natgillin/translations-raw huggingface dataset:
In nine clinical trials in different indications 206 patients were treated with Zavesca at dosages of 50- 200 mg t. i. d. for an average duration of 2.2 years.
Povprečno zdravljenje je trajalo 2, 2 leti.
Of these patients, 90 had type 1 Gaucher disease, and 40 had Niemann-Pick type C disease.
Med temi bolniki jih je imelo 90 Gaucherjevo bolezen tipa 1 in 40 Niemann- Pickovo bolezen tipa C.
Adverse reactions were generally of mild to moderate severity and occurred with similar frequency across indications and dosages tested.
Neželeni učinki so bili v glavnem blagi do zmerni in so se pojavljali s podobno pogostnostjo za vse preskušane indikacije in odmerke.
The most common adverse reactions were gastrointestinal, with diarrhoea and other abdominal complaints, and weight loss.
Najpogostejši so bili neželeni učinki v prebavilih (diareja in druge trebušne težave) ter izguba telesne mase.
Adverse drug reactions, defined as treatment-emergent adverse events reported as related to treatment by the investigator and occurring in > 1% of patients, are listed in the table below by body system and frequency (very common: ≥ 1/ 10, common: ≥ 1/ 100 < 1/ 10).
Neželeni učinki zdravila, ki so se pojavili zaradi zdravljenja, o katerih so poročali raziskovalci, da so povezani z zdravljenjem in so se pojavili pri > 1% bolnikov, so navedeni v spodnji preglednici, razvrščeni po organskem sistemu in pogostnosti (zelo pogost: ≥ 1/ 10, pogost: ≥ 1/ 100 < 1/ 10).
Blood and lymphatic system disorders Common
Bolezni krvi in limfatičnega sistema Pogosto
Metabolism and Nutrition Disorders Very common Common
Presnovne in prehranske motnje Zelo pogosto Pogosto
Weight loss Anorexia, decreased appetite
izguba telesne mase anoreksija, zmanjšan apetit
Insomnia, libido decreased
nespečnost, zmanjšan libido
Nervous System Disorders Very common Common
Bolezni živčevja Zelo pogosto Pogosto Bolezni prebavil
Tremor, Peripheral neuropathy, headache, paraesthesia, dizziness, ataxia, hypoaesthesia
tremor periferna nevropatija, glavobol, parestezije, omotičnost, ataksija, hipoestezija
Gastrointestinal Disorders Very common Common
Zelo pogosto Pogosto
Diarrhoea, flatulence, abdominal pain Nausea, vomiting, abdominal distension/ discomfort, constipation, dyspepsia
diareja, flatulenca, bolečine v trebuhu navzea, bruhanje, trebušna distenzija/ nelagodje v trebuhu, zaprtje, dispepsija
Musculoskeletal and connective tissue disorders Common
Bolezni mišično- skeletnega sistema in vezivnega tkiva Pogosto
Investigations
Splošne težave in spremembe
Fatigue, asthenia
na mestu aplikacije
5
5 Pogosto
Nerve conduction studies abnormal,
nenormalne študije prevodnosti živcev
Weight loss has been observed in approximately 60% of patients.
Izgubo telesne mase so opazili pri približno 60% bolnikov.
The greatest effect was at 12 months, with a mean weight loss of 6– 7% of body weight, with a subsequent tendency for an increase in weight towards the baseline value.
Največji učinek je bil pri 12 mesecih s povprečno izgubo 6– 7% telesne mase s kasnejšo nagnjenostjo k povečanju telesne mase proti izhodiščni vrednosti.
Zavesca has been studied in indications where certain events reported as adverse drug reactions, such as neurological symptoms/ signs and thrombocytopenia could also be due to the underlying conditions.
Zdravilo Zavesca so preučevali v zvezi z indikacijami, pri katerih bi bili lahko nekateri neželeni učinki, kot so nevrološki simptomi/ znaki in trombocitopenija tudi posledica bolezni, zaradi katere se bolnik zdravi.
Isolated cases of cognitive dysfunction have been reported during clinical trials of Zavesca in type 1 Gaucher disease.
Med kliničnim preskušanjem zdravila Zavesca so poročali o posamičnih primerih kognitivne disfunkcije pri bolnikih z Gaucherjevo boleznijo tipa 1.
A causal relationship to Zavesca has not been established.
Vzročne zveze z zdravilom Zavesca niso ugotovili.
Zavesca has been administered at doses of up to 3000 mg/ day for up to six months in HIV positive patients during clinical trials.
Zdravilo Zavesca so v kliničnih preskušanjih uporabljali v odmerkih do 3000 mg/ dan do šest mesecev pri bolnikih s HIV.
Leukopenia and neutropenia have also been observed in a similar group of patients receiving 800 mg/ day or higher dose.
Levkopenijo in nevtropenijo so opazili pri podobni skupini bolnikov, ki so jemali 800 mg/ dan ali več.
1 Pharmacodynamic properties
5. 1 Farmakodinamične lastnosti
Other alimentary tract and metabolism products, ATC Code:
Oznaka ATC:
A16AX06
A16AX06.
The European Medicines Agency (EMEA) will review any new information, which may become available every year and this SPC will be updated as necessary.
Evropska agencija za zdravila (EMEA) bo vsako leto ponovno pregledala vse nove podatke, ki bodo na voljo in posodobila povzetek glavnih značilnosti zdravila, če bo potrebno.
Miglustat is an inhibitor of glucosylceramide synthase, the enzyme responsible for the first step in the synthesis of most glycolipids.
Miglustat je zaviralec glukozilceramid- sintaze, ki je odgovoren za prvi korak v sintezi večine glikolipidov.
In vitro, glucosylceramide synthase is inhibited by miglustat with an IC50 of 20-37 µM.
Glukozilceramid- sintazo in vitro zavira miglustat z IC50, ki je 20- 37 µM.
In addition, inhibitory action on a non-lysosomal glycosylceramidase has been demonstrated experimentally in vitro The inhibitory action on glucosylceramide synthase forms the rationale for substrate reduction therapy in Gaucher disease.
Zaviralno delovanje na glukozilceramid- sintazo je podlaga za zdravljenje z zmanjšanjem substrata pri Gaucherjevi bolezni.
Twenty-eight patients with mild to moderate type 1 Gaucher disease were enrolled in this 12-month non-comparative study, and 22 patients completed the study.
V 12- mesečno neprimerjalno študijo so vključili 28 bolnikov z blago do zmerno obliko Gaucherjeve bolezni tipa 1, študijo je zaključilo 22 bolnikov.
At 12 months, there was a mean reduction in liver organ volume of 12.1% and a mean reduction in spleen volume of 19.0%.
Po 12 mesecih je bilo povprečno zmanjšanje volumna jeter 12, 1% in povprečno zmanjšanje volumna vranice 19, 0%.
A mean increase in haemoglobin concentration of 0.26 g/ dl and a mean platelet count increase of 8.29 × 109/ l were observed.
Opažali so povprečno povišanje koncentracije hemoglobina za 0, 26 g/ dl ter povprečno povečanje koncentracije trombocitov za 8, 29 × 109/ l.
Clinical benefit has been assessed at 24 and 36 months in 13 patients.
Po 24 in 36 mesecih so pri 13 bolnikih ocenili
After 3 years of continuous Zavesca treatment, mean reductions in liver and spleen organ volume were 17.5% and 29.6%, respectively.
Po treh letih stalnega jemanja zdravila Zavesca je bilo povprečno zmanjšanje volumna jeter 17, 5% in vranice 29, 6%.
There was a mean increase of 22.2 × 109/ l in platelet count, and a mean increase of 0.95 g/ dl in haemoglobin concentration.
Povprečno povečanje koncentracije trombocitov je bilo 22, 2 × 109/ l, povprečno povišanje koncentracije hemoglobina pa 0, 95 g/ dl.
6 A second open, controlled study randomised 36 patients who had received a minimum of 2 years of treatment with ERT, into three treatment groups: continuation with Cerezyme, Cerezyme in combination with Zavesca, or switch to Zavesca.
V drugi odprti kontrolirani študiji so v tri skupine zdravljenja naključno razporedili 36 bolnikov, ki so se že vsaj 2 leti zdravili z ERT: nadaljevanje zdravljenja z zdravilom Cerezyme, kombinacija zdravil Cerezyme in Zavesca ali prehod na zdravilo Zavesca.
This study was conducted over a 6-month randomised comparison period followed by 18 months extension where all patients received Zavesca monotherapy.
To študijo so izpeljali prek 6- mesečnega randomiziranega primerjalnega obdobja, ki mu je sledilo 18- mesečno podaljšanje, v katerem so vsi bolniki prejemali monoterapijo zdravila Zavesca.
When compared to the measurements at 6 months, disease control was unchanged after 18 and 24 months of Zavesca monotherapy (20 and 6 patients, respectively).
6 bolnikov).
Bone manifestations of type 1 Gaucher disease were evaluated in 3 open-label clinical studies in patients treated with miglustat 100 mg t. i. d. for up to 2 years (n = 72).
Manifestacije Gaucherjeve bolezni vrste 1 na kosteh so bile ocenjene v 3 odprtih kliničnih študijah pri bolnikih, zdravljenih z miglustatom 100 mg trikrat dnevno do 2 leti (n = 72).
In a pooled analysis of uncontrolled data, bone mineral density Z-scores at the lumbar spine and femoral neck increased by more than 0.1 units from baseline in 27 (57%) and 28 (65%) of the patients with longitudinal bone density measurements.
V zbrani analizi nekontroliranih podatkov so se vrednosti Z mineralne gostote kosti ledvene hrbtenice in vratu stegnenice povečale za več kot 0, 1 enoto od izhodišča pri 27 (57%) in 28 (65%) bolnikov pri vzdolžni smeri merjenja gostote kosti.
Niemann-Pick type C disease is a very rare, invariably progressive and eventually fatal neurodegenerative disorder characterised by impaired intracellular lipid trafficking.
Niemann- Pickova bolezen tipa C je zelo redka, vedno progresivna in končno smrtna nevrodegenerativna bolezen, za katero je značilno zmanjšano intracelično prehajanje maščob.
Data to support safety and efficacy of Zavesca in Niemann-Pick type C disease come from a prospective open-label clinical trial and a retrospective survey.
Podatki, ki podpirajo varnost in učinkovitost zdravila Zavesca pri Niemann- Pickovi bolezni tipa C so bili zbrani v prospektivnem odprtem kliničnem preskušanju in v retrospektivni analizi.
The clinical trial included 29 adult and juvenile patients in a 12-month controlled period, followed by extension therapy for an average total duration of 3.9 years and up to 5.6 years.
V klinično preskušanje je bilo vključenih 29 odraslih bolnikov in mladostnikov v 12- mesečnem kontroliranem obdobju, ki mu je sledil podaljšek zdravljenja s povprečnim skupnim trajanjem 3, 9 let in do 5, 6 let.
In addition 12 paediatric patients were enrolled in an uncontrolled substudy for an overall average duration of 3.1 years and up to 4.4 years.
V nekontrolirano podštudijo je bilo za celokupno povprečno obdobje 3, 1 leta in do 4, 4 let vključenih 12 pediatričnih bolnikov.
The survey included a case series of 66 patients treated with Zavesca outside of the clinical trial for a mean duration of 1.5 years.
Študija je vključevala klinično serijo 66 bolnikov, zdravljenih z zdravilom Zavesca, izven kliničnega preskušanja s povprečnim trajanjem 1, 5 let.
The usual dose of Zavesca in adult patients was 200 mg t. i. d., and was adjusted according to body surface area in paediatric patients.
Običajni odmerek zdravila Zavesca pri odraslih bolnikih je bil 200 mg trikrat dnevno, ustrezno prilagojen na površino telesa pri pediatričnih bolnikih.
Overall the data show that treatment with Zavesca can reduce the progression of clinically relevant neurological symptoms in patients with Niemann-Pick type C disease.
Na splošno so podatki pokazali, da lahko zdravljenje z zdravilom Zavesca zmanjša napredovanje klinično značilnih nevroloških simptomov pri bolnikih z Niemann- Pickovo boleznijo tipa C.
The benefit of treatment with Zavesca for neurological manifestations in patients with Niemann-Pick type C disease should be evaluated on a regular basis, e. g. every 6 months; continuation of therapy should be re-appraised after at least 1 year of treatment with Zavesca, (see section 4.4).
Koristi zdravljenja z zdravilom Zavesca pri nevroloških manifestacijah pri bolnikih z Niemann - Pickovo boleznijo tipa C je treba redno preverjati, npr. vsakih 6 mesecev; za nadaljevanje terapije se je treba ponovno odločiti po vsaj 1- letnem zdravljenju z zdravilom Zavesca (glejte poglavje 4. 4).
Pharmacokinetic parameters of miglustat were assessed in healthy subjects, in a small number of patients with type 1 Gaucher disease, Fabry disease, HIV-infected patients, and in adults, adolescents and children with Niemann-Pick type C disease or type 3 Gaucher disease.
7 Farmakokinetične parametre miglustata so ovrednotili pri zdravih osebah, pri majhnem številu bolnikov z Gaucherjevo boleznijo tipa 1, pri bolnikih s Fabryjevo boleznijo, pri bolnikih, okuženih z virusom HIV in pri odraslih, mladoletnikih in otrocih z Niemann- Pickovo boleznijo tipa C ali Gaucherjevo boleznijo tipa 3.
7 absorption (Cmax was decreased by 36% and tmax delayed 2 hours), but has no statistically significant effect on the extent of absorption of miglustat (AUC decreased by 14%).
Sočasno jemanje s hrano zniža raven absorpcije (Cmax se je znižal za 36% in tmax podaljšal za 2 uri), vendar nima statistično značilnega učinka na absorpcijo miglustata (AUC se je znižala za 14%).
Miglustat is mainly eliminated by renal excretion, with urinary recovery of unchanged drug accounting for 70-80% of the dose.
Miglustat se pretežno izloča skozi ledvice, pri čemer znaša rekuperacija nespremenjenega zdravila v urinu 70- 80% odmerka.
Apparent oral clearance (CL/ F) is 230 ± 39 ml/ min.
Navidezni peroralni očistek (CL/ F) je 230 ± 39 ml/ min.
The average half-life is 6– 7 hours.
Povprečni razpolovni čas je 6– 7 ur.
Following administration of a single dose of 100 mg 14C-miglustat to healthy volunteers, 83% of the radioactivity was recovered in urine and 12% in faeces.
Po uporabi enega odmerka 100 mg 14C- miglustata pri zdravih prostovoljcih je bilo 83% radioaktivno označenega zdravila izločenega z urinom in 12% z blatom.
The most abundant metabolite in urine was miglustat glucuronide accounting for 5% of the dose.
V urinu je bilo največ presnovka miglustat glukuronida, ki je predstavljal 5% odmerka.
The pharmacokinetics of miglustat is similar in adult type 1 Gaucher disease patients and Niemann-Pick type C disease patients when compared to healthy subjects.
Farmakokinetika miglustata je podobna pri odraslnih bolnikih z Gaucherjevo boleznijo tipa 1 in bolenikih z Niemann- Pickovo boleznijo tipa C, primerjano pri odraslih osebah.
Pharmacokinetic data were obtained in paediatric patients with type 3 Gaucher disease aged 3 to 15 years, and patients with Niemann-Pick type C disease aged 5– 16 years.
Farmakokinetični podatki so bili pridobljeni pri pediatričnih bolnikih z Gaucherjevo boleznijo tipa 3, starih 3 do 15 let, in pri bolnikih z Niemann- Pickovo boleznijo tipa C, starih 5– 16 let.
Dosing in children at 200 mg t. i. d. adjusted for body surface area resulted in Cmax and AUCτ values which were approximately two-fold those attained after 100 mg t. i. d. in type 1 Gaucher disease patients, consistent with the dose-linear pharmacokinetics of miglustat.
Odmerjanje 200 mg trikrat dnevno pri otrocih, prilagojeno na površino telesa, je povzročilo vrednosti Cmax in AUCτ, ki so bile približno dvakratne od tistih, pridobljenih po odmerku 100 mg trikrat dnevno pri bolnikih z Gaucherjevo boleznijo tipa 1, kar je skladno z farmakokinetiko miglustata, linearno z odmerkom.
At steady state, the concentration of miglustat in cerebrospinal fluid of six type 3 Gaucher disease patients was 31.4– 67.2% of that in plasma.
V stanju dinamičnega ravnovesja je koncentracija miglustata v cerebrospinalni tekočini šestih bolnikov z Gaucherjevo bolezijo tipa 3 predstavljala 31, 4– 67, 2% koncentracije v plazmi.
Limited data in patients with Fabry disease and impaired renal function showed that CL/ F decreases with decreasing renal function.
Malo podatkov za bolnike s Fabryjevo boleznijo in okvarjenim delovanjem ledvic je pokazalo, da se CL/ F s slabšanjem ledvičnega delovanja zmanjšuje.
While the numbers of subjects with mild and moderate renal impairment were very small, the data suggest an approximate decrease in CL/ F of 40% and 60% respectively, in mild and moderate renal impairment (see section 4.2).
Medtem ko je bilo število bolnikov z blago in zmerno okvarjenim delovanjem ledvic zelo majhno, podatki kažejo na zmanjšanje CL/ F za približno 40% pri blago in 60% pri zmerno okvarjenem delovanju ledvic (glejte poglavje 4. 2).
Data in severe renal impairment are limited to two patients with creatinine clearance in the range 18 – 29 ml/ min and cannot be extrapolated below this range.
Podatki za hudo okvarjeno delovanje ledvic so omejeni na dva bolnika, ki sta imela očistek kreatinina v območju 18 – 29 ml/ min in podatkov ne moremo ekstrapolirati pod to območje.
These data suggest a decrease in CL/ F by at least 70% in patients with severe renal impairment.
Ti podatki kažejo na zmanjšanje CL/ F za vsaj 70% pri bolnikih s hudo okvarjenim delovanjem ledvic.
Further effects seen in animals at doses that result in exposure levels similar to or moderately higher than the clinical exposure level were: changes in lymphoid organs in all species tested, transaminase changes, vacuolation of thyroid and pancreas, cataracts, nephropathy and myocardial changes in rats.
Poleg tega so tu še učinki, zabeleženi pri živalih pri odmerkih, ki povzročijo ravni izpostavljenosti, ki so podobne ali malo višje kot klinične ravni izpostavljenosti: spremembe v limfoidnih organih pri vseh testiranih vrstah, spremembe vrednosti transaminaz,
These findings were considered to be secondary to debilitation.
8 vakuolizacija ščitnice in trebušne slinavke, katarakte, nefropatija ter spremembe miokarda pri podganah.
Administration of miglustat to male and female Sprague-Dawley rats by oral gavage for 2 years at dose levels of 30, 60 and 180 mg/ kg/ day resulted in an increased incidence of testicular interstitial cell (Leydig cell) hyperplasia and adenomas in male rats at all dose levels.
Dajanje miglustata samcem in samicam podgan Sprague- Dawley po sondi 2 leti v odmerkih 30, 60 in 180 mg/ kg/ dan je povzročilo povišano incidenco hiperplazije testikularnih intersticijskih (Leydigovih) celic in adenomov pri samcih pri vseh odmerkih.
The systemic exposure at the lowest dose was below or comparable to that observed in humans (based on AUC0-∞) at the recommended human dose.
Sistemska izpostavljenost pri najnižjem odmerku je bila pod tisto, ki so jo opazili pri ljudeh (na podlagi AUC0- ∞) ob priporočenem odmerjanju, ali primerljiva z njo.
8 Administration of miglustat to male and female CD1 mice by oral gavage at dose levels of 210, 420 and 840/ 500 mg/ kg/ day (dose reduction after half a year) for 2 years resulted in an increased incidence of inflammatory and hyperplastic lesions in the large intestine in both sexes.
Dajanje miglustata samcem in samicam miši CD1 po sondi 2 leti v odmerkih 210, 420 in 840/ 500 mg/ kg/ dan (zmanjšanje odmerka na pol leta) je povzročilo povišano incidenco vnetnih in in hiperplastičnih lezij v debelem črevesu pri obeh spolih..
Based on mg/ kg/ day and corrected for differences in faecal excretion, the doses corresponded to 8, 16 and 33/ 19 times the highest recommended human dose (200 mg t. i. d.).
Odmerki, ki temeljijo na mg/ kg/ dan in korekciji zaradi razlik pri izločanju fecesa, ustrezajo 8, 16 in 33/ 19- kratnem najvišjem priporočenem odmerku pri človeku (200 mg trikrat dnevno).
Miglustat affected embryo/ foetal survival in rats and rabbits, dystocia was reported, post-implantation losses were increased, and an increased incidence of vascular anomalies occurred in rabbits.
Miglustat je vplival na preživetje zarodkov/ plodov pri podganah in zajcih, poročali so o distociji, povečale so se izgube po implantaciji in povečala se je incidenca žilnih nepravilnosti pri zajcih.
Sodium starch glycollate, Povidone (K30), Magnesium stearate.
Vsebina kapsule: natrijev karboksimetilškrob, povidon (K30), magnezijev stearat.
Gelatin, Water, Titanium dioxide (E171).
Ovoj kapsule: želatina, voda, titanov dioksid (E171).
Black iron oxide (E172) Shellac.
Tiskarsko črnilo: črn železov oksid (E172), šelak.
ACLAR/ ALU blister strips supplied as a box of 4 blister strips, each blister strip containing 21 capsules providing a total of 84 capsules.
ACLAR/ ALU pretisni omoti, štirje pretisni omoti na škatlo, vsak pretisni omot vsebuje 21 kapsul, skupaj 84 kapsul.
9 6.6 Special precautions for disposal
6. 6 Posebni varnostni ukrepi za odstranjevanje
Name and address of the manufacturer responsible for batch release Actelion Pharmaceuticals Deutschland GmbH Basler Strasse 63-65 79100 Freiburg Germany
Actelion Pharmaceuticals Deutschland GmbH Basler Strasse 63- 65 79100 Freiburg Nemčija
Risk Management Plan The MAH commits to performing the studies and additional pharmacovigilance activities detailed in the Pharmacovigilance Plan, as agreed in version 3 of the Risk Management Plan (RMP), dated 8 September 2008, presented in Module 1.8.2. of the Marketing Authorisation and any subsequent updates of the RMP agreed by the CHMP.
Načrt za obvladovanje tveganja Imetnik dovoljenja za promet se obvezuje, da bo izvajal študije in dodatne farmakovigilančne dejavnosti, podrobno opisane v načrtu farmakovigilance, sprejetem v različici 3 načrta za obvladovanje tveganja (RMP), z dne 8. septembra 2008, predstavljenem v modulu 1. 8. 2. vloge za pridobitev dovoljenja za promet in vseh nadaljnih posodobitvah RMP, s katerimi soglaša Odbor za zdravila za uporabo v humani medicini (CHMP)
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
Posodobljen načrt obvladovanja tveganj je treba predložiti tudi • ob prejemu novih informacij, ki lahko vplivajo na obstoječe specifikacije o varnosti, načrt farmakovigilance ali na dejavnosti za zmanjševanjem tveganja • v roku 60 dni po doseženem pomembnem mejniku (pri farmakovigilanci ali zmanjševanju tveganja) • na zahtevo Evropske agencije za zdravila (EMEA)
The MAH will continue to submit yearly PSURs unless otherwise specified by the CHMP.
Imetnik dovoljenja za promet bo letno predložil dopolnila k periodičnemu poročilu o varnosti zdravila, razen če CHMP določa drugače.
Clinical aspects:
12 Klinični vidiki:
Actelion commits to continue the comprehensive Zavesca Post-Marketing Surveillance Plan (PMSP) in the EU, which will educate prescribers on the appropriate use of Zavesca and
Actelion se zaveže, da bo v EU nadaljeval z obsežnim Načrtom nadzora po uvedbi na trg (Post - Marketing Surveillance Plan - PMSP) zdravila Zavesca, s katerim bo izdajatelje receptov poučil o pravilni uporabi zdravila Zavesca, hkrati pa bo od čim več bolnikov, ki uživajo zdravilo Zavesca, aktivno pridobival podatke o varnosti in tako redno dopolnjeval periodično poročilo o varnosti zdravila (PSUR - Periodic Safety Update Report).
The PMSP, as previously submitted to and agreed by CHMP will continue to be implemented prior to launch of Zavesca in each Member State.
Načrt nadzora po uvedbi na trg, ki je bil predhodno predložen CHMP in ga je ta odobril, se bo pred uvedbo na trg v vsaki državi članici še naprej izvajal.
Summary safety information gathered will be provided alongside the Annual Re-assessment/ PSURs.
Povzetek zbranih podatkov o varnosti bo predložen skupaj z letno ponovno presojo in rednimi dopolnili periodičnih poročil o varnosti zdravila.
Actelion will ensure that a capture protocol is continued for all patients currently receiving Zavesca in the ongoing type 1 Gaucher disease clinical studies.
Actelion bo zagotovil, da se bo protokol zajema nadaljeval za vse bolnike, ki trenutno prejemajo zdravilo Zavesca v potekajočih kliničnih študijah Gaucherjeve bolezni tipa 1.
Continued safety and efficacy assessments will be included as well as further periodic neurological and neuropsychological evaluations.
Vključene bodo nadaljnje ocene varnosti in učinkovitosti ter občasne nevrološke in nevropsihološke evalvacije.
This study is referenced as OGT 918-016.
Oznaka te študije je OGT 918- 016.
The final study report will be provided to the CHMP upon completion.
Končno poročilo o študiji bo ob zaključku predloženo CHMP.
Actelion will conduct an open-label study to provide safety and efficacy data for patients switched from Enzyme Replacement Therapy (ERT) to Zavesca.
Actelion bo izvedel odprto študijo za pridobitev podatkov o varnosti in učinkovitosti za bolnike, ki so encimsko nadomestno zdravljenje (ERT - Enzyme Replacement Therapy) zamenjali z zdravilom Zavesca.
This study is referenced as OGT 918-011.
Oznaka te študije je OGT 918- 011.