natgillin/translations-raw huggingface dataset:
If lumacaftor/ivacaftor is used in these patients, they should be closely monitored after the initiation of treatment and the dose should be reduced (see sections 4.2, 4.8, and 5.2).
Če uporabljamo lumakaftor/ivakaftor pri teh bolnikih, jih moramo po uvedbi zdravljenja skrbno spremljati, odmerek pa moramo zmanjšati (glejte poglavja 4.2, 4.8 in 5.2).
Treatment with lumacaftor/ivacaftor resulted in no significant improvement in ppFEV1 relative to placebo in patients with CF heterozygous for the F508del mutation in the CFTR gene (treatment difference 0.60 [P = 0.5978]) and no meaningful improvements in BMI or weight (see section 4.4).
Zdravljenje z lumakaftorjem/ivakaftorjem ni povzročilo niti signifikantnega izboljšanja ppFEV1 glede na placebo pri bolnikih s CF, heterozigotnih za mutacijo F508del na genu CFTR (razlika zaradi zdravljenja 0,60 [P = 0,5978]) niti pomembnih izboljšanj ITM ali telesne mase (glejte poglavje 4.4).
Secondary endpoints included absolute change from baseline in sweat chloride at Week 24 and absolute change in sweat chloride from Week 24 at Week 26 (see Pharmacodynamic effects) as well as the endpoints listed in Table 8.
Sekundarna končna stanja so vključevala absolutno spremembo kloridov v znoju od izhodišča v 24. tednu in absolutno spremembo kloridov v znoju od 24. tedna v 26. tednu (glejte Farmakodinamični učinki), pa tudi končna stanja, navedena v preglednici 8.
Immunosuppressants: ciclosporin, everolimus, sirolimus, tacrolimus (used after organ transplant)
Zdravila za zaviranje imunske odzivnosti: ciklosporin, everolimus, sirolimus, takrolimus (uporabljajo se po presaditvi organov)
While taking Orkambi, you may notice that your child's breathing is easier, that they do not get ill as often, and/or that it is easier to gain weight.
Med jemanjem zdravila Orkambi boste mogoče opazili, da vaš otrok lažje diha, da ne zboli tako pogosto in/ali da lažje pridobi telesno maso.
• Printing ink: shellac; iron oxide black (E172); propylene glycol; and ammonium hydroxide.
• tiskarsko črnilo: šelak; črni železov oksid (E172); propilenglikol; in amonijev hidroksid.
• Obligation to conduct post-authorisation measures:
• Obveznost izvedbe ukrepov po pridobitvi dovoljenja za promet: Imetnik dovoljenja za promet z zdravilom mora v določenem časovnem okviru izvesti naslednji ukrep:
In the subgroup of 28 patients who were initiated at the full dose of lumacaftor/ivacaftor (2 tablets every 12 hours), the incidence was 71.4%, and in the 18 patients who were initiated at a reduced dose of lumacaftor/ivacaftor (1 tablet every 12 hours for up to 2 weeks, and subsequently increased to the full dose), the incidence was 55.6%.
V podskupini 28 bolnikov, ki so jim uvedli lumakaftor/ivakaftor v polnem odmerku (2 tableti vsakih 12 ur), je bila incidenca 71,4 %, pri 18 bolnikih, ki so jim uvedli lumakaftor/ivakaftor v zmanjšanem odmerku (1 tableta vsakih 12 ur do 2 tedna, nato pa zvečanje na polni odmerek), pa je bila incidenca 55,6 %.
The side effects reported with Orkambi and ivacaftor alone (one of the active substances of Orkambi) are listed below and may occur with the use of Orkambi.
Neželeni učinki, o katerih poročajo pri zdravilu Orkambi in pri ivakaftorju samem (ena od učinkovin zdravila Orkambi), so navedeni v nadaljevanju in se lahko pojavijo pri jemanju zdravila Orkambi.
Trial 7 was a 24-week, placebo-controlled, Phase 3 clinical study in 204 patients with CF aged 6 to 11 years old (mean age 8.8 years).
Preskušanje 7 je bila 24-tedenska, s placebom kontrolirana klinična študija faze 3 pri 204 bolnikih s CF, starih 6 do 11 let (povprečna starost 8,8 leta).
Relative change in ppFEV1 at Week 24 (%)
Relativna sprememba ppFEV1 v 24. tednu (%)
The exact mechanisms by which lumacaftor improves cellular processing and trafficking of F508del-CFTR and ivacaftor potentiates F508del-CFTR are not known.
Natančni mehanizmi, s katerimi lumakaftor izboljša celično procesiranje in transport F508del-CFTR in ivakaftor ojačuje F508del-CFTR, niso znani.
The typical CL/F (CV) of ivacaftor when given in combination with lumacaftor was estimated to be 25.1 L/hr (40.5%) for patients with CF.
Tipični CL/F (CV) ivakaftorja, kadar so ga dajali bolnikom s CF skupaj z lumakaftorjem, so ocenjevali na 25,1 l/h (40,5 %).
Your child's doctor may request another test to verify results.
Otrokov zdravnik bo mogoče preveril rezultate z dodatno preiskavo.
• Tablet coating: polyvinyl alcohol; titanium dioxide (E171); macrogol 3350; talc; carmine (E120);
• filmska obloga tablete: polivinilalkohol; titanov dioksid (E171); makrogol 3350; smukec; karmin (E120); briljantno modro FCF (E133); in indigotin (E132);
• Abnormal periods, including the absence or infrequent menses, or more frequent or heavier menstrual bleeding
• motnje menstrualnega ciklusa, vključno z odsotnostjo menstruacij ali redkimi menstruacijami,
Trial 3 was a Phase 3, parallel-group, multicentre, rollover extension study in patients with CF that included patients aged 12 years and older from Trial 1 and Trial 2.
Dolgoročna preverjalna študija varnosti in učinkovitosti Preskušanje 3 je bila multicentrična preverjalna podaljšana študija 3. faze s paralelnimi skupinami pri bolnikih s CF, ki je vključila bolnike, stare 12 let ali starejše, iz preskušanja 1 in preskušanja 2.
• Upper respiratory tract infection
• okužbe zgornjih dihal,
† For patients rolled over from Trials 1 and 2 (lumacaftor/ivacaftor-to-lumacaftor/ivacaftor group) total exposure was up to 120 weeks.
† Pri bolnikih, ki so nadaljevali iz preskušanj 1 in 2 (skupina z lumakaftorjem/ivakaftorjem, ki je nadaljevala z lumakaftorjem/ivakaftorjem), je trajala skupna izpostavljenost do 120 tednov.
Talk to your doctor before taking Orkambi if you have been told you have liver or kidney disease as your doctor may need to adjust the dose of Orkambi.
Posvetujte se z zdravnikom, preden začnete jemati zdravilo Orkambi, če so vam povedali, da imate bolezen jeter ali ledvic, ker vam bo zdravnik mogoče moral prilagoditi odmerek zdravila Orkambi.
The primary endpoint was improvement in lung function as determined by the mean absolute change from baseline at Day 56 in ppFEV1.
Primarno končno stanje je bilo izboljšanje funkcije pljuč, ugotovljeno s povprečno absolutno spremembo ppFEV1 od izhodišča 56. dne.
The median (range) tmax of lumacaftor is approximately 4.0 hours (2.0; 9.0) in the fed state.
Mediana (razpon) tmax lumakaftorja po obroku hrane je približno 4,0 ure (2,0; 9,0).
In this trial, 10 patients (homozygous for F508del-CFTR mutation) completed dosing with lumacaftor alone 400 mg q12h for 28 days followed by the addition of ivacaftor 250 mg q12h for an additional 28 days, and 25 patients (homozygous or heterozygous for F508del) completed dosing with placebo.
V tem preskušanju je 10 bolnikov (homozigotnih za mutacijo F508del-CFTR) dokončalo odmerjanje samo z lumakaftorjem 400 mg na 12 ur 28 dni, ki mu je sledil dodatek ivakaftorja 250 mg na 12 ur dodatnih 28 dni, 25 bolnikov (homozigotnih ali heterozigotnih za
Read all of this leaflet carefully before your child starts taking this medicine because it contains important information for you.
Preden vaš otrok začne jemati zdravilo, natančno preberite navodilo, ker vsebuje za vas pomembne podatke!
2 to 5 years and weighing less than 14 kg
2 do 5 let, telesna masa manj kot 14 kg
• Ear pain, ear discomfort*
• bolečina v ušesu, neugodje v ušesu*,
Transaminase elevations have been observed more frequently in paediatric patients than in adult patients.
Pri pediatričnih bolnikih so pogosteje opažali zvišanje transminaz kot pri odraslih bolnikih.
For dose adjustments for patients with hepatic impairment see Table 2.
Za prilagoditve odmerkov pri bolnikih z okvaro jeter glejte preglednico 2.
If you have moderate or severe problems with liver function, your doctor may need to reduce the dose of Orkambi as your liver will not clear Orkambi as fast as in people who have normal liver function.
Če imate zmerne ali resne težave z delovanjem jeter, vam bo moral zdravnik mogoče zmanjšati odmerek zdravila Orkambi, ker vaša jetra ne odstranijo zdravila Orkambi iz krvi tako hitro kot pri ljudeh, pri katerih jetra normalno delujejo.
If your child has moderate or severe problems with liver function, your child's doctor may need to reduce the dose of Orkambi as your child's liver will not clear Orkambi as fast as in children who have normal liver function.
Če ima vaš otrok zmerne ali resne težave z delovanjem jeter, mu bo moral otrokov zdravnik mogoče zmanjšati odmerek zdravila Orkambi, ker njegova jetra ne odstranijo zdravila Orkambi iz krvi tako hitro kot pri otrocih, pri katerih jetra normalno delujejo.
Lumacaftor is a CFTR corrector that acts directly on F508del-CFTR to improve its cellular processing and trafficking, thereby increasing the quantity of functional CFTR at the cell surface.
Lumakaftor je popravljalec CFTR, ki deluje neposredno na F508del-CFTR in izboljša njegovo celično procesiranje in transport, s čimer zveča količino funkcionalnega CFTR na površini celice.
However, when initiating lumacaftor/ivacaftor in patients taking strong CYP3A inhibitors, the dose should be adjusted (see sections 4.2 and 4.4).
Kadar pa uvajamo lumakaftor/ivakaftor bolnikom, ki jemljejo močne zaviralce CYP3A, je treba odmerek prilagoditi (glejte poglavji 4.2 in 4.4).
The proportion of patients who experienced a systolic blood pressure value > 140 mmHg or a diastolic blood pressure > 90 mmHg on at least two occasions was 3.4% and 1.5% in patients treated with lumacaftor/ivacaftor, respectively, compared with 1.6% and 0.5% in patients who received placebo (see section 4.4).
Delež bolnikov, ki so imeli ob vsaj dveh priložnostih vrednost sistoličnega krvnega tlaka > 140 mm Hg ali diastoličnega krvnega tlaka > 90 mm Hg , je bil 3,4 % oziroma 1,5 % pri bolnikih, zdravljenih z lumakaftorjem/ivakaftorjem, v primerjavi z 1,6 % oziroma 0,5 % pri bolnikih, ki so prejemali placebo (glejte poglavje 4.4).
White to off-white granules.
zrnca Bela do belkasta zrnca.
Post-marketing cases of liver function decompensation including liver failure leading to death have been reported in CF patients with pre-existing cirrhosis with portal hypertension who were treated with lumacaftor/ivacaftor (see section 4.4).
O primerih dekompenzacije funkcije jeter, vključno z odpovedjo jeter, ki je povzročila smrt, po prihodu zdravila na trg so poročali pri bolnikih s CF, ki se imeli že od prej cirozo s portalno hipertenzijo in so se zdravili z lumakaftorjem/ivakaftorjem (glejte poglavje 4.4).
* For the endpoints listed, absolute change from baseline is the mean absolute change from baseline at Week 24.
* Za navedena končna stanja je absolutna sprememba od izhodišča povprečna absolutna sprememba od izhodišča v 24. tednu.
Some examples of soft foods include puréed fruits, flavoured yogurt, and milk or juice.
Nekateri primeri mehke hrane so sadna kaša, jogurti prijetnega okusa in mleko ali sadni sok.
Lumacaftor and ivacaftor are inhibitors of BCRP.
Lumakaftor in ivakaftor sta zaviralca BCRP.
If the patient's genotype is unknown, an accurate and validated genotyping method should be performed to confirm the presence of the F508del mutation on both alleles of the CFTR gene.
Če bolnikov genotip ni znan, je treba pred začetkom zdravljenja z natančno in validirano metodo genotipizacije potrditi prisotnost mutacije F508del na obeh alelih gena CFTR.
Of the 1108 patients who received any treatment in Trial 1 or Trial 2, 1029 (93%) were dosed and received active treatment (lumacaftor 600 mg once daily/ivacaftor 250 mg q12h or lumacaftor 400 mg q12h/ivacaftor 250 mg q12h) in Trial 3 for up to an additional 96 weeks (i.e. up to a total of 120 weeks).
Od 1108 bolnikov, ki so v preskušanju 1 ali preskušanju 2 prejeli kakršno koli zdravljenje, so 1029 (93 %) določili odmerek in jih aktivno zdravili (lumakaftor 600 mg enkrat na dan/ivakaftor 250 mg/12 h ali lumakaftor 400 mg/12h/ivakaftor 250 mg/12h) v preskušanju 3 do največ dodatnih 96 tednov (tj. skupaj največ 120 tednov).
Potential for lumacaftor/ivacaftor to affect other medicinal products CYP3A substrates
Možnost, da bo lumakaftor/ivakaftor vplival na druga zdravila Substrati CYP3A
If you have poor lung function your doctor may monitor you more closely when you start Orkambi.
Če imate slabše delovanje pljuč, vas bo zdravnik mogoče skrbneje spremljal, ko boste začeli jemati zdravilo
The net effect of lumacaftor/ivacaftor therapy is expected to be strong CYP3A induction.
Pričakovati je, da bo neto učinek zdravljenja z lumakaftorjem/ivakaftorjem močna indukcija CYP3A.
Caution is recommended while using Orkambi in patients with severe renal impairment (creatinine clearance less than or equal to 30 mL/min) or end-stage renal disease (see sections 4.4 and 5.2).
Previdnost je priporočljiva, kadar uporabljamo zdravilo Orkambi pri bolnikih s hudimi okvarami ledvic (očistek kreatinina 30 ml/min ali manj) ali končno ledvično odpovedjo (glejte poglavji 4.4 in 5.2).
2 to 5 years and weighing 14 kg or greater
2 do 5 let, telesna masa 14 kg ali več
Co-administration of lumacaftor/ivacaftor with rifampicin, a strong CYP3A inducer, had minimal effect on the exposure of lumacaftor, but decreased ivacaftor exposure (AUC) by 57%.
Sočasno dajanje lumakaftorja/ivakaftorja z rifampicinom, ki je močan induktor CYP3A, je minimalno vplivalo na izpostavljenost lumakaftorju, a je zmanjšalo izpostavljenost ivakaftorju (AUC) za 57 %.
• Mix the entire contents of a sachet with one teaspoon (5 mL) of age-appropriate soft food or liquid.
• Zmešajte vso vsebino vrečice z eno čajno žličko (5 ml) za starost primerne mehke hrane ali tekočine.
Some examples of age-appropriate soft foods and liquids include puréed fruits, flavoured yogurt, and milk or juice.
Primeri za starost primerne mehke hrane ali tekočine so sadna kaša, jogurti prijetnega okusa in mleko ali sadni sok.
Ivacaftor is a CFTR potentiator that facilitates increased chloride transport by potentiating the channel-open probability (or gating) of the CFTR protein at the cell surface.
Ivakaftor je ojačevalec CFTR, ki olajša zvečan transport kloridov, tako da zveča verjetnost odprtih kanalčkov (“gating”) beljakovine CFTR na površini celice.
Do not give your child a double dose to make up for the forgotten dose.
Ne dajte otroku dvojnega odmerka, če ste mu pozabili dati prejšnji odmerek.
A fat-containing meal or snack should be consumed just before or just after dosing (see section 5.2).
Masten obrok ali prigrizek naj bolnik pojé tik pred odmerkom zdravila ali tik po njem (glejte poglavje 5.2).
Therefore, Orkambi should not be used in children under the age of 2 years.
Orkambi pri otrocih, mlajših od 2 let, ne sme uporabljati.
The typical apparent clearance, CL/F (CV), of lumacaftor was estimated to be 2.38 L/hr (29.4%) for patients with CF.
Tipični navidezni očistek, CL/F (CV), lumakaftorja so pri bolnikih s CF ocenili na 2,38 l/h (29,4 %).
The incidence of transaminase-related adverse reactions was 5.1% and 4.6% in lumacaftor/ivacaftor-treated patients and those who received placebo, respectively.
Incidenca neželenih učinkov, povezanih s transaminazami, je bila 5,1 % oziroma 4,6 % pri bolnikih, zdravljenih z lumakaftorjem/ivakaftorjem, oziroma pri tistih, ki so prejemali placebo.
Key Secondary Endpoints* Absolute change in BMI at Week 24 (kg/m2)
Ključna sekundarna cilja preskušanja* Absolutna sprememba ITM v 24. tednu (kg/m2)
The median (range) tmax of ivacaftor is approximately 4.0 hours (2.0; 6.0) in the fed state.
Mediana (razpon) tmax ivakaftorja po obroku hrane je približno 4,0 ure (2,0; 6,0).
Brilliant blue FCF aluminium lake (E133) Indigo carmine aluminium lake (E132)
briljantno modro FCF (E133) indigotin (E132)
Table 8: Mean (SD) lumacaftor and ivacaftor exposure by age group
Preglednica 8: Povprečna (SD) izpostavljenost lumakaftorju in ivakaftorju po starostnih skupinah
• Meats, oily fish
• meso, mastne ribe;
Absolute change in BMI at Week 24 (kg/m
Absolutna sprememba ITM v 24. tednu (kg/m2)
In the lumacaftor/ivacaftor patients, the treatment difference for absolute change in ppFEV1 from baseline through Week 24 was 2.4 (P = 0.0182).
Pri bolnikih, ki so prejemali lumakaftor/ivakaftor, je bila razlika zaradi zdravljenja v absolutni spremembi ppFEV1 od izhodišča do konca 24. tedna 2,4 (P = 0,0182).
• Antidepressant medicines (used for the treatment of depression) for example: citalopram, escitalopram, sertraline, bupropion
• antidepresivi (uporabljajo se za zdravljenje depresije), na primer: citalopram, escitalopram, sertralin, bupropion;
Following multiple oral dose administration of ivacaftor in combination with lumacaftor, the exposure of ivacaftor generally increased with dose from 150 mg every 12 hours to 250 mg every 12 hours.
Po dajanju večkratnih peroralnih odmerkov ivakaftorja skupaj z lumakaftorjem se je izpostavljenost ivakaftorju na splošno zvečevala z odmerkom od 150 mg na 12 ur do 250 mg na 12 ur.
Mean lumacaftor (SD) AUCss (μg/mL*h)
Povprečna (SD) AUCss lumakaftorja (μg/ml*h)
• Confusion
• zmedenost,
The use of lumacaftor/ivacaftor in organ transplant patients has not been studied.
Uporabe lumakaftorja/ivakaftorja pri bolnikih s presajenimi organi niso proučevali.
Your child's doctor will do some blood tests to check your child's liver before and while she/he is taking Orkambi, particularly during the first year.
Otrokov zdravnik bo naredil nekatere krvne preiskave, s katerimi bo preveril otrokova jetra, preden bo otrok začel jemati zdravilo Orkambi, zlasti v prvem letu.
• Oral hypoglycaemics (used for the management of type 2 diabetes): repaglinide
• peroralni antidiabetiki (uporabljajo se za zdravljenje sladkorne bolezni tipa 2): repaglinid.
Ivacaftor produced concentration-dependent inhibitory effect on hERG (human ether-à-go-go related gene) tail currents, with an IC15 of 5.5 µM, compared to the Cmax (1.5 µM) for ivacaftor at the therapeutic dose for lumacaftor/ivacaftor.
Ivakaftor je povzročil od koncentracije odvisen zaviralni učinek na terminalne tokove hERG (gen ‘human ether-à-go-go related gene') z IC15 5,5 µM, kar je primerljivo s Cmax (1,5 µM) ivakaftorja pri terapevtskem odmerku za lumakaftor/ivakaftor.
It is a medicine used for long-term treatment of cystic fibrosis (CF) in patients aged 6 years and older who have a specific change (called F508del mutation) affecting the gene for a protein called cystic fibrosis transmembrane conductance regulator (CFTR), which plays an important role in regulating the flow of mucus in the lungs.
To je zdravilo, ki se uporablja za dolgoročno zdravljenje cistične fibroze (CF) pri bolnikih, starih 6 let ali več, ki imajo določeno spremembo (imenuje se mutacija F508del), ki vpliva na gen za beljakovino, ki se imenuje regulator transmembranske prevodnosti pri cistični fibrozi (CFTR), ki ima pomembno vlogo pri uravnavanju toka sluzi v pljučih.
Respiratory events such as shortness of breath or chest tightness were seen in patients when starting Orkambi, especially in patients who have poor lung function.
Pri bolnikih, ki začnejo jemati zdravilo Orkambi, so opazili dogodke v zvezi z dihanjem, na primer kratko sapo ali stiskanje v prsih, posebno pri bolnikih, ki so imeli slabše delovanje pljuč.
Co-administration with sensitive CYP3A substrates or CYP3A substrates with a narrow therapeutic index is not recommended (see section 4.5).
Sočasna uporaba z občutljivimi substrati CYP3A ali substrati CYP3A z ozkim terapevtskim oknom se ne priporoča (glejte poglavje 4.5).
↔ LUM, IVA ↓ bupropion Due to induction of CYP2B6 by LUM
↔ LUM, IVA ↓ bupropion Zaradi indukcije CYP2B6 z LUM
During Trials 1 and 2, the incidence of respiratory adverse reactions (e.g., chest discomfort, dyspnoea, and respiration abnormal) was 26.3% in lumacaftor/ivacaftor-treated patients compared to 17.0% in patients who received placebo.
Med preskušanjima 1 in 2 je bila incidenca respiracijskih neželenih učinkov (npr. neugodje v prsih, dispneja in nenormalno dihanje) pri bolnikih, zdravljenih z lumakaftorjem/ivakaftorjem, 26,3 %, pri bolnikih, ki so prejemali placebo, pa 17,0 %.
Number of events requiring intravenous antibiotics per patient-year (95% CI) (rate per 48 wks)
Število dogodkov, zaradi katerih so bili potrebni intravenski antibiotiki, na bolnika-leto (95 % IZ) (pogostnost na 48 tednov)
At week 24, the proportion of patients who remained free from pulmonary exacerbations was significantly higher for patients treated with lumacaftor/ivacaftor compared with placebo.
V 24. tednu je bil delež bolnikov, pri katerih se ni pojavilo pljučno poslabšanje, značilno večji pri bolnikih, zdravljenih z lumakaftorjem/ivakaftorjem, kot pri tistih, ki so prejemali placebo.
Orkambi contains two active substances, lumacaftor and ivacaftor.
Zdravilo Orkambi vsebuje dve učinkovini, lumakaftor in ivakaftor.
The exposures are similar between adults and the paediatric population based on population (PK) analyses as presented in Table 8 below:
Izpostavljenosti na podlagi populacijskih farmakokinetičnih (FK) analiz so bile pri pediatrični populaciji podobne kot pri odraslih, kot kaže preglednica 8 v nadaljevanju:
It is a medicine used for long-term treatment of cystic fibrosis (CF) in children aged 2 years and older who have a specific change (called F508del mutation) affecting the gene for a protein called cystic fibrosis transmembrane conductance regulator (CFTR), which plays an important role in regulating the flow of mucus in the lungs.
To je zdravilo, ki se uporablja za dolgoročno zdravljenje cistične fibroze (CF) pri otrocih, starih 2 leti ali več, ki imajo določeno spremembo (imenuje se mutacija F508del), ki vpliva na gen za beljakovino, ki se imenuje regulator transmembranske prevodnosti pri cistični fibrozi (CFTR), ki ima pomembno vlogo pri uravnavanju toka sluzi v pljučih.
Final Report: December 2025
december 2025.
Trial in patients with CF who are heterozygous for the F508del mutation in the CFTR gene Trial 4 was a multicentre, double-blind, randomised, placebo-controlled, Phase 2 trial in 125 patients with CF aged 18 years and older who had a ppFEV1 of 40 to 90, inclusive, and have the F508del mutation on one allele plus a second allele with a mutation predicted to result in the lack of CFTR production or a CFTR that is not responsive to ivacaftor in vitro.
Preskušanje pri bolnikih s CF, ki so heterozigotni za mutacijo F508del na genu CFTR Preskušanje 4 je bilo multicentrično, dvojno slepo, randomizirano preskušanje 2. faze, kontrolirano s placebom, pri 125 bolnikih s CF, starih 18 let ali več, ki so imeli ppFEV1 40 - 90 vključno in mutacijo F508del na enem alelu plus drugi alel z mutacijo, za katero je bilo napovedano, da bo preprečila nastajanje CFTR ali povzročila nastajanje CFTR, ki se in vitro ne odziva na ivakaftor.
Other efficacy variables included relative change from baseline in ppFEV1, absolute change from baseline in BMI, absolute change from baseline in CFQ-R Respiratory Domain the proportion of patients achieving ≥ 5% relative change from baseline in ppFEV1 at Week 24, and the number of pulmonary exacerbations (including those requiring hospitalisation or IV antibiotic therapy) through Week 24.
Druge učinkovitostne spremenljivke so vključevale relativno spremembo ppFEV1 od izhodišča, absolutno spremembo ITM od izhodišča, absolutno spremembo CFQ-R respiracijske domene od izhodišča, delež bolnikov, ki so dosegli ≥ 5 % relativno spremembo ppFEV1 od izhodišča v 24. tednu, in število pljučnih poslabšanj (vključno s tistimi, pri katerih je bila potrebna hospitalizacija ali i.v.
Talk to your child's doctor before taking Orkambi if you have been told your child has liver or kidney disease as the doctor may need to adjust the dose of Orkambi.
Posvetujte se z otrokovim zdravnikom, preden začnete otroku dajati zdravilo Orkambi, če so vam povedali, da ima vaš otrok bolezen jeter ali ledvic, ker bo zdravnik mogoče moral prilagoditi odmerek zdravila Orkambi.