FrancophonIA/ELRC3.0 huggingface dataset:
English: Studies to evaluate the mutagenic and carcinogenic potential of sebelipase alfa have not been performed.
Danish: Der er ikke udført studier til evaluering af sebelipase alfas mutagene og karcinogene potentiale.
English: • Warning and precautions on the the risk of hypersensitivity including anaphylaxis or ADA development, with particular reference to symptoms, time to onset and severity.
Danish: • Advarsler og forsigtighedsregler vedrørende risikoen for overfølsomhedsreaktioner, herunder anafylaksi eller udvikling af ADA med særlig henvisning til symptomer, tid til debut og sværhedsgrad.
English: Almac Pharma Services Seagoe Industrial Estate Craigavon BT63 5UA United Kingdom
Danish: Almac Pharma Services Seagoe Industrial Estate Craigavon BT63 5UA Storbritannien
English: Children and adults with LAL deficiency
Danish: Børn og voksne med LAL-mangel
English: In LAL-CL03, sebelipase alfa was eliminated from the systemic circulation with a median T1/2 of 0.1 hr (range: 0.1-0.2) at the 3 mg/kg once weekly dose (n = 4).
Danish: I LAL-CL03 blev sebelipase alfa elimineret fra den systemiske cirkulation med en median-T1/2 på 0,1 t (spredning: 0,1-0,2) ved 3 mg/kg én gang ugentligt (n=4).
English: Patients were randomised to receive KANUMA at a dose of 1 mg/kg (n = 36) or placebo (n = 30) once every other week for 20 weeks in the double-blind period.
Danish: Patienterne blev randomiseret til at få KANUMA i en dosis på 1 mg/kg (n=36) eller placebo (n=30) én gang hver anden uge i 20 uger i den dobbeltblinde periode.
English: The dose you or your child receives is based on your or your child's body weight.
Danish: Den dosis, du eller dit barn skal have, beregnes ud fra kropsvægten.
English: Hypersensitivity reactions including anaphylaxis
Danish: Overfølsomhedsreaktioner, herunder anafylaksi
English: This means that it replaces the missing or defective LAL enzyme in patients with LAL deficiency.
Danish: Det vil sige, at den erstatter det manglende eller defekte LAL-enzym hos patienter med LAL-mangel.
English: These medicines may include antihistamines, fever-reducing medicines and/or corticosteroids (a type of antiinflammatory medicines).
Danish: Disse lægemidler kan omfatte antihistaminer, febernedsættende medicin og/eller kortikosteroider (medicin mod betændelseslignende reaktioner (inflammation)).
English: • Details on how to monitor for potential ADA formation following initiation of treatment with Kanuma, particularly in patients on Kanuma who experience clinically important hypersensitivity reactions or potential loss of clinical response.
Danish: • Detaljerede oplysninger om, hvordan monitorering for potentiel ADA-dannelse efter initiering af Kanuma-behandling udføres, særligt hos patienter, der oplever klinisk betydende overfølsomhedsreaktioner eller potentielt tab af klinisk respons.
English: The management of hypersensitivity reactions may include temporarily interrupting the infusion, lowering the infusion rate, and/or treatment with antihistamines, antipyretics, and/or corticosteroids.
Danish: Behandling af overfølsomhedsreaktioner kan omfatte midlertidig afbrydelse af infusionen, sænkning af infusionshastigheden og/eller behandling med antihistaminer, antipyretika og/eller kortikosteroider.
English: Tell your doctor if you or your child is on a controlled sodium diet.
Danish: Fortæl det til lægen, hvis du eller dit barn er på natrium- eller saltfattig diæt.
English: If not used immediately, in-use storage times and conditions prior to use are the responsibility of the user and would normally not be longer than 24 hours at 2°C to 8°C, or up to 12 hours below 25°C, unless dilution has taken place in controlled and validated aseptic conditions.
Danish: Hvis den ikke anvendes straks, er opbevaringstider og -betingelser for den brugsklare infusionsvæske brugerens ansvar og vil normalt ikke være længere end 24 timer ved 2 °C til 8 °C eller op til 12 timer ved temperaturer under 25 °C, medmindre fortynding har fundet sted under kontrollerede og validerede aseptiske forhold.
English: The difference in exposures to sebelipase alfa between the once weekly 0.35 mg/kg and 3 mg/kg groups was more than dose proportional, with a 8.6-fold increase in dose resulting in a 9.6-fold increase in exposure for AUC and a 10.0-fold increase for Cmax.
Danish: Forskellen i eksponering for sebelipase alfa mellem de grupper, der fik 0,35 mg/kg og 3 mg/kg en gang ugentligt, var mere end dosisproportional; en stigning på 8,6 gange i dosis medførte en stigning på 9,6 gange i eksponering (AUC) og en stigning på 10,0 gange i Cmax.
English: Clear glass vial (Type I) with a siliconised butyl rubber stopper, and an aluminium seal with a plastic flip-off cap, containing 10 ml of concentrate.
Danish: Klart glashætteglas (type I) med en silikoniseret butylgummiprop og en aluminiumforsegling med flipoff-låg af plastic, indeholdende 10 ml koncentrat.
English: Non-clinical data reveal no special hazard for humans based on conventional studies of safety pharmacology, repeated-dose toxicity in rats and monkeys, or fertility, embryo-foetal and peri- and postnatal development in rats and rabbits.
Danish: Prækliniske data viser ingen speciel risiko for mennesker vurderet ud fra konventionelle studier af sikkerhedsfarmakologi, toksicitet efter gentagne doser hos rotter og aber, fertilitet samt embryoføtal og peri- og postnatal udvikling hos rotter og kaniner.
English: Following the occurrence of a hypersensitivity reaction, appropriate pre-treatment should be considered according to the standard of care (see section 4.4).
Danish: Efter forekomst af en overfølsomhedsreaktion skal relevant forbehandling overvejes i overensstemmelse med gældende retningslinjer (se pkt.
English: You must not be given KANUMA: - if you or your child has experienced life-threatening allergic reactions to sebelipase alfa that cannot be managed when you or your child receives the medicine again, or to egg or any of the other ingredients of this medicine (listed in section 6).
Danish: Du må ikke få KANUMA: hvis du eller dit barn har oplevet livstruende allergiske reaktioner på sebelipase alfa, som ikke kan kontrolleres, når du eller dit barn får medicinen igen, eller på æg eller på et af de øvrige indholdsstoffer i KANUMA (angivet i afsnit 6).
English: KANUMA has no or negligible influence on the ability to drive and use machines.
Danish: KANUMA har ingen eller kun ubetydelig indflydelse på evnen til at føre motorkøretøj og betjene maskiner.
English: The other ingredients are trisodium citrate dihydrate (see section 2 under ‘KANUMA contains sodium'), citric acid monohydrate, human serum albumin, and water for injections.
Danish: Øvrige indholdsstoffer: Trinatriumcitratdihydrat (se afsnit 2 under ‘KANUMA indeholder natrium'), citronsyremonohydrat, humant serumalbumin og vand til injektionsvæsker.
English: There are no clinical data on the effects of sebelipase alfa on fertility.
Danish: Der foreligger ingen kliniske data om sebelipase alfas virkning på fertilitet.
English: The safety and efficacy of KANUMA in patients older than 65 years have not been evaluated and no alternative dose regimens can be recommended for these patients.
Danish: KANUMAs sikkerhed og virkning hos patienter over 65 år er ikke evalueret, og alternative dosisregimer for disse patienter kan ikke anbefales, se pkt.
English: From baseline to week 48, the mean reductions for ALT and AST were -34.0 U/l and -44.5 U/l, respectively.
Danish: Fra baseline til uge 48 var middelreduktionerne i ALAT og ASAT henholdsvis -34,0 E/l og -44,5 E/l.
English: Dose adjustments may be considered based on how well you or your child responds to treatment.
Danish: Dosisjustering kan overvejes alt efter, hvor godt behandlingen virker på dig eller dit barn.
English: KANUMA at doses up to 1 mg/kg once weekly resulted in improvements in alanine aminotransferase (ALT) and aspartate aminotransferase (AST) levels and weight gain within the first several weeks of treatment.
Danish: KANUMA i doser op til 1 mg/kg én gang ugentligt medførte forbedringer i alaninaminotransferase (ALAT)- og aspartataminotransferase (ASAT)-niveauer og vægtstigning inden for de første flere ugers behandling.
English: Primary Endpoint Normalisation of ALT
Danish: Primært endepunkt Normalisering af ALAT
English: What KANUMA contains
Danish: KANUMA indeholder
English: Reported as part of an adverse reaction in a single patient receiving KANUMA in LAL-CL02
Danish: Indberettet som en del af en bivirkning hos en enkelt patient, der fik KANUMA i LAL-CL02
English: Diarrhoea b,e
Danish: Almindelig Diarré b,e
English: There are no data from studies in breast-feeding women.
Danish: Der foreligger ingen data fra studier hos ammende kvinder.
English: Chronic toxicity studies in juvenile cynomolgous monkeys showed no toxicity at doses up to 3 times the recommended dose in infants and 10 times the recommended dose in adults/children.
Danish: Studier af kronisk toksicitet hos juvenile cynomolgus viste ingen toksicitet ved doser på op til 3 gange den anbefalede dosis til spædbørn og 10 gange den anbefalede dosis til voksne/børn.
English: No accumulation was seen at 1 mg/kg (once weekly or once every other week) or 3 mg/kg once weekly.
Danish: Der sås ingen akkumulering ved 1 mg/kg (én gang ugentligt eller én gang hver anden uge) og 3 mg/kg én gang ugentligt.
English: The European Medicines Agency has deferred the obligation to submit the results of studies with KANUMA in one or more subsets of the paediatric population in LAL deficiency (see section 4.2 for information on paediatric use).
Danish: Det Europæiske Lægemiddelagentur har udsat forpligtelsen til at fremlægge resultaterne af studier med KANUMA i en eller flere undergrupper af den pædiatriske population ved LAL-mangel (se pkt.
English: Lysosomal acid lipase (LAL) deficiency
Danish: Lysosomal sur lipase (LAL)-mangel
English: Fifty-six of 84 patients (67%) who received sebelipase alfa during clinical studies (LAL-CL01/LALCL04, LAL-CL02 and LAL-CL03) were in the paediatric and adolescent age range (1 month up to 18 years).
Danish: 56 af 84 patienter (67 %), der fik sebelipase alfa i de kliniske studier (LAL-CL01/LAL-CL04, LALCL02 og LAL-CL03), var børn og unge (1 måned op til 18 år).
English: In LAL-CL03, 6 of 9 KANUMA-treated infants survived beyond 12 months (67% 12-month survival, 95% CI: 30% to 93%).
Danish: I LAL-CL03 overlevede 6 ud af 9 KANUMA-behandlede spædbørn længere end til 12-måneders alderen (67 % 12-måneders overlevelse, 95 % CI: 30 % til 93 %).
English: Trisodium citrate dihydrate (see leaflet for further information) Citric acid monohydrate
Danish: Trinatriumcitratdihydrat (se indlægssedlen for yderligere oplysninger) Citronsyremonohydrat
English: Prior to dilution, the solution in the vials should be inspected visually; the solution should be clear to slightly opalescent, colourless to slightly coloured (yellow).
Danish: Før fortynding skal koncentratet i hætteglassene inspiceres visuelt; koncentratet skal være klart til svagt opaliserende, farveløst til svagt farvet (gul).
English: Evaluated in patients with abnormal baseline values (n = 36 for KANUMA; n = 29 for placebo).
Danish: Evalueret hos patienter med abnorme baseline-værdier (n=36 for KANUMA; n=29 for placebo).
English: Other medicines and KANUMA Tell your doctor if you or your child are using, have recently used or might use any other medicines.
Danish: Brug af anden medicin sammen med KANUMA Fortæl lægen, hvis du eller dit barn bruger anden medicin eller har gjort det for nylig.
English: Study LAL-CL08: an open-label, Phase 2 study in infants with rapidly progressive LAL Deficiency to explore long-term safety and efficacy data.
Danish: Studie LAL-CL08: et åbent, fase 2-studie hos spædbørn med hurtigt progredierende LAL-mangel med henblik på udforskning af data for langsigtet sikkerhed og virkning.
English: LAL deficiency is a rare disease associated with significant morbidity and mortality, which affects individuals from infancy through adulthood.
Danish: LAL-mangel er en sjælden sygdom, som er forbundet med signifikant morbiditet og mortalitet, og som rammer individer fra spædbarnsalderen til og med voksenlivet.
English: Patients received sebelipase alfa at 0.35 mg/kg once weekly for the first 2 weeks and then 1 mg/kg once weekly.
Danish: Patienterne fik sebelipase alfa med 0,35 mg/kg én gang ugentligt de første 2 uger og derefter 1 mg/kg én gang ugentligt.
English: This medicine works by lowering the build-up of fat that causes medical complications, including impaired growth, liver damage and heart complications.
Danish: Medicinen virker ved at sænke den ophobning af fedt, som giver de sundhedsmæssige komplikationer, herunder nedsat vækst, leverskade og komplikationer med hjertet.
English: The MAH shall ensure that in each Member State where Kanuma is marketed, all healthcare professionals who are expected to use Kanuma have access to the educational material.
Danish: Indehaveren af markedsføringstilladelsen skal sikre, at sundhedspersonalet i de enkelte medlemsstater, hvor Kanuma markedsføres, har adgang til undervisningsmaterialet.
English: d. Do not use if the solution is cloudy, or if foreign particulate matter is present.
Danish: d. Må ikke anvendes, hvis koncentratet er uklart, eller hvis der er fremmedlegemer i form af partikler til stede.
English: This medicine is an enzyme replacement therapy.
Danish: Denne medicin bruges som enzymerstatning.
English: Efficacy was assessed by comparing the survival experience of KANUMA-treated patients who survived past 12 months of age in LAL-CL03 with a historical cohort of untreated infants presenting with LAL deficiency with similar clinical characteristics.
Danish: Effekten blev vurderet ved at sammenligne overlevelseserfaringen for KANUMA-behandlede patienter, der overlevede længere end til 12-måneders alderen i LAL-CL03, med en historisk kohorte af ubehandlede spædbørn, som præsenterede med LAL-mangel med lignende kliniske karakteristika.
English: Placebo patients had persistently elevated serum transaminase and abnormal serum lipid levels during the double-blind period.
Danish: Patienterne i placebo-gruppen havde vedvarende forhøjede serum-aminotransferaser og abnorme serumlipidniveauer i den dobbeltlinde periode.
English: , abdominal pain b,e abdominal distension, nausea b e
Danish: , abdominalsmerter b,e abdominal distension, kvalme b e
English: All patients were receiving 1 mg/kg once every other week.
Danish: Alle patienterne fik 1 mg/kg én gang hver anden uge.
English: Non-interventional post-authorisation safety study (PASS): LAL Deficiency Registry: Non-interventional, multicentre, prospective disease and clinical outcome registry of patients with Lysosomal Acid Lipase Deficiency to further understand the disease, its progression and any associated complication, and to evaluate the long-term efficacy (normalisation of hepatic function) and safety
Danish: Ikke-interventionssikkerhedsundersøgelser efter tilladelse til markedsføring (PASS): LAL-mangel-register: Ikke-interventionelt, multicenter-, prospektivt register over sygdom og kliniske resultater for patienter med lysosomal sur lipase-mangel med det formål at opnå yderligere forståelse af sygdommen, dens progression og eventuelle tilknyttede komplikationer og at evaluere Kanumas langsigtede virkning (normalisering af leverfunktionen) og sikkerhed (særligt
English: There is limited information of sebelipase alfa pharmacokinetics in non-Caucasian ethnic groups.
Danish: Der foreligger begrænsede oplysninger om sebelipase alfas farmakokinetik hos ikke-kaukasiske befolkningsgrupper.
English: KANUMA contains the active substance sebelipase alfa.
Danish: KANUMA indeholder det aktive stof sebelipase alfa.
English: The following endpoints were assessed: normalisation of ALT, decrease in LDL-cholesterol, decrease in non-HDL-cholesterol, normalisation of AST, decrease in triglycerides, increase in HDLcholesterol, decrease in liver fat content assessed by multi-echo gradient echo magnetic resonance imaging (MEGE-MRI), and improvement in hepatic steatosis measured by morphometry.
Danish: Følgende endepunkter blev vurderet: Normalisering af ALAT, fald i LDL-kolesterol, fald i ikke-HDLkolesterol, normalisering af ASAT, fald i triglycerider, stigning i HDL-kolesterol, fald i leverfedtindhold vurderet ved multi-ekko-gradient-ekko magnetisk resonans-scanning (MEGE-MRI) og forbedring i hepatisk steatose målt ved morfometri.
English: Each vial of KANUMA is intended for single use only.
Danish: Hvert hætteglas med KANUMA er kun beregnet til engangsbrug.
English: Based on clinical response, dose escalation to 3 mg/kg once weekly occurred as early as 1 month and up to 20 months after starting treatment at 1 mg/kg.
Danish: Baseret på klinisk respons blev dosis optrappet til 3 mg/kg én gang ugentligt så tidligt som 1 måned og op til 20 måneder efter påbegyndelse af behandlingen med 1 mg/kg.
English: It is not known whether sebelipase alfa is excreted in human milk.
Danish: Det er ukendt, om sebelipase alfa/metabolitter udskilles i human mælk.
English: If you or your child has an egg allergy or a history of allergies to eggs, tell your doctor or nurse (see You must not be given KANUMA).
Danish: Hvis du eller dit barn er allergisk over for æg eller tidligere har reageret allergisk på æg, skal du fortælle det til lægen eller sundhedspersonalet (se Du må ikke få KANUMA).
English: Due to the proteinaceous nature of the product, slight flocculation (e.g., thin translucent fibres) may be present in the vialed solution and is acceptable for use.
Danish: Da præparatet er et proteinprodukt, kan der være let flokkulering (f.eks. tynde, gennemskinnelige fibre) til stede i koncentratet i hætteglassene, og dette er acceptabelt.
English: Open-label period
Danish: Åben periode
English: In LAL-CL03, 4 of 7 evaluable infants (57%) developed ADA during treatment with KANUMA.
Danish: I LAL-CL03 udviklede 4 ud af 7 evaluerbare spædbørn (57 %) ADA under behandlingen med KANUMA.
English: The educational material is aimed to encourage healthcare professionals to enrol patients in the prospective disease and clinical outcome registry of patients with Lysosomal Acid Lipase (LAL) Deficiency to monitor for efficacy and safety of Kanuma (LAL Deficiency Registry), with particular regard to hypersensitivity reactions, including anaphylaxis, and anti-drug antibodies (ADA) development impacting response to drug.
Danish: Undervisningsmaterialet sigter mod at opfordre sundhedspersonale til at indskrive patienter i det prospektive register over sygdom og kliniske resultater for patienter med lysosomal sur lipase (LAL)mangel med henblik på at overvåge Kanumas virkning og sikkerhed (LAL-mangel-register), særligt med hensyn til overfølsomhedsreaktioner, herunder anafylaksi, og hvordan udvikling af antistoffer mod lægemidlet (anti-drug antibodies (ADA)) påvirker lægemiddelresponset.
English: The pharmacokinetics of sebelipase alfa in children and adults were determined using a population pharmacokinetic analysis of 65 patients with LAL deficiency who received intravenous infusions of KANUMA at 1 mg/kg once every other week in LAL-CL02.
Danish: Sebelipase alfas farmakokinetik hos børn og voksne blev bestemt ved en farmakokinetisk populationsanalyse af 65 patienter med LAL-mangel, som fik intravenøse infusioner af KANUMA i en dosis på 1 mg/kg én gang hver anden uge i LAL-CL02.
English: QUALITATIVE AND QUANTITATIVE COMPOSITION Each ml of concentrate contains 2 mg sebelipase alfa*.
Danish: KVALITATIV OG KVANTITATIV SAMMENSÆTNING Hver ml koncentrat indeholder 2 mg sebelipase alfa*.
English: If treated with KANUMA, you or your child may experience a side effect while you or your child is being given the medicine or during the hours following the infusion (see section 4).
Danish: Hvis du eller dit barn bliver behandlet med KANUMA, kan du eller dit barn få en bivirkning, mens du eller dit barn får medicinen, eller i timerne efter infusionen (se afsnit 4).
English: A decision must be made whether to discontinue breast-feeding or to discontinue/abstain from sebelipase alfa therapy taking into account the benefit of breast-feeding for the child and the benefit of therapy for the woman.
Danish: Det skal besluttes, om amning skal ophøre eller behandling med sebelipase alfa seponeres, idet der tages højde for fordelene ved amning for barnet i forhold til de terapeutiske fordele for moderen.
English: Medical or healthcare professionals are encouraged to participate and enrol all patients diagnosed with LAL deficiency in the LAL deficiency registry.
Danish: Læger eller sundhedspersonale opfordres til at tage del i og indskrive alle patienter, der får stillet diagnosen LAL-mangel, i LAL-mangel-registeret.
English: In patients who had received KANUMA during the double-blind period, reductions in ALT levels during the first 20 weeks of treatment were maintained and further improvements were seen in lipid parameters including LDL-cholesterol and HDL-cholesterol levels.
Danish: Hos de patienter, der havde fået KANUMA i den dobbeltblinde periode, blev de reducerede ALAT-niveauer fra de første 20 ugers behandling opretholdt, og der sås yderligere forbedringer i lipidparametre, inklusive LDL-kolesterol- og HDL-kolesterol-niveauer.
English: Lipid accumulation in the intestinal wall leads to malabsorption and growth failure.
Danish: Lipidakkumulering i tarmvæggen medfører malabsorption og væksthæmning.
English: AUCss = Area under the plasma concentration time curve at steady-state Cmax = Maximum concentration
Danish: AUCss = Areal under plasmakoncentration/tid-kurven ved steady-state Cmax = Maksimumkoncentration Tmax = Tid til maksimumkoncentration CL = Clearance Vc = Centralt fordelingsvolumen T1/2 = Halveringstid
English: What you need to know before KANUMA is given
Danish: Det skal du vide, før du begynder at få KANUMA
English: No adverse findings were observed in rat and rabbit embryofoetal development studies at doses up to at least 10 times the adult/children recommended dose and in rat fertility and peri- postnatal development studies at doses up to 10 times the adult/children recommended dose.
Danish: Der blev ikke observeret negative virkninger i studier af rotters og kaniners embryoføtale udvikling ved doser på op til mindst 10 gange den anbefalede dosis til voksne/børn eller i studier af rotters fertilitet og peri- og postnatale udvikling ved doser på op til 10 gange den anbefalede dosis til voksne/børn.
English: LAL deficiency is an autosomal recessive lysosomal storage disorder characterised by a genetic defect resulting in a marked decrease or loss in activity of the lysosomal acid lipase (LAL) enzyme.
Danish: LAL-mangel er en autosomal, recessiv lysosomal lagringsforstyrrelse, som er kendetegnet ved en genetisk defekt, som medfører et markant fald i eller tab af aktiviteten af enzymet lysosomal sur lipase (LAL).
English: For patients who have signs and symptoms of the disease when they are infants, the recommended starting dose is 1 mg/kg once weekly.
Danish: For patienter, der har tegn og symptomer på sygdommen, mens de er spæde, er den anbefalede startdosis 1 mg pr. kg kropsvægt én gang om ugen.
English: In a separate open-label study (LAL-CL01/LAL-CL04) in adult patients with LAL deficiency, improvements in serum transaminase and lipid levels were sustained through the 104-week treatment period.
Danish: I et separat åbent studie (LAL-CL01/LAL-CL04) hos voksne patienter med LAL-mangel blev forbedringerne i serum-aminotransferase og serum-lipider opretholdt gennem hele den 104 uger lange behandlingsperiode.
English: LDL-cholesterol, mean % change from baseline non-HDL-cholesterol, mean % change from baseline Normalisation of AST
Danish: LDL-kolesterol, gennemsnitlig procentvis ændring fra baseline ikke-HDL-kolesterol, middel % ændring fra baseline Normalisering af ASAT
English: Mechanism of action Sebelipase alfa is a recombinant human lysosomal acid lipase (rhLAL).
Danish: Virkningsmekanisme Sebelipase alfa er en rekombinant human lysosomal sur lipase (rhLAL).
English: Dose escalation to 3 mg/kg once weekly should be considered based on clinical response.
Danish: Dosisoptrapning til 3 mg/kg én gang ugentligt skal overvejes baseret på klinisk respons.