natgillin/translations-raw huggingface dataset:
Den 24. maj 2019 meddelte Mitsubishi Tanabe Pharma GmbH officielt Udvalget for Lægemidler til Mennesker (CHMP), at virksomheden ønsker at trække sin ansøgning tilbage om markedsføringstilladelse for Radicava til behandling af amyotrofisk lateral sklerose (ALS).
On 24 May 2019, Mitsubishi Tanabe Pharma GmbH officially notified the Committee for Medicinal Products for Human Use (CHMP) that it wishes to withdraw its application for a marketing authorisation for Radicava intended for the treatment of amyotrophic lateral sclerosis (ALS).
I betragtning af det klare behov for yderligere dokumentation for Radicavas effektivitet overvejede udvalget muligheden for en betinget godkendelse, som ville give virksomheden mulighed for at fremlægge flere oplysninger på et senere tidspunkt.
Given the clear need for further evidence of Radicava's effectiveness, the Committee considered the possibility of a conditional approval, which would allow the company to provide more data at a later stage.
Når patienter i placebogruppen senere skiftede til Radicava, var der ingen mærkbar effekt.
When patients in the placebo group were later switched to Radicava there was no noticeable effect.
Desuden noterede CHMP sig betydelige forskelle mellem de to grupper, som kan have påvirket de endelige resultater - f.eks. det forhold, at et større antal patienter i Radicava-gruppen havde mindre alvorlige sygdomme.
Furthermore, the CHMP noted important differences between the two groups which could have influenced the final results - such as the fact that a higher number of patients in the Radicava group had less severe disease.
Virksomheden oplyste til CHMP, at tilbagetrækningen ikke vil have nogen betydning for igangværende kliniske undersøgelser.
The company informed the CHMP that the withdrawal does not have any impact on ongoing clinical trials.
CHMP var også bekymret over varigheden af den eventuelt gavnlige virkning af Radicava, og bemærkede, at 24 uger (et skæringspunkt i hovedstudiet) var alt for kort, og at data fra undersøgelsens udvidelsesfase var vanskelige at tolke.
The CHMP was also concerned about the duration of any benefits from Radicava, noting that 24 weeks (a cut-off point in the main study) was too short and that data from the extension phase of the study were difficult to interpret.
Det vides ikke nøjagtigt, hvordan Radicava virker hos patienter med ALS, men det menes at neutralisere iltholdige molekyler, der kaldes "frie radikaler", og som har været koblet sammen med nerveskader hos patienter med ALS.
The exact way Radicava works in patients with ALS is not known but it is thought to neutralise oxygencontaining molecules known as ‘free radicals', which have been linked to nerve damage in patients with ALS.
Virksomheden vil fortsætte sine igangværende programmer med særlig udleveringstilladelse, mens der føres drøftelser med de nationale myndigheder, som allerede har givet tilladelse til anvendelse med særlig udleveringstilladelse.
The company will continue with its ongoing compassionate use programmes pending discussions with national authorities that have already granted approval for compassionate use.
Under evalueringen konsulterede CHMP en gruppe af eksperter på området for at indhente deres synspunkter om resultaterne af undersøgelsen, det foreslåede register og patientpopulationen, som eventuelt kunne behandles med Radicava.
During the evaluation, the CHMP consulted a group of experts in the field to obtain their views on the study results, the proposed registry and the patient population that could potentially benefit from treatment with Radicava.
I sit brev til agenturet om tilbagetrækningen af ansøgningen anførte virksomheden, at tilbagetrækningen skyldtes ændringer i udvalgets foreløbige udtalelse om data fra studiet.
In its letter notifying the Agency of the withdrawal of the application, the company stated that the withdrawal was based on the Committee's provisional opinion on the study data.
ALS er en sygdom i nervesystemet, hvor nervecellerne i hjernen og rygmarven, der styrer de frivillige bevægelser, gradvist forringes.
ALS is a disease of the nervous system, where nerve cells in the brain and spinal cord that control voluntary movement gradually deteriorate, causing loss of muscle function and paralysis.
Radicava forventedes anvendt til behandling af amyotrofisk lateral sklerose (ALS).
Radicava was expected to be used to treat patients with amyotrophic lateral sclerosis (ALS).
På tidspunktet for tilbagetrækningen af ansøgningen havde CHMP visse betænkeligheder og var af den foreløbige opfattelse, at Radicava ikke kunne godkendes.
At the time of the withdrawal, the CHMP had some concerns and was of the provisional opinion that Radicava could not have been approved.
Udvalget bemærkede, at hovedstudiet viste betydelige forbedringer i ALSFRS-R-score hos de patienter, der fik Radicava, sammenlignet med dem, der fik placebo.
The Committee noted that the main study did show significant improvements in ALSFRS-R scores in patients receiving Radicava compared with those receiving placebo.
Radicava blev udpeget som "lægemiddel til sjældne sygdomme" til behandling af ALS den 19. juni 2015.
Radicava was designated an ‘orphan medicine' (a medicine to be used in rare diseases) on 19 June 2015 for ALS.
Det var hensigten, at det skulle markedsføres som en opløsning til infusion (drop i en vene).
It was to be available as a solution for infusion (drip) into a vein.
Virksomheden fremlagde resultater fra et hovedstudie, der omfattede 137 patienter med ALS, som enten fik Radicava eller placebo (en uvirksom behandling).
The company presented results from a main study of 137 patients with ALS who received either Radicava or placebo (a dummy treatment).
Virksomheden foreslog et registerstudie, hvor patienter, der blev behandlet med Radicava, kunne sammenlignes med patienter, der tidligere havde modtaget andre behandlinger mod ALS.
The company proposed a registry study whereby patients treated with Radicava could be compared with patients who received other treatments for ALS in the past.
Yderligere oplysninger om lægemidler til sjældne sygdomme findes her.
Further information on the orphan designation can be found here.
På tidspunktet for tilbagetrækningen var udvalget af den opfattelse, at fordelene ved Radicava ikke opvejede risiciene, da virkningen ikke var blevet påvist.
At the time of the withdrawal, the Committee was of the opinion that, because of lack of proven effectiveness, the benefits of Radicava did not outweigh its risks.
Hvordan virker Radicava?
How does Radicava work?
I studiet anvendte man den standardiserede vurderingsskala "ALS functional rating scale revised" (ALS-R) til at undersøge,
The study looked at how much patients' symptoms changed over 24 weeks, using a standard rating scale known as ‘ALS functional rating scale revised'
Disse tests skal tilbydes uden beregning.
This testing should be provided free of charge.
Medlemsstaterne skal sikre, at alle de betingelser eller begrænsninger med hensyn til sikker og effektiv anvendelse af lægemidlet, som er beskrevet nedenfor, indføres nationalt:
The Member States must ensure that all conditions or restrictions with regard to the safe and effective use of the medicinal product described below are implemented in their national territory:
Patienterne skal forsynes med et patientkort, som dokumenterer det negative resultat.
The patients should receive a patient card that documents the negative result.
Derudover skal IgE-testen gentages, hvis der udvikles symptomer på kaninallergi.
In addition, IgE testing should be repeated if symptoms of rabbit allergy develop.
• Kopier af patientkort, som skal gives til patienter, før de får Ruconest
• Copies of the patient card to be given to patients before they receive Ruconest
• Risikoen for dannelse af anti-C1INH antistoffer og dermed den potentielle risiko for dannelse af neutraliserende antistoffer.
• The risk of formation of anti-C1INH antibodies and therefore the potential risk of formation of neutralising antibodies.
Kun patienter med påvist negative resultater i sådan en test må behandles med Ruconest.
Only patients who have been shown to have negative test results should be treated with Ruconest.
• Inden produktet markedsføres i hver enkelt medlemsstat, skal indehaveren af markedsføringstilladelsen aftale informationsmaterialets indhold og format med den nationale kompetente myndighed.
• Prior to launch of the product in the Member State, the national competent authority shall agree the content and format of the educational material with the Marketing Authorisation Holder.
• Oplysningsmateriale til lægen
• Educational material for the physician.
o Testen for IgE-antistof skal gentages en gang om året eller efter 10 behandlinger, alt efter hvad der indtræffer først.
o IgE testing should be repeated once a year or after 10 treatments, whichever occurs first.
• Nødvendigheden af at informere patienterne om tidlige tegn på overfølsomhedsreaktioner herunder nældefeber, generel urticaria, trykken for brystet, hvæsende vejrtrækning, hypotension og anafylaksi, og at de skal kontakte lægen, hvis disse symptomer opstår.
• The need to inform patients about the early signs of hypersensitivity reactions including hives, generalised urticaria, tightness of the chest, wheezing, hypotension and anaphylaxis, and that they should alert their physician if these symptoms occur.
• Det faktum, at Ruconest er udvundet af mælken fra transgene kaniner og indeholder spor af kaninprotein (værtsrelaterede urenheder - Host Related Impurities, HRI).
• The fact that Ruconest is derived from milk of transgenic rabbits and contains trace of rabbit proteins (Host Related Impurities, HRI).
Informationspakken skal indeholde følgende:
The educational pack should contain the following:
Patientkortet skal indeholde følgende vigtige punkter:
The patient card should contain the following key elements:
• Indehaveren af markedsføringstilladelsen skal ved markedsføringen sikre, at alle læger, som forventes at ordinere Ruconest, har modtaget en informationspakke.
• The Marketing Authorisation Holder (MAH) should ensure that, at launch, all Healthcare Professionals who are expected to prescribe Ruconest are provided with an Educational pack.
o En protokol for udførelse af en hudpriktest med Ruconest og en plan for intravenøs testdosering for patienter med en negativ hudpriktest, inklusive kriterier for fortolkning af resultater, for patienter med kliniske kendetegn på komælksallergi.
o A protocol for performing a skin prick test (SPT) with Ruconest and an intravenous test dosing schedule in patients with a negative skin prick test, including criteria for interpreting results, for patients with clinical features of cow's milk allergy.
• Vigtigheden af, at der monitoreres for kliniske tegn og symptomer på overfølsomhed, og at patienten skal kontakte lægen, hvis sådanne symptomer udvikles under eller efter administration af Ruconest.
• The importance of monitoring for clinical signs and symptoms of hypersensitivity and that patients should alert their doctor if they develop such symptoms during or after receiving Ruconest.
Oplysningsmaterialet til lægen skal indeholde information om følgende vigtige punkter:
The educational material for the prescriber should include information on the following key elements:
• At Ruconest udvindes af mælken fra transgene kaniner og indeholder spor af kaninprotein
• That Ruconest is derived from milk of transgenic rabbits and contains trace of rabbit proteins
• Patienter med klinisk evidens for komælksallergi kan have antistoffer, som krydsreagerer med urenhederne fra kaninmælken i Ruconest.
• That patients with clinical evidence of cow's milk allergy may have antibodies cross reacting with the rabbit milk impurities in Ruconest.
• At patienten er blevet testet negativ for IgE anti-kaninepithel inden for det seneste år.
• That they have been tested negative for IgE anti rabbit (dander) within the last year.
• Patientkortet skal indeholde et felt, hvor patienten kan notere dato og dosis for hver behandling med Ruconest (hver 10. behandling markeres)
• The patient card should include an area where patients can record the date and dose of every treatment by Ruconest (highlighting every tenth treatment)
• Patienterne skal bære kortet på sig og altid vise det til sundhedspersonale, som behandler for akutte anfald af hereditært angioødem.
• That they should be asked to carry the card and always show it to any health care professional treating them for acute attacks of hereditary angioedema.
• At patienten får Ruconest til behandling af akutte anfald af hereditært angioødem
• That they are receiving Ruconest for treatment of acute attack of hereditary angioedema
• En påmindelse om, at testen for IgE anti-kaninepithel skal gentages en gang om året eller efter 10 behandlinger, alt efter hvad der indtræffer først.
• a reminder that IgE anti rabbit (dander) testing should be repeated once a year or after 10 treatments, whichever occurs first.
• Patientkortet skal indeholde et felt, hvor patienten kan notere resultaterne fra den seneste test for IgE anti-kaninepithel og datoen for testen
• The patient card should include an area where patients can record the results of their last IgE anti Rabbit (dander) and the date of the test
• Den potentielle risiko for en immunkompleks medieret type III overfølsomhedsreaktion på grund af dannelsen af antistoffer direkte mod HRI.
• The potential risk of an immune complex-mediated type III hypersensitivity reaction due to the formation of antibodies directed against Host Related Impurities (HRI).
• Ruconest er kontraindiceret for alle patienter med kendt kaninallergi eller med mistanke om kaninallergi eller med positive serum IgE-antistoffer mod kaninepithel på grund af risikoen for alvorlige allergiske reaktioner.
• That Ruconest is contra indicated in all patients with known or suspected rabbit allergy or with positive serum IgE antibodies against rabbit dander due to the risk of major allergic reactions, therefore:
o Før påbegyndelse af behandling med Ruconest skal alle patienter testes for tilstedeværelsen af IgE-antistoffer mod kaninepithel.
o Before initiating treatment with Ruconest all patients should be tested for the presence of IgE antibodies against rabbit epithelium (dander).
Rådgivning om laboratorietestprogrammet vedrørende immunogenicitet til registrering af disse antistoffer for at følge op på mistanke om immunkompleks medieret sygdom og om den procedure, som skal følges for indsamling og fremsendelse af blodprøve til virksomhedens centrale laboratorium.
Advice about the immunogenicity laboratory testing program for detecting these antibodies for following up suspected immune complex-mediated disease, and about the procedure to follow for the collection and shipment of a blood sample to the company's central laboratory.
• Behandling med Ruconest skal påbegyndes under ledelse og overvågning af en læge, som har erfaring med diagnosticering og behandling af hereditært angioødem.
• That Ruconest should be initiated under the guidance and supervision of a physician experienced in the diagnosis and treatment of hereditary angioedema and should be administered by a health care professional.
Rådgivning om laboratorietestprogrammet vedrørende immunogenicitet tilbudt af virksomheden for at følge op på mistanke om udvikling af neutraliserende antistoffer og om den procedure, som skal følges for indsamling og fremsendelse af blodprøve til virksomhedens centrale laboratorium.
Advice about the immunogenicity laboratory testing program for these antibodies provided by the company for following up suspected emergence of neutralising antibodies and information about the procedure to follow for the collection and shipment of a blood sample to the company's central laboratory.
• Produktresume og indlægsseddel for Ruconest
• Summary of Product Characteristics and Patient Information Leaflet for Ruconest
o Der gives information om den korrekte metode, som skal anvendes ved laboratorietest af serum IgE-antistoffer mod kaninepithel.
o Information about the appropriate methodology to be used for laboratory testing of serum IgE antibodies against rabbit epithelium (dander)
Der var tre behandlingsundersøgelser og en forebyggende undersøgelse.
There were three treatment studies and one prevention study.
Europa-Kommissionen udstedte en markedsføringstilladelse med gyldighed i hele Den Europæiske Union for Mycamine den 25. april 2008.
The European Commission granted a marketing authorisation valid throughout the European Union for Mycamine on 25 April 2008.
De fleste af patienterne i disse to undersøgelser var inficeret med human immundefekt virus (HIV).
Most of the patients in these two studies were infected with human immunodeficiency virus (HIV).
Den fuldstændige EPAR for Mycamine findes på agenturets websted: ema.europa.eu/Find medicine/Human medicines/European Public Assessment Reports.
The full EPAR for Mycamine can be found on the Agency's website: ema.europa.eu/Find medicine/Human medicines/European Public Assessment Reports.
Mycamine anvendes til spædbørn, børn og voksne i følgende tilfælde:
Mycamine is used in babies, children and adults in the following situations:
Dosis afhænger af, hvad lægemidlet bruges til og af patients vægt og respons på behandlingen.
The dose depends on what it is being used for, the patient's weight, and the response to treatment.
Mycamine gives en gang dagligt som en infusion af cirka en times varighed.
Mycamine is given once a day as an infusion lasting around one hour.
Det indeholder det aktive stof micafungin.
It contains the active substance micafungin.
Det forklarer, hvordan Udvalget for Lægemidler til Mennesker (CHMP) vurderede lægemidlet og nåede frem til sin udtalelse til fordel for udstedelse af en markedsføringstilladelse og til sine anbefalinger om anvendelsesbetingelserne for Mycamine.
It explains how the Committee for Medicinal Products for Human Use (CHMP) assessed the medicine to reach its opinion in favour of granting a marketing authorisation and its recommendations on the conditions of use for Mycamine.
Dette dokument er et sammendrag af den europæiske offentlige vurderingsrapport (EPAR) for Mycamine.
This is a summary of the European public assessment report (EPAR) for Mycamine.
Det virker ved at påvirke produktionen af en grundlæggende komponent i svampens cellevæg kaldet 1,3-β-D-glucan.
It works by interfering with the production of an essential component of the fungal cell wall called 1,3 β D glucan.
Svampeceller behandlet med Mycamine har ukomplette eller defekte cellevægge, hvilket gør dem skrøbelige og ude af stand til at vokse.
Fungal cells treated with Mycamine have incomplete or defective cell walls, making them fragile and unable to grow.
Tjeklisten minder de ordinerende læger om, hvordan medicinen anvendes sikkert.
This checklist will remind prescribers how to use the medicine safely.
Virksomheden, som fremstiller Mycamine, sikrer, at ordinerende læger i alle medlemsstater modtager en tjekliste før lanceringen af lægemidlet.
The company that makes Mycamine will ensure that prescribers in all Member States receive a checklist before the launch of the medicine.
Mycamine bør kun anvendes efter nøje afvejning af risici og fordele, især hos patienter med eksisterende leverproblemer.
Mycamine should only be used after careful assessment of its risks and benefits, particularly in patients who have existing liver problems.
Behandlingen skal ophøre, hvis der er vedvarende stigninger i leverenzymer.
Treatment should be stopped if there are persistent increases in liver enzymes.
Det primære mål for virkningen var antallet af patienter, som ikke udviklede en svampeinfektion under behandlingen eller i løbet af de efterfølgende fire uger.
The main measure of effectiveness was the number of patients who did not develop a fungal infection during treatment or the following four weeks.
‘Invasiv' betyder, at svampen har spredt sig til væv og blodkar.
‘Invasive' means that the fungus has spread into tissue and blood vessels;
I alle tre undersøgelser var det primære mål for virkningen antallet af patienter, hos hvem behandlingen var vellykket, baseret på bedring i symptomer og udryddelse af svampen ved behandlingens afslutning.
In all three studies, the main measure of effectiveness was the number of patients in whom treatment was successful, based on an improvement in symptoms and eradication of the fungus at the end of treatment.
EPAR - sammendrag for offentligheden micafungin
EPAR summary for the public micafungin
Andre oplysninger om Mycamine:
Other information about Mycamine
Mycamin var lige så effektivt som sammenligningslægemidlet ved behandling af candidiasis.
Mycamine was as effective as the comparator medicines in the treatment of candidiasis.
Hvis du ønsker yderligere oplysninger om behandling med Mycamine, kan du læse indlægssedlen (også en del af denne EPAR) eller kontakte din læge eller dit apotek.
For more information about treatment with Mycamine, read the package leaflet (also part of the EPAR) or contact your doctor or pharmacist.
Behandling med Mycamine bør indledes af en læge, som har erfaring med behandling af svampeinfektioner og efter overvejelse af officiel eller national vejledning i brug af lægemidler mod svampeinfektioner.
Treatment with Mycamine should be initiated by a doctor who has experience managing fungal infections, and after considering official or national guidance on the use of antifungal medicines.
• for at forhindre infektion med Candida hos patienter, som får en knoglemarvstransplantation, eller som forventes at få neutropeni (lavt niveau af neutrofiler, en art hvide blodlegemer) i 10 dage eller mere.
• to prevent infection with Candida in patients who are having a bone marrow transplant or who are expected to have neutropenia (low levels of neutrophils, a type of white blood cell) for 10 days or more.
Virkningen af Mycamine er blevet undersøgt i fire hovedundersøgelser, hvor det blev sammenlignet med andre lægemidler mod svampeinfektioner.
The effectiveness of Mycamine has been studied in four main studies, in which it was compared with other antifungal medicines.
Hvilke foranstaltninger træffes der for at sikre risikofri anvendelse af Mycamine?
What measures are being taken to ensure the safe use of Mycamine?
Da der er observeret leverskader og tumorer hos rotter, som har fået Mycamine i lange perioder, bør patienter monitoreres for leverproblemer under behandling med Mycamine ved hjælp af blodtest.
Because liver damage and tumours have been seen in rats receiving Mycamine for long periods, patients should be monitored for liver problems during Mycamine treatment using blood tests.
• for at behandle invasiv candidiasis (en type svampeinfektion, som forårsages af en gærlignende svamp ved navn Candida).
• to treat invasive candidiasis (a type of fungal infection caused by a yeast-like fungus called
Det aktive stof i Mycamine, micafungin, er et middel mod svamp, som tilhører gruppen ‘echinocandiner'.
The active substance in Mycamine, micafungin, is an antifungal medicine, which belongs to the group ‘echinocandins'.
Mycamine bør ikke anvendes til personer, som kan være overfølsomme (allergiske) over for micafungin, andre echinocandiner eller andre af indholdsstofferne.
Mycamine should not be used in people who may be hypersensitive (allergic) to micafungin, other echinocandins, or any of the other ingredients.