onset patients compared to infantile- onset patients.
Ma kien hemm l- ebda differenza fil- profil farmakokinetiku ta ’ Myozyme f’ pazjenti li fihom il- marda bdiet tard fil- ħajja meta mqabbla ma ’ pazjenti li fihom il- marda bdiet fl- infanzja.
Preclinical data reveal no special hazards for humans based on studies of safety pharmacology, single and repeat dose toxicity.
L- informazzjoni ta ’ qabel l- użu kliniku ma żvelat l- ebda perikli speċjali għall- bnedmin, ibbażata fuq studji konvenzjonali dwar is- sigurtà farmakologika, it- tossiċità ta ’ doża waħidha jew ripetuta.
No significant adverse findings were observed in a mouse embryofoetal
L- ebda sejbiet avversi sinifikanti ma kienu osservati fi studju embrijufetali fuq il- ġrieden.
Data from a mouse fertility and early embryonic development study were not conclusive.
Informazzjoni minn studju dwar il- fertilità fil- ġrieden u l- iżvilupp bikri embrijoniku ma kinitx konklussiva.
The potential effects on reproductive and developmental functions have not been fully characterised.
L- effetti potenzjali fuq il- funzjonijiet riproduttivi u ta ’ waqt l- iżvilupp ma kinux ikkaratterizzati b’ mod sħiħ.
Mannitol Sodium phosphate monobasic monohydrate Sodium phosphate dibasic heptahydrate Polysorbate 80
Mannitol Sodium phosphate monobasic monohydrate Sodium phosphate dibasic heptahydrate Polysorbate 80
After dilution, an immediate use is recommended.
Wara d- dilwizzjoni, l- użu immedjat hu rakkomandat.
However, chemical and physical in-use stability has been demonstrated for 24 hours at 2 to 8°C when stored under protection from light.
Madankollu, l- istabbiltà kimika u fiżika waqt l - użu kienet murija għal 24 siegħa f’ temperatura ta ’ minn 2 sa 8°C meta jinħażen u jkun protett mid - dawl.
50 mg of powder in a vial (Clear Type 1 glass) with a stopper (siliconised butyl) and a seal (aluminium) with a flip-off cap (plastic).
50 mg ta ’ trab f’ kunjett (ħġieġ Ċar ta ’ Tip 1) b’ tapp (siliconised butyl) u siġill (ta ’ l- aluminju) b’ għatu flip- off (tal- plastik).
Pack sizes of 1, 10 or 25 vials.
Daqsijiet tal- pakkett ta ’ 1, 10 jew 6 kunjetti.
6.6 Special precautions for disposal and other handling
11 6. 6 Prekawzjonijiet speċjali li għandhom jittieħdu meta jintrema u għal immaniġġar ieħor
Myozyme has to be reconstituted with water for injections, then diluted with 9 mg/ ml (0.9%) sodium chloride solution and then administered by intravenous infusion.
Myozyme għandu jkun rikostitwit bl- ilma għall- injezzjonijiet, imbagħad dilwit b’ 9 mg/ ml (0. 9%) soluzzjoni ta ’ sodium chloride u mbagħad jingħata permezz ta ’ infużjoni ġol- vini.
Reconstitution and dilution should be performed in accordance with good practice rules, particularly for the respect of asepsis.
Ir- rikostituzzjoni u d- dilwizzjoni għandhom jitwettqu b’ konformità mar- regoli ta ’ prattika tajba, partikularment fir - rigward ta ’ asepsis.
Due to the proteinaceous nature of the product, particle formation may occur in the reconstituted solution and final infusion bags.
Minħabba n- natura proteinaceous tal- prodott, jista ’ jifforma frak fis- soluzzjoni rikostitwita u fil- boroż ta ’ l- infużjoni finali.
Therefore, a 0.2 micron low protein binding in-line filter should be used for administration.
Għalhekk, filtru in- line low protein binding ta ’ 0. 2 micron għandu jintuża għall - għoti.
It was demonstrated that the use of a 0.2 micron in-line filter removes visible particles and does not result in an apparent loss of protein or activity.
Intwera li l- użu ta ’ filtru in- line ta ’ 0. 2 micron ineħħi partikuli viżibbli u ma jirriżultax f’ telf apparenti ta ’ proteina jew ta ’ l- attività.
Determine the number of vials to be reconstituted based on the individual patient’ s dose regimen (mg/ kg) and remove the required vials from the refrigerator in order to allow them to reach room temperature (approximately 30 minutes).
Stabbilixxi n- numru ta ’ kunjetti li ser ikunu rikostitwiti, ibbażat fuq il- kors tad- doża tal- pazjent individwali (mg/ kg) u neħħi l- kunjetti meħtieġa mill- friġġ sabiex tħallihom jilħqu t- temperatura tal - kamra (madwar 30 minuta).
As Myozyme does not contain a preservative, each vial of Myozyme is for single use only.
Minħabba li Myozyme ma fih l- ebda preservattiv, kull kunjett ta ’ Myozyme hu għall- użu ta ’ darba biss.
11 Use Aseptic Technique
Uża t- Teknika Asettika
Reconstitute each 50 mg vial of Myozyme with 10.3 ml water for injections.
Irrikostitwixxi kull kunjett ta ’ 50 mg ta ’ Myozyme b’ 10. 3 ml ta ’ ilma għall- injezzjonijiet.
Add the water for injections by slow drop-wise addition down the side of the vial and not directly onto the lyophilized cake.
Żid l- ilma għall- injezzjonijiet permezz ta ’ żieda ta ’ taqtir bil- mod man- naħa tal- kunjett u mhux direttament fuq is- sustanza lajofilizzata.
Tilt and roll each vial gently.
Mejjel u irrombla kull kunjett bil- mod.
Do not invert, swirl or shake the vial.
Taqlibx ta ’ taħt fuq, iddawwarx u iċċaqlaqx il- kunjett.
The reconstituted volume is 10.5 ml containing 5 mg/ ml, and appears as a clear, colourless to pale yellow solution which may contain particles in the form of thin white strands or translucent fibres.
Il- volum rikostitwit hu ta ’ 10. 5 ml li fih 5 mg/ ml, u jidher bħala soluzzjoni ċara li tkun minn bla kulur sa isfar ċar, u li jista ’ jkun fiha frak fis- sura ta ’ ħjut rqaq bojod jew fibri transluċidi.
Perform an immediate inspection of the reconstituted vials for particulate matter and discoloration.
Eżamina immedjatament il- kunjetti rikostitwiti għal frak u tibdil fil- kulur.
If upon immediate inspection foreign particles other than those described above are observed, or if the solution is discoloured, do not use.
Jekk meta teżaminahom tosserva frak li ma jkunx bħal dak deskritt hawn fuq, jew jekk is- soluzzjoni tkun bidlet il- kulur, tużahx.
The pH of the reconstituted solution is approximately 6.2.
Il- pH tas- soluzzjoni rikostitwita hu ta ’ madwar 6. 2.
After reconstitution, it is recommended to promptly dilute the vials (see below).
Wara r- rikostituzzjoni hu rakkomandat li tiddilwa immedjatament il- kunjetti (ara hawn taħt).
Dilution
Dilwizzjoni
When reconstituted as above, the reconstituted solution in the vial contains 5 mg alglucosidase alfa per ml.
Meta tkun rikostitwita kif deskritt hawn fuq, is- soluzzjoni rikostitwita fil- kunjett ikun fiha 5 mg ta ’ alglucosidase alfa f’ kull ml.
The reconstituted volume allows accurate withdrawal of 10.0 ml (equal to 50 mg) from each vial.
Il- volum rikostitwit jippermetti ġbid preċiż ta ’ 10. 0 ml (li hu l- istess daqs 50 mg) minn kull kunjett.
This should then be further diluted as follows:
Dan, imbagħad, għandu jkun dilwit b’ mod addizzjonali kif ġej:
Slowly withdraw the reconstituted solution from each vial until the volume for the patient’ s dose is obtained.
Iġbed bil - mod is- soluzzjoni rikostitwita minn kull kunjett sakemm jinkiseb il- volum għad- doża tal- pazjent.
The recommended final concentration of alglucosidase in the infusion bags ranges from 0.5 mg/ ml to 4 mg/ ml.
Il - konċentrazzjoni finali rakkomandata ta ’ alglucosidase fil- boroż ta ’ l- infużjoni tvarja minn 0. 5 mg/ ml sa 4 mg/ ml.
Remove airspace within the infusion bag.
Neħħi l- arja mill- borża ta ’ l- infużjoni.
Also remove an equal volume of 0.9% sodium chloride, that will be replaced with reconstituted Myozyme.
Neħħi wkoll l- istess volum ta ’ 0. 9% sodium chloride, li ser ikun sostitwit b’ Myozyme rikostitwit.
Slowly inject the reconstituted Myozyme directly into the 0.9% sodium chloride solution.
Injetta bil- mod Myozyme rikostitwit direttament ġoż- 0. 9% soluzzjoni ta ’ sodium chloride.
Gently invert or massage the infusion bag to mix the diluted solution.
Aqleb ta ’ taħt fuq jew immassaġġja l- borża ta ’ l- infużjoni biex tħallat is- soluzzjoni dilwita.
Do not shake or excessively agitate the infusion bag.
Iċċaqlaqx jew tħarrek b’ mod eċċessiv il- borża ta ’ l- infużjoni.
The final infusion solution should be administered as close to preparation time as possible.
Is- soluzzjoni ta ’ l- infużjoni finali għandha tingħata qrib tal- ħin tal- preparazzjoni kemm jista jkun.
Genzyme Europe B. V., Gooimeer 10, NL-1411 DD Naarden, The Netherlands
Genzyme Europe B. V., Gooimeer 10, NL- 1411 DD Naarden, l- Olanda
29 March 2006
29 ta ’ Marzu, 2006
13 A.
13 ANNESS II
Name and address of the manufacturers of the biological active substance
KUNDIZZJONIJIET TA ’ L- AWTORIZZAZZJONI GĦAT - TQEGĦID FIS- SUQ
Genzyme Corp., 500 Soldiers Field Road, Allston, MA 02134, USA Genzyme Corp.
Genzyme Corp., 500 Soldiers Field Road, Allston, MA 02134, Stati Uniti ta ’ l- Amerika Genzyme Corp.
45, 51, 76 and 80 New York Avenue, Framingham, MA 01701, USA
45, 51, 76 and 80 New York Avenue, Framingham, MA 01701, Stati Uniti ta ’ l- Amerika
Genzyme Ltd., 37 Hollands Road, Haverhill, Suffolk CB9 8PU, United Kingdom
Genzyme Ltd., 37 Hollands Road, Haverhill, Suffolk CB9 8PU, Ir- Renju Unit
Pharmacovigilance system The MAH must ensure that the system of pharmacovigilance, as described in version 14 April 2008 presented in Module 1.8.1. of the Marketing Authorisation Application, is in place and functioning before and whilst the product is on the market.
Sistema ta 'farmakoviġilanza Id- Detentur ta' l- Awtorizzazzjoni tal- Manifattura jrid jiżgura li s- sistema ta ’ farmakoviġilanza, kif deskritta fil- verżjoni tal- 14 t’ April, 2008, ippreżentata f' Modulu 1. 8. 1. ta ’ l- Applikazzjoni ta 'l - Awtorizzazzjoni għat- Tqegħid fis- Suq, tkun implimentata u taħdem qabel u waqt li l- prodott jibda jinbiegħ.
Risk Management Plan The MAH commits to performing the studies and additional pharmacovigilance activities detailed in the Pharmacovigilance Plan, as agreed in version 1 of the Risk Management Plan (RMP) presented in Module 1.8.2. of the Marketing Authorisation Application and any subsequent updates of the RMP agreed by the CHMP.
Pjan dwar l- Immaniġġjar tar- Riskju Id- Detentur ta 'l- Awtorizzazzjoni tal- Manifattura jrid ikun obbligat li jwettaq l- istudji u attivitajiet addizzjonali ta' farmakoviġilanza deskritti fil- Pjan tal- Farmakoviġilanza, kif maqbul f' verżjoni 1 tal - Pjan dwar l- Immaniġġjar tar- Riskju (Risk Management Plan - RMP) ippreżentat f' Modulu 1. 8. 2. ta 'l - Applikazzjoni ta' l- Awtorizzazzjoni għat- Tqegħid fis- Suq, u kwalunkwe aġġornamenti sussegwenti tal- RMP li kienu maqbula mis- CHMP (Committee for Medicinal Products for Human Use).
In addition, an updated RMP should be submitted • When new information is received that may impact on the current Safety Specification, Pharmacovigilance Plan or risk minimisation activities • Within 60 days of an important (pharmacovigilance or risk minimisation) milestone being reached • At the request of the EMEA
Flimkien ma 'dan, RMP aġġornat għandu jkun sottomess • Meta informazzjoni ġdida tkun irċevuta li jista' jkollha impatt fuq l- Ispeċifikazzjoni tas - Sigurtà kurrenti, fuq il- Pjan tal- Farmakoviġilanza jew fuq attivitajiet li jimminimizzaw ir - riskju • Fi żmien 60 jum minn meta tintlaħaq milestone importanti (farmakoviġilanza jew minimizzazzjoni tar- riskju) • Fuq talba tal- EMEA
16 PARTICULARS TO APPEAR ON THE OUTER PACKAGING
17 TAGĦRIF LI GĦANDU JIDHER FUQ IL- PAKKETT TA ’ BARRA
Myozyme 50 mg powder for concentrate for solution for infusion Alglucosidase alfa
Myozyme 50 mg, trab għal konċentrat għal soluzzjoni għall- infużjoni Alglucosidase alfa
Each vial contains 50 mg of alglucosidase alfa
Kull kunjett fih 50 mg ta ’ alglucosidase alfa
1 vial of powder for concentrate for solution for infusion.
1 kunjett ta ’ trab għal konċentrat għal soluzzjoni għall- infużjoni.
25 vials of powder for concentrate for solution for infusion.
25 kunjett ta ’ trab għal konċentrat għal soluzzjoni għall- infużjoni.
Intravenous use Read the package leaflet before use.
Użu għal ġol- vini Aqra l- fuljett ta ’ tagħrif qabel l- użu.
For single use only
Jintuża darba biss
Store at 2°C-8°C (in a refrigerator)
Aħżen f’ temperatura ta ’ 2°C- 8°C (fi friġġ)
Any unused product should be discarded
Kwalunkwe prodott mhux użat għandu jintrema
{NATURE/ TYPE}
NATURA/ TIP
50 mg per vial
50 mg f’ kull kunjett
Store at 2°C-8°C (in a refrigerator) Genzyme Europe B. V. -NL
Aħżen f’ temperatura ta ’ 2°C- 8°C (fi friġġ) Genzyme Europe B. V. - NL
What Myozyme is and what it is used for 2.
X’ inhu Myozyme u għalxiex jintuża 2.
Before you use Myozyme 3.
Qabel ma tuża Myozyme 3.
How to use Myozyme 4.
Kif għandek tuża Myozyme 4.
How to store Myozyme 6.
Kif taħżen Myozyme 6.
WHAT MYOZYME IS AND WHAT IT IS USED FOR
X’ INHU MYOZYME U GĦALXIEX JINTUŻA
Myozyme is used to treat patients who have a confirmed diagnosis of Pompe disease.
Myozyme jintuża għall- kura pazjenti li jkollhom dijanjosi kkonfermata tal- marda ta ’ Pompe.
People with Pompe disease have low levels of an enzyme called α -glucosidase.
Nies bil- marda ta ’ Pompe għandhom livelli baxxi ta ’ enzima msejħa α- glucosidase.
This enzyme helps the body control levels of glycogen (a type of carbohydrate).
Din l- enzima tgħin lill- ġisem jikkontrolla l- livelli ta ’ glycogen (tip ta ’ carbohydrate).
Glycogen provides the body with energy, but in Pompe disease the levels can get too high.
Glycogen jipprovdi l- ġisem bl - enerġija, iżda f’ nies bil- marda ta ’ Pompe dawn il- livelli jistgħu jkunu żżejjed.
Myozyme is an artificial enzyme called alglucosidase alfa – this can replace the natural enzyme which is lacking in Pompe disease.
Myozyme hu enzima artifiċjali msejħa alglucosidase alfa – din tista ’ tissostitwixxi l- enzima naturali li tkun nieqsa fil- marda ta ’ Pompe.
The benefits of Myozyme in patients with late onset Pompe disease are not known yet.
Il- benefiċċji ta ’ Myozyme f’ pazjenti li fihom il- marda ta ’ Pompe ddum ma sseħħ, mhumiex magħrufa s’ issa.
BEFORE YOU USE MYOZYME
QABEL MA TUŻA MYOZYME
Do not use Myozyme If you are allergic (hypersensitive) to alglucosidase alfa or any of the other ingredients of Myozyme.
- jekk inti allerġiku/ a (għandek sensittività eċċessiva) għal alglucosidase alfa jew sustanzi oħra
Take special care with Myozyme If you are treated with Myozyme, you may experience a reaction while you are being given the medicine or during the next 2 hours.
Oqgħod attent ħafna b’ Myozyme Jekk inti tkun ikkurat/ a b’ Myozyme, jista ’ jkollok reazzjoni waqt li tkun qed tingħata l- mediċina jew matul is- sagħtejn ta ’ wara.
This is known as an infusion-associated reaction and can sometimes be very severe.
Din hi magħrufa bħala reazzjoni marbuta ma ’ l- infużjoni u xi kultant tista tkun severa ħafna.
If you experience a reaction like this, you should tell your doctor immediately.
Jekk ikollok reazzjoni bħal din, għandek tgħid lit- tabib tiegħek immedjatament.
You may need to be given additional medicines to prevent an allergic reaction (e. g. antihistamines and/ or corticosteroids) or antipyretics.
Jista ’ jkun hemm il- bżonn li tingħata mediċini addizzjonali biex tiġi evitata reazzjoni allerġika (eż. antistamini u/ jew kortikosterojdi) jew mediċini kontra d- deni.
Different groups of patients using Myozyme The information in this leaflet applies to all patient groups including children, adolescents, adults and the elderly.
Gruppi differenti ta ’ pazjenti li jużaw Myozyme L- informazzjoni f’ dan fuljett tapplika għall- grupp kollha ta ’ pazjenti li jinkludu tfal, adolexxenti, adulti u l- anzjani.
21 Please tell your doctor if you are taking or have recently taken any other medicines, including medicines obtained without a prescription.
22 Meta tuża mediċini oħra Jekk jogħġbok għid lit- tabib tiegħek jekk qiegħed tieħu jew ħadt dan l- aħħar xi mediċini oħra, anki dawk mingħajr riċetta.
Pregnancy and breast-feeding There is no experience of the use of Myozyme in pregnant women.
Tqala u treddigħ M’ hemm l- ebda esperjenza dwar l- użu ta ’ Myozyme f’ nisa tqal.
Myozyme should not be used during pregnancy unless clearly necessary.
Myozyme m’ għandux jintuża waqt it - tqala jekk m’ hemmx bżonn ċar.
Ask you doctor or pharmacist for advice before taking any medicine.
Itlob il- parir tat- tabib jew ta ’ l- ispiżjar tiegħek qabel tieħu xi mediċina.
HOW TO USE MYOZYME
KIF GHANDEK TUŻA MYOZYME
Instructions for proper use Myozyme is given through a drip into a vein (by intravenous infusion).
Struzzjonijiet biex tużah kif suppost Myozyme jingħata minn ġo dripp ġo vina (permezz ta ’ infużjoni ġol- vini).
It is supplied as a powder which will be mixed with sterile water before it is given.
Hu fornut bħala trab li ser jitħallat b’ ilma sterili qabel jingħata.
Myozyme is only used under the supervision of a doctor who is knowledgeable in the treatment of Pompe disease.
Myozyme jintuża biss taħt is- superviżjoni ta ’ tabib b’ esperjenza fil- kura tal- marda ta ’ Pompe.
The recommended dosage of Myozyme is 20 mg/ kg body weight given once every 2 weeks.
Id- dożaġġ rakkomandat ta ’ Myozyme hu ta ’ 20 mg/ kg ta ’ piż tal- ġisem mogħti darba kull ġimagħtejn.
If you use more Myozyme than you should There are no cases of overdose reported.
Jekk tuża Myozyme aktar milli suppost M’ hemm l- ebda każijiet ta ’ doża eċċessiva li kienu rrappurtati.
If you forget to use Myozyme If you have missed an infusion, please contact your doctor.
Jekk tinsa tuża Myozyme Jekk tkun insejt tieħu infużjoni, jekk jogħġbok ikkuntattja lit- tabib tiegħek.
Like all medicines, Myozyme can cause side effects, although not everybody gets them.
Bħal kull mediċina oħra, Myozyme jista ’ jkollu effetti sekondarji, għalkemm ma jidhrux fuq kulħadd.
Side effects were mainly seen while patients were being given the medicine or shortly after (“ infusion related effects”).
L- effetti sekondarji kienu osservati primarjament waqt li l- pazjenti kienu qed jingħataw il- mediċina jew ftit wara (“ effetti marbuta ma ’ l- infużjoni ”).
Some of these infusion related side effects became serious.
Xi effetti sekondarji marbuta ma ’ l- infużjoni kienu severi.
Should you experience any reaction like this, please tell your doctor immediately.
Jekk ikollok xi reazzjoni bħal din, jekk jogħġbok għid lit- tabib tiegħek immedjatament.
• Hives • Rash • Paleness • Redness of the skin • (Facial) flushing • Increased or high blood pressure • Bluish discolouration of the skin • Fever or increased body temperature • Chills • Cough • Increased breathing rate • Increased or decreased heart rate • Vomiting • Agitation • Tremor • Swelling around the eyes • Abnormal breathing sounds
• Urtikarja • Raxx • Sfura • Ħmura tal- ġilda • Fwawar (tal- wiċċ) • Żieda fil- pressjoni jew pressjoni għolja • Kulur tal- ġilda jagħti fil- blu • Deni jew żieda fit- temperatura tal- ġisem • Tertir • Sogħla • Żieda fir- rata tan- nifs • Żieda jew tnaqqis fir- rata ta ’ taħbit tal- qalb • Rimettar • Aġitazzjoni • Rogħda
22 • Difficulty in breathing • Headache • Cold extremities (e. g. hands, feet) • Tingling • Pain or local reaction at the site of the drip • Dizziness • Irritability • Itchy skin • Retching • Low blood pressure • Bronchospasm • Low level of oxygen in the blood • Swelling of the face, swelling of the throat or severe combined swelling of the face, throat and tongue due to a severe allergic reaction • Swelling of the arms and legs • Feeling hot • Increased sweating • Eyes tearing • Mottled skin • Nausea • Restlessness • Wheezing • Heart stopping
23 • Nefħa madwar l- għajnejn • Ħsejjes anormali meta tieħu n- nifs • Diffikultà biex tieħu n- nifs • Uġigħ ta ’ ras • Kesħa fl- estremitajiet (eż. idejn, saqajn) • Tnemnim • Uġigħ jew reazzjoni lokali fis- sit tad- dripp • Sturdament • Irritabilità • Ħakk fil- ġilda • Dardir • Pressjoni baxxa • Bronkospażmu • Livell baxx ta ’ ossiġnu fid- demm • Nefħa tal- wiċċ, nefħa tal- gerżuma jew nefħa severa kombinata tal- wiċċ, tal- gerżuma u ta ’ l - ilsien minħabba reazzjoni allerġika severa • Nefħa fid- dirgħajn u r- riġlejn • Tħoss is- sħana • Żieda fl- għaraq • Għajnejn idemmgħu • Ġilda bi tbajja ’ • Nawseja • Nuqqas ta ’ kwiet f’ ġismek • Tħarħir • Il- qalb tieqaf
Life threatening reactions, including very severe generalised allergic reactions and anaphylactic shock, have been reported in some patients.
Reazzjonijiet ta ’ periklu għall- ħajja, li jinkludu reazzjonijiet allerġiċi ġeneralizzati severi ħafna u xokk anafilattiku, kienu rrappurtati f’ xi pazjenti.
HOW TO STORE MYOZYME
KIF TAĦŻEN MYOZYME
Store in a refrigerator (2°C – 8°C).
Aħżen fi frigg (2°C – 8°C).
Do not use after the expiry date stated on the labelling after the letters ‘ EXP’.
Tużax wara d- data ta ’ skadenza li tidher fuq it- tikketta wara ‘ JIS ’.
It is recommended that Myozyme is used immediately after it has been mixed with sterile water.
Hu rakkomandat li Myozyme jintuża immedjatament wara li jkun tħallat bl- ilma sterili.
However it can be kept for up to 24 hours if it is kept cool (2°C – 8°C) and in the dark.
Madankollu, jista ’ jinżamm sa 24 siegħa jekk it jinżamm f’ temperatura kiesħa (2°C – 8°C) u fid- dlam.
What Myozyme contains
X’ fih Myozyme
- The active substance is alglucosidase alfa 50 mg.
- Is- sustanza attiva hi alglucosidase alfa 50 mg.
After reconstitution, the solution contains 5 mg of alglucosidase alfa/ ml and after dilution, the concentration varies from 0.5 mg to 4 mg/ ml.
Wara r- rikostituzzjoni, is- soluzzjoni jkun fiha 5 mg ta ’ alglucosidase alfa/ ml u wara d- dilwizzjoni, il- konċentrazzjoni tvarja minn 0. 5 mg sa 4 mg/ ml.
- mannitol,
- mannitol,
- sodium phosphate monobasic monohydrate
- sodium phosphate monobasic monohydrate
- sodium phosphate dibasic heptahydrate
- sodium phosphate dibasic heptahydrate
- polysorbate 80
- polysorbate 80
What Myozyme looks like and contents of the pack Myozyme, 50 mg, is presented as a powder for concentrate for solution for infusion (in a vial- pack: sizes of 1, 10 or 25).
Id- dehra tal Myozyme u l- kontenuti tal- pakkett Myozyme, 50 mg, hu ppreżentat bħala trab għal konċentrat għal soluzzjoni għall- infużjoni (f’ kunjett - pakkett: daqsijiet ta ’ 1, 10 jew 25).
Myozyme is supplied as a white to off-white powder.
Myozyme hu fornut bħala trab ta ’ kulur minn abjad sa abjad jagħti fil- griż.
After reconstitution it is a clear, colourless to pale yellow solution, which may contain particles.
Wara r- rikostituzzjoni, isir soluzzjoni ċara, bla kulur sa isfar ċar, li jista ’ jkun fiha l- frak.
The reconstituted solution must be further diluted.
Is- soluzzjoni rikostitwita trid tkun dilwita b’ mod addizzjonali.
Marketing Authorisation Holder Genzyme Europe B. V., Gooimeer 10, 1411 DD, Naarden, The Netherlands
Id- Detentur ta ’ l- Awtorizzazzjoni għat- Tqegħid fis- Suq Genzyme Europe B. V., Gooimeer 10, 1411 DD, Naarden, l- Olanda
Manufacturer Genzyme Ltd., 37 Hollands Road, Haverhill, Suffolk CB9 8PU, United Kingdom
Manifattur Genzyme Ltd., 37 Hollands Road, Haverhill, Suffolk CB9 8PU, Ir- Renju Unit
Detailed information on this medicine is available on the European Medicines Agency (EMEA) web site: http: / /www. emea. europa. eu/.
Informazzjoni dettaljata dwar din il- mediċina tinsab fuq il- website ta ’ l- Aġenzija Ewropeja dwar il - Mediċini (EMEA): http: // www. emea. europa. eu /.
There are also links to other websites about rare diseases and treatments.
Hemm ukoll links għall- websites oħrajn dwar mard rari u kura.
< ------------------------------------------------------------------------------------------------------------------------- The following information is intended for medical or healthcare professionals only:
< -------------------------------------------------------------------------------------------------------------------------- --- It- tagħrif li jmiss qed jingħata biss għall- professjonisti fil- qasam mediku:
Instructions for use – reconstitution, dilution and administration
Struzzjonijiet għall- użu – rikostituzzjoni, dilwizzjoni u għoti
It was demonstrated that the use of a 0.2 micron in-line filter removes visible particles and does not result in an apparent loss of protein or activity.
Kien muri li l- użu ta ’ filtru in- line ta ’ 0. 2 micron ineħħi partikuli viżibbli u ma jirriżultax f’ telf apparenti ta ’ proteina jew attività.
As Myozyme does not contain a preservative, each vial of Myozyme is for single use only.
Minħabba li Myozyme ma fihx preservattiv, kull kunjett ta ’ Myozyme hu għall- użu ta ’ darba biss.
Use Aseptic Technique
Uża t- Teknika Asettika
• Reconstitution Reconstitute each 50 mg vial of Myozyme with 10.3 ml water for injections.
• Rikostituzzjoni Irrikostitwixxi kull kunjett ta ’ 50 mg ta ’ Myozyme b’ 10. 3 ml ta ’ ilma għall- injezzjonijiet.
The reconstituted volume is 10.5 ml containing 5 mg enzyme/ ml, and appears as a clear, colourless to pale yellow solution which may contain particles in the form of thin white strands or translucent fibres.
Il- volum rikostitwit hu ta ’ 10. 5 ml li fih 5 mg/ ml, u jidher bħala soluzzjoni ċara li tkun minn bla kulur sa isfar ċar, u li jista ’ jkun fiha frak fis- sura ta’ ħjut rqaq bojod jew fibri
After reconstitution it is recommended to promptly dilute the vials (see below).
Wara r- rikostituzzjoni hu rakkomandat li tiddilwa immedjatament il- kunjetti (ara hawn taħt).
• Dilution When reconstituted as above, the reconstituted solution in the vial contains 5 mg alglucosidase alfa per ml.
• Dilwizzjoni Meta tkun rikostitwita kif deskritt hawn fuq, is- soluzzjoni rikostitwita fil- kunjett ikun fiha 5 mg ta ’ alglucosidase alfa f’ kull ml.
The reconstituted volume allows accurate withdrawal of 10.0 ml (equal to 50 mg) from each vial.
Il- volum rikostitwit jippermetti ġbid preċiż ta ’ 10. 0 ml (li huma l- istess daqs 50 mg) minn kull kunjett.
Slowly withdraw the reconstituted solution from each vial until the volume for the patient’ s dose is obtained.
Iġbed bil- mod is- soluzzjoni rikostitwita minn kull kunjett sakemm jinkiseb il- volum għad- doża tal- pazjent.
The recommended final concentration of alglucosidase in the infusion bags ranges from 0.5 mg/ ml to 4 mg/ ml.
Il- konċentrazzjoni finali rakkomandata ta ’ alglucosidase fil- boroż ta ’ l- infużjoni tvarja minn 0. 5 mg/ ml sa 4 mg/ ml.
Also remove an equal volume of 0.9% sodium chloride, that will be replaced with
Neħħi wkoll l- istess volum ta ’ 0. 9% sodium chloride, li ser ikun sostitwit b’ Myozyme rikostitwit.
• Administration It is recommended to start the administration of the diluted solution within three hours.
• Kif Jingħata Hu rakkomandat li tibda l- għoti tas- soluzzjoni dilwita fi żmien tliet sigħat.
The total time between reconstitution and completion of the infusion should not exceed 24 hours.
Il- ħin totali bejn ir - rikostituzzjoni u t- tlestija ta ’ l- infużjoni m’ għandux jaqbeż l- 24 siegħa.
The recommended dosage regimen of Myozyme is 20 mg/ kg of body weight administered once every 2 weeks as an intravenous infusion.
Il- kors tad- dożaġġ rakkomandat ta ’ Myozyme hu ta ’ 20 mg/ kg ta ’ piż tal- ġisem, mogħti darba kull ġimagħtejn bħala infużjoni ġol- vini.
It is recommended that the infusion begin at an initial rate of 1 mg/ kg/ hr and be gradually increased by 2 mg/ kg/ hr every 30 minutes if there are no signs of IARs (Infusion Associated Reactions) until a maximum rate of 7 mg/ kg/ hr is reached.
Hu rakkomandat li l- infużjoni tinbeda b’ rata inizjali ta ’ 1 mg/ kg/ siegħa u tiżdied gradwalment b’ 2 mg/ kg/ siegħa kull 30 minuta jekk ma jkun hemm l- ebda sinjali ta ’ reazzjonijiet assoċjati ma ’ l- infużjoni (IARs) sakemm tintlaħaq rata massima ta ’ 7 mg/ kg/ siegħa.
What is Naglazyme?
X’ inhu Naglazyme?
Naglazyme is a solution for infusion (drip into a vein).
Naglazyme huwa soluzzjoni għall- infużjoni (dripp fil- vina).
It contains the active substance galsulfase.
Fih is- sustanza attiva galsulfase.
What is Naglazyme used for?
Għal xiex jintuża Naglazyme?
Naglazyme is used to treat patients who have mucopolysaccharidosis VI (MPS VI or Maroteaux-Lamy syndrome).
Naglazyme jintuża fit- trattament ta ’ pazjenti li għandhom mukopolisakkaridożi VI (MPS VI jew is - sindromu ta ’ Maroteaux- Lamy).
This disease is caused by the lack of an enzyme called arylsulfatase B, which is needed to break down substances in the body called glycosaminoglycans (GAGs).
Din il- marda hija kkawżata min- nuqqas tal- enzima arylsulfatase B, li hija meħtieġa għat- tkissir tas- sustanzi fil- ġisem magħrufa bħala glikosaminoglikani (GAGs).
If the enzyme is not present, GAGs cannot be broken down and they build up in the cells.
Jekk l - enzima ma tkunx preżenti, il- GAGs ma jkunux jistgħu jitkissru u jkomplu jakkumulaw fiċ- ċelluli.
This causes the signs of the disease, the most noticeable being a short body, a large head and difficulty moving about.
Dan jikkawża s- sinjali tal- marda, li l- aktar evidenti huma ġisem qasir, ras kbira, u diffikultà fil- mobilità.
The disease is usually diagnosed in infants between one and five years of age.
Normalment, issir dijanjożi tal- marda fi tfal ta ’ bejn sena u ħames snin.
Because the number of patients with MPS VI is low, the disease is considered ‘ rare’, and Naglazyme was designated an ‘ orphan medicine ’ (a medicine used in rare diseases) on 14 February 2001.
Peress li n- numru ta ’ pazjenti li jbatu mill- MPS VI huwa wieħed baxx, il- marda hi kkunsidrata bħala ‘ rari ’, u fl- 14 ta’ Frar 2001, Naglazyme ġie kklassifikat bħala ‘ mediċina orfni ’ (mediċina użata f’ mard rari).
How is Naglazyme used?
Kif jintuża Naglazyme?
Naglazyme treatment should be supervised by a doctor who has experience in the management of patients with MPS VI or similar diseases.
Trattament b’ Naglazyme għandu jkun issorveljat minn tabib b’ esperjenza fil- kontroll ta ’ pazjenti b’ MPS VI jew mard simili.
It should be given where resuscitation equipment is available in case of a medical emergency.
Għandu jingħata fejn ikun hemm tagħmir ta ’ risuxxitazzjoni disponibbli f’ każ ta ’ emerġenza medika.
Naglazyme is given as a four-hour infusion once a week.
Naglazyme jingħata bħala infużjoni ta ’ erba ’ sigħat darba fil- ġimgħa.
Before each infusion, patients should be given an antihistamine to reduce the risk of an allergic reaction.
Qabel kull infużjoni, il- pazjenti għandhom jingħataw antistamina biex jitnaqqas ir- riskju ta ’ reazzjoni allerġika.
Patients may also be given a medicine to prevent fever.
Il- pazjenti jistgħu jingħataw ukoll mediċina għall- prevenzjoni tad- deni.
Although patients under five years of age were not included in the main study of Naglazyme, it is important that they are treated if they have a severe form of MPS VI.
Minkejja li pazjenti taħt il- ħames snin ma kinux inklużi fl- istudju ewlieni ta ’ Naglazyme, huwa importanti li jiġu ttrattati jekk ikollhom forma gravi ta ’ MPS VI.