natgillin/translations-raw huggingface dataset:
Zavescas virkning på Gauchers sygdom type 1 blev undersøgt i en hovedundersøgelse, der omfattede 28 patienter med let til moderat Gauchers sygdom type 1, som ikke kunne eller ikke ville modtage enzymsubstitutionsbehandling.
For mild to moderate type 1 Gaucher disease, the effectiveness of Zavesca was investigated in one main study involving 28 patients who were unable or unwilling to receive ERT.
Undersøgelsen målte, om Zavesca havde en effekt på størrelsen af leveren og milten, og om lægemidlet forbedrede blodbilledet, f. eks. koncentrationen af hæmoglobin (et protein, som findes i de røde blodlegemer, der transporterer ilt rundt i kroppen) og trombocyttallet (komponenter, som hjælper blodet med at størkne).
The study measured if Zavesca had an effect on the size of the liver and spleen, and if it improved blood counts, such as the levels of haemoglobin (a protein found in red blood cells that carries oxygen around the body) and platelet counts (components that help the blood to clot).
Virkningen af Zavesca til behandling af Niemann- Picks sygdom type C blev undersøgt i en hovedundersøgelse af 31 patienter, hvoraf 12 var under 12 år.
For Niemann-Pick type C disease, the effectiveness of Zavesca was studied in one main study involving 31 patients, 12 of whom were less than 12 years old.
I undersøgelsen sammenlignede man virkningen af standardbehandling suppleret med Zavesca med virkningen af standardbehandling alene.
The study compared the effects of adding Zavesca to standard care (measures used to relieve symptoms) with standard care alone.
Det primære mål for virkningen var ændringen i den hastighed, hvormed patienterne foretog hurtige vandrette øjenbevægelser efter et års behandling, men undersøgelsen så også på andre neurologiske symptomer såsom patienternes evne til at synke og intellektuelle funktion.
The main measure of effectiveness was the change in the speed at which the patients made saccadic horizontal eye movements after a year, but the study also looked at other neurological symptoms such as the patients ’ ability to swallow and their intellectual function.
Der blev også gennemført en spørgeundersøgelse blandt 66 patienter, der var blevet behandlet med Zavesca.
A survey of 66 patients treated with Zavesca was also carried out.
Efter et års undersøgelse af Gauchers sygdom type 1 var levervolumenet reduceret med 12% og miltvolumenet med 19, 0%.
In the study of type 1 Gaucher disease, there was a reduction in liver organ size of 12% and a reduction in spleen size of 19% after a year.
I gennemsnit blev hæmoglobinkoncentrationen øget med 0, 26 g/ dl og blodpladetallet med 8, 29 millioner pr. milliliter.
On average, the levels of haemoglobin increased by 0.26 g per decilitre and platelet counts increased by 8.29 million per millilitre.
Fordelene ved Zavesca blev opretholdt i løbet af tre års kontinuerlig behandling.
The benefits of Zavesca were maintained over three years of continuous treatment.
I undersøgelsen af Niemann- Picks sygdom type C var forbedringen i øjenbevægelserne den samme for patienter, der var blevet behandlet med og uden Zavesca.
In the study of Niemann-Pick type C disease, the improvement in eye movements was similar in patients treated with and without Zavesca.
Der var der tegn på forbedringer i evnen til at synke og den intellektuelle funktion hos patienter behandlet med Zavesca.
However, there were signs of improvement in swallowing ability and intellectual function in the patients treated with Zavesca.
De hyppigste bivirkninger ved Zavesca (som optræder hos flere end 1 ud af 10 patienter) er vægttab, rysten, diarré, luft i maven og mavesmerter.
The most common side effects with Zavesca (seen in more than 1 patient in 10) are weight loss, tremor (shaking), diarrhoea, flatulence (gas) and abdominal pain (stomach ache).
Den fuldstændige liste over de indberettede bivirkninger ved Zavesca fremgår af indlægssedlen.
For the full list of all side effects reported with Zavesca, see the Package Leaflet.
Zavesca må ikke anvendes til personer, der kan være overfølsomme (allergiske) over for miglustat eller andre af hjælpestofferne.
Zavesca should not be used in people who may be hypersensitive (allergic) to miglustat or any of the other ingredients.
Udvalget for Humanmedicinske Lægemidler (CHMP) konkluderede, at fordelene ved Zavesca kun er større end risiciene ved oral behandling af voksne patienter med let eller moderat Gauchers sygdom type 1, som ikke er egnet til enzymsubstitutionsbehandling, og ved behandling af progressive neurologiske manifestationer hos voksne og pædiatriske patienter med Niemann- Picks sygdom type C.
The Committee for Medicinal Products for Human Use (CHMP) decided that Zavesca’ s benefits are greater than its risks for the oral treatment of adult patients with mild to moderate type 1 Gaucher disease for whom ERT is unsuitable, and of progressive neurological manifestations in adult and paediatric patients with Niemann-Pick type C disease.
Udvalget anbefalede udstedelse af markedsføringstilladelse for Zavesca.
The Committee recommended that Zavesca be given marketing authorisation.
Zavesca er blevet godkendt under "særlige omstændigheder", da det ikke er muligt at få fuldstændige oplysninger om lægemidlet, fordi sygdommen er sjælden.
Zavesca has been authorised under ‘ Exceptional Circumstances’ because, as the disease is rare, it has not been possible to obtain complete information on the medicine.
Hvert år gennemgår Det Europæiske Lægemiddelagentur (EMEA) eventuelle nye, tilgængelige oplysninger og ajourfører dette sammendrag efter behov.
Every year, the European Medicines Agency (EMEA) will review any new information that may become available and this summary will be updated as necessary.
Hvilke oplysninger afventes der stadig for Zavesca?
2/ 3 What information is still awaited for Zavesca?
Den virksomhed, der fremstiller Zavesca, skal gennemføre følgende undersøgelser vedrørende lægemidlets effekt og sikkerhed ved Gauchers sygdom type 1:
The company that makes Zavesca will complete the following studies to address the product’ s effectiveness and safety in type 1 Gaucher disease:  a post-marketing plan to inform doctors on how to use Zavesca and to get patients to report information on the safety of the medicine;  a study to follow up patients already in clinical trials, to continue assessment of the medicine’ s safety and effectiveness and to find out more about the neurological aspects of the disease;  a study to look at the effectiveness and safety of Zavesca in patients who switch from ERT;  a further study of the natural history of the disease, together with the European Working Group for Gaucher Disease (EWGGD).
Europa- Kommissionen udstedte en markedsføringstilladelse med gyldighed i hele Den Europæiske Union for Zavesca den 20. november 2002.
The European Commission granted a marketing authorisation valid throughout the European Union for Zavesca on 20 November 2002.
Indehaveren af markedsføringstilladelsen er Actelion Registration Ltd.
The marketing authorisation holder is Actelion Registration Ltd.
Markedsføringstilladelsen blev fornyet den 20. november 2007.
The marketing authorisation was renewed on 20 November 2007.
Oplysninger om udpegelsen af Zavesca som lægemiddel til sjældne sygdomme kan ses her (Gauchers sygdom type 1) og her (Niemann- Picks sygdom type C).
The records of Zavesca’ s designation as an orphan medicine are available here (type 1 Gaucher disease) and here (Niemann-Pick type C disease).
Den fuldstændige EPAR for Zavesca findes her.
The full EPAR for Zavesca can be found here.
Zavesca 100 mg hårde kapsler.
Zavesca 100 mg hard capsules.
Hvide kapsler med "OGT 918" trykt i sort på overdelen og "100" trykt i sort på underdelen.
White capsules with “ OGT 918” printed in black on the cap and “ 100” printed in black on the body.
4. 4 og 5. 1).
Zavesca may be used only in the treatment of patients for whom enzyme replacement therapy is unsuitable (see sections 4.4 and 5.1).
4. 4 og 5. 1).
Zavesca is indicated for the treatment of progressive neurological manifestations in adult patients and paediatric patients with Niemann-Pick type C disease (see sections 4.4, and 5.1).
Behandlingen bør ledes af læger, der har kendskab til behandling af Gauchers sygdom eller Niemann - Picks sygdom, type C.
Therapy should be directed by physicians who are knowledgeable in the management of Gaucher disease or Niemann-Pick type C disease, as appropriate.
Der er ingen erfaring med anvendelse af Zavesca til patienter under 18 år med Gauchers sygdom, type 1.
There is no experience with the use of Zavesca in patients with type 1 Gaucher disease under the age of 18.
Anvendelsen af Zavesca anbefales derfor ikke til børn eller unge med Gauchers sygdom, type 1.
The use of Zavesca is therefore not recommended in children or adolescents with type 1 Gaucher disease.
Der foreligger ingen erfaring med anvendelsen af Zavesca til patienter over 70 år.
There is no experience with the use of Zavesca in patients over the age of 70.
Dosis ved Niemann- Picks sygdom, type C
Dosage in Niemann-Pick type C disease
Den anbefalede dosis til behandling af voksne og unge patienter med Niemann- Picks sygdom, type C, er 200 mg tre gange dagligt.
The recommended dose for the treatment of adult and adolescent patients with Niemann-Pick type C disease is 200 mg three times a day.
2 Dosis til patienter under 12 år skal justeres på basis af legemsoverflade som vist nedenfor:
Dosing in patients under the age of 12 years should be adjusted on the basis of body surface area as illustrated below:
200 mg tre gange dagligt 200 mg to gange dagligt 100 mg tre gange dagligt 100 mg to gange dagligt 100 mg én gang dagligt
200 mg three times a day 200 mg twice a day 100 mg three times a day 100 mg twice a day 100 mg once a day
4. 4).
The benefit to the patient of treatment with Zavesca should be evaluated on a regular basis (see section 4.4).
Der er begrænset erfaring med anvendelsen af Zavesca til patienter under 4 år med Niemann- Picks sygdom, type C.
There is limited experience with the use of Zavesca in Niemann-Pick type C disease patients under the age of 4 years.
Nyreinsufficiens
Renal Impairment
Hos patienter med en justeret kreatinin- clearance på 50- 70 ml/ min/ 1, 73 m2 bør indgivelsen starte med en dosis på 100 mg to gange dagligt hos patienter med Gauchers sygdom, type 1 og en dosis på 200 mg to gange dagligt (justeret efter legemsoverflade hos patienter under 12 år) hos patienter med Niemann- Picks sygdom, type C.
In patients with an adjusted creatinine clearance of 50– 70 ml/ min/ 1.73 m2, administration should commence at a dose of 100 mg twice daily in patients with type 1 Gaucher disease and at a dose of 200 mg twice daily (adjusted for body surface area in patients below the age of 12) in patients with Niemann-Pick type C disease.
Hos patienter med en justeret kreatinin- clearance på 30- 50 ml/ min/ 1, 73 m2 bør indgivelsen starte med en dosis på 100 mg én gang dagligt hos patienter med Gauchers sygdom, type 1 og en dosis på 100 mg to gange dagligt (justeret efter legemsoverflade hos patienter under 12 år) hos patienter med Niemann - Picks sygdom, type C.
In patients with an adjusted creatinine clearance of 30– 50 ml/ min/ 1.73 m2, administration should commence at a dose of 100 mg once daily in patients with type 1 Gaucher disease and at a dose of 100 mg twice daily (adjusted for body surface area in patients below the age of 12) in patients with Niemann-Pick type C disease.
4. 4 og 5. 2).
Use in patients with severe renal impairment (creatinine clearance < 30 ml/ min/ 1.73 m2) is not recommended (see sections 4.4 and 5.2).
Leverinsufficiens
Hepatic Impairment
Selv om der ikke er udført nogen direkte sammenligninger med enzymsubstitutionsterapi (ERT) hos behandlingsnaive patienter med Gauchers sygdom, type 1, er der intet bevis for at Zavesca har en effektivitets - eller sikkerhedsfordel i forhold til ERT.
Although no direct comparisons with Enzyme Replacement Therapy (ERT) have been performed in treatment-naive patients with type 1 Gaucher disease, there is no evidence of Zavesca having an efficacy or safety advantage over ERT.
5. 1).
ERT is the standard of care for patients who require treatment for type 1 Gaucher disease (see section 5.1).
Effektivitet og sikkerhed for Zavesca er ikke specifikt evalueret hos patienter med svær Gauchers sygdom.
The efficacy and safety of Zavesca has not been specifically evaluated in patients with severe Gaucher disease.
Cirka 38% af patienterne i kliniske studier med Gauchers sygdom, type 1 og 58% af patienterne i kliniske studier med Niemann- Picks sygdom, type C, rapporterede om tremor ved behandling.
Approximately 38% of patients in clinical trials in type 1 Gaucher disease, and 58% of patients in a clinical trial in Niemann-Pick type C disease reported tremor on treatment.
Regelmæssig monitorering af vitamin B12- niveau anbefales på grund af den høje forekomst af vitamin B12- mangel hos patienter med Gauchers sygdom, type 1.
Regular monitoring of vitamin B12 level is recommended because of the high prevalence of vitamin B12 deficiency in patients with type 1 Gaucher disease.
3 Der er indberettet tilfælde af perifer neuropati hos patienter behandlet med Zavesca med eller uden samtidige tilstande, så som vitamin B12- mangel og monoklonal gammopati.
Peripheral B
Perifer neuropati synes at være mere almindelig hos patienter med Gauchers sygdom, type I sammenlignet med befolkningen i almindelighed.
neuropathy seems to be more common in patients with type 1 Gaucher disease compared to the general population.
Alle patienter bør underkastes neurologisk vurdering ved påbegyndelse af behandling og løbende derefter.
All patients should undergo baseline and repeat neurological evaluation.
Patienter, der udvikler symptomer, så som følelsesløshed og snurren, bør have en omhyggelig revurdering af risk/ benefit.
Patients who develop symptoms such as numbness and tingling should have a careful re-assessment of risk-benefit.
Gastrointestinale hændelser, herunder hovedsageligt diarré, er observeret hos mere end 80% af patienterne, enten ved behandlingens start eller intermitterende under behandlingen (se pkt.
Gastrointestinal events, mainly diarrhoea, have been observed in more than 80% of patients, either at the outset of treatment or intermittently during treatment (see section 4.8).
Mekanismen er sandsynligvis hæmning af disaccharidaser i mave/ tarmkanalen.
The mechanism is probably inhibition of disaccharidases in the gastrointestinal tract.
Størstedelen af tilfældene er milde og forventes at svinde spontant ved behandling.
The majority of cases are mild and are expected to resolve spontaneously on therapy.
I klinisk praksis er det observeret, at diarré reagerer på kostændringer (nedsættelse af indtaget af lactose og andre kulhydrater), på indtagelse af Zavesca uden for måltiderne og/ eller på lægemidler mod diarré, såsom loperamid.
In clinical practice, diarrhoea has been observed to respond to diet modification (reduction of lactose and other carbohydrate intake), to taking Zavesca away from meals, and/ or to anti-diarrhoeal medicinal products such as loperamide.
Patienter med kronisk diarré eller andre vedvarende gastrointestinale hændelser, som ikke reagerer på disse indgreb, skal undersøges nærmere i overensstemmelse med klinisk praksis.
Patients with chronic diarrhoea or other persistent gastrointestinal events that do not respond to these interventions should be investigated according to clinical practice.
Zavesca er ikke evalueret til patienter med tidligere tilfælde af signifikant gastrointestinal sygdom, herunder inflammatorisk tarmsygdom.
Zavesca has not been evaluated in patients with a history of significant gastrointestinal disease, including inflammatory bowel disease.
4. 6 og 5. 3).
Studies in the rat have shown that miglustat adversely affects spermatogenesis and sperm parameters, and reduces fertility (see sections 4.6 and 5.3).
På grund af begrænset erfaring bør Zavesca anvendes med forsigtighed hos patienter med nyre - eller leverinsufficiens.
Due to limited experience, Zavesca should be used with caution in patients with renal or hepatic impairment.
5. 2).
There is a close relationship between renal function and clearance of miglustat, and exposure of miglustat is markedly increased in patients with severe renal impairment (see section 5.2).
Anvendelse af Zavesca til patienter med svær nyreinsufficiens (kreatinin- clearance < 30 ml/ min/ 1, 73 m2) kan ikke anbefales.
Use of Zavesca in patients with severe renal impairment (creatinine clearance < 30 ml/ min/ 1.73 m2) is not recommended.
Niemann- Picks sygdom, type C
Niemann-Pick type C disease
Fortsat behandling skal revurderes efter mindst 1 års behandling med Zavesca.
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.
Der har i den tidlige fase af behandlingen med miglustat hos nogle pædiatriske patienter med Niemann- Picks sygdom, type C, været rapporteret reduceret vækst, hvor den initialt reducerede vægtøgning kan være ledsaget og efterfulgt af reduceret højdevækst.
Reduced growth has been reported in some paediatric patients with Niemann-Pick type C disease in the early phase of treatment with miglustat where the initial reduced weight gain may be accompanied or followed by reduced height gain.
Fordel/ risiko- balancen for fortsat behandling skal revurderes på individuel basis.
Growth should be monitored in paediatric and adolescent patients during treatment with Zavesca; the benefit/ risk balance should be re-assessed on an individual basis for continuation of therapy.
Der er observeret let nedsat thrombocyttal uden associeret blødning hos nogle patienter med Niemann - Picks sygdom, type C, som er behandlet med Zavesca.
Mild reductions in platelet counts without association to bleeding were observed in some patients with Niemann-Pick type C disease treated with Zavesca.
Hos patienter, der var inkluderet i den kliniske undersøgelse, havde 40- 50% af patienterne ved baseline thrombocyttal, der lå under den nedre normale grænseværdi.
In patients included in the clinical trial, 40%-50% of patients had platelet counts below the lower limit of normal at baseline.
Begrænsede data tyder på, at samtidig indgivelse af Zavesca og Cerezym hos patienter med Gauchers sygdom, type 1 kan medføre reduceret eksponering for miglustat (reduktioner på cirka 22% i Cmax og 14% i AUC blev observeret i en lille parallel- gruppeundersøgelse).
Limited data suggest that co-administration of Zavesca and Cerezyme in patients with type 1 Gaucher disease may result in decreased exposure to miglustat (approximate reductions of 22% in Cmax and 14% in AUC were observed in a small parallel-group study).
Denne undersøgelse tyder også på, at Zavesca ingen eller begrænset effekt har på Cerezyms farmakokinetik.
This study also indicated that Zavesca has no or limited effect on the pharmacokinetics of Cerezyme.
Der foreligger ikke tilstrækkelige data om brugen af miglustat hos gravide kvinder.
4 There are no adequate data from the use of miglustat in pregnant women.
5. 3).
Studies in animals have shown reproductive toxicity, including dystocia (see section 5.3).
Kvinder i den fødedygtige alder bør anvende prævention.
Contraceptive measures should be used by women of childbearing potential.
Det vides ikke, om miglustat udskilles i modermælk.
It is not known if miglustat is secreted in breast milk.
Zavesca bør ikke anvendes under amning.
Zavesca should not be used during breast-feeding.
4. 4 og 5. 3).
Male patients should maintain reliable contraceptive methods while taking Zavesca and for 3 months after finishing treatment (see sections 4.4 and 5.3)
Der er ikke foretaget undersøgelser af Zavescas indflydelse på evnen til at føre motorkøretøj eller betjene maskiner.
No studies on the effects of Zavesca on the ability to drive or use machines have been performed.
Dog er der indberettet svimmelhed som en meget almindelig bivirkning, og patienter, der lider af svimmelhed, bør ikke køre bil eller betjene maskiner.
However, dizziness has been reported as a very common adverse event, and patients suffering from dizziness should not drive or operate machinery.
I ni kliniske undersøgelser med forskellige indikationer blev 206 patienter behandlet med Zavesca i doser på 50- 200 mg t. i. d. i en periode på gennemsnitligt 2, 2 år.
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.
Ud af disse patienter havde 90 Gauchers sygdom, type 1 og 40 havde Niemann- Picks sygdom, type C.
Of these patients, 90 had type 1 Gaucher disease, and 40 had Niemann-Pick type C disease.
Bivirkningerne var generelt af mild til moderat sværhedsgrad og forekom med ens hyppighed uafhængigt af indikation og dosering.
Adverse reactions were generally of mild to moderate severity and occurred with similar frequency across indications and dosages tested.
De hyppigst forekommende bivirkninger var gastrointestinale bivirkninger med diarré og andre abdominale gener samt vægttab.
The most common adverse reactions were gastrointestinal, with diarrhoea and other abdominal complaints, and weight loss.
Bivirkninger, der er defineret som behandlingskrævende bivirkninger, og som af behandleren er rapporteret som værende relaterede til behandlingen, og som forekommer hos > 1% af patienterne, er angivet i tabellen nedenfor efter organsystem og frekvens (meget almindelig: ≥1/ 10, almindelig ≥1/ 100, < 1/ 10)..
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).
Bivirkningerne inden for hver frekvensgruppe er angivet i forhold til hvor alvorlige de er, med de alvorligste først.
Within each frequency grouping, adverse reactions are presented in order of decreasing seriousness.
Trombocytopeni
Blood and lymphatic system disorders Common
Metabolisme og ernæring
Thrombocytopenia
Meget almindelig Almindelig
Metabolism and Nutrition Disorders Very common Common
Vægttab Anoreksi, nedsat appetit
Weight loss Anorexia, decreased appetite
Søvnløshed, nedsat libido
Insomnia, libido decreased
Nervesystemet Meget almindelig Almindelig
Nervous System Disorders Very common Common
Tremor Perifer neuropati, hovedpine, paræstesi, svimmelhed, ataksi, hypoæstesi
Tremor, Peripheral neuropathy, headache, paraesthesia, dizziness, ataxia, hypoaesthesia
Meget almindelig Almindelig
Gastrointestinal Disorders Very common Common
Dyspepsi
Diarrhoea, flatulence, abdominal pain Nausea, vomiting, abdominal distension/ discomfort, constipation, dyspepsia
Almene symptomer og reaktioner på administrationsstedet
General disorders and administration site reactions Common:
5 Almindelig
Investigations
Undersøgelser
5
Unormale nerveimpulsoverledningsstudier
Nerve conduction studies abnormal,
60% af patienterne.
Weight loss has been observed in approximately 60% of patients.
Den største virkning forekom efter 12 måneder med et gennemsnitligt vægttab på 6- 7% af legemsvægten med en efterfølgende tendens til vægtforøgelse hen imod værdien ved behandlingens påbegyndelse.
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.
Zavesca er undersøgt for indikationer, hvor visse hændelser rapporteret som bivirkninger, som f. eks. neurologiske symptomer/ tegn og trombocytopeni, også kunne skyldes den underliggende sygdomme.
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.
Enkeltstående tilfælde af kognitiv dysfunktion har været rapporteret i kliniske forsøg med Zavesca ved Gauchers sygdom, type 1.
Isolated cases of cognitive dysfunction have been reported during clinical trials of Zavesca in type 1 Gaucher disease.
En kausal sammenhæng med Zavesca er ikke fastlagt.
A causal relationship to Zavesca has not been established.