Atidarsagene autotemcel

Orchard Therapeutics Announces Multiple Presentations at ASGCT 2024

Retrieved on: 
tisdag, maj 7, 2024
Patient, Safety, SAN, Poster, Abstract, Kyowa Kirin, Cell, NOD2, Transplant, Hurler syndrome, POC, Skeleton, Gene, Society, Comparison, HSC, Atidarsagene autotemcel, MLD, Therapy, Communications satellite

TOKYO, LONDON and BOSTON, May 07, 2024 (GLOBE NEWSWIRE) -- Orchard Therapeutics, recently acquired by Kyowa Kirin with the goal of accelerating the delivery of new gene therapies to patients around the globe, today announced four oral and four poster presentations from across its hematopoietic stem cell (HSC) gene therapy platform will be featured at the 27th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) taking place May 7-11, 2024, in Baltimore.

Key Points: 
  • TOKYO, LONDON and BOSTON, May 07, 2024 (GLOBE NEWSWIRE) -- Orchard Therapeutics, recently acquired by Kyowa Kirin with the goal of accelerating the delivery of new gene therapies to patients around the globe, today announced four oral and four poster presentations from across its hematopoietic stem cell (HSC) gene therapy platform will be featured at the 27th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) taking place May 7-11, 2024, in Baltimore.
  • In addition, Orchard Therapeutics will give an invited oral presentation highlighting the potential of OTL-104, a pre-clinical HSC gene therapy developed by its in-house research team, to address a severe and treatment refractory form of Crohn’s disease.
  • “Our presentations at ASGCT add to the compendium of evidence supporting the transformative impact and broad applicability of our approach,” said Leslie Meltzer, Ph.D., chief medical officer of Orchard Therapeutics.
  • Title: Non-neurological, Non-Skeletal Outcomes After Autologous Hematopoietic Stem Cell Gene therapy in Hurler Patients: Retrospective Comparison with Allogeneic Hematopoietic Stem Cell Transplantation​
    Date/Time: Friday, May 10 from noon to 1:30 p.m. and from 5:30 to 7:00 p.m.
    Date/Time: Friday, May 10 from noon to 1:30 p.m. and from 5:30 to 7:00 p.m.

Table of decisions of labelling exemption requests falling under article 63 of Directive 2001/83/EC examined by the Quality Review of Documents (QRD) Group

Retrieved on: 
torsdag, april 25, 2024
Artesunate, QRD, Cysteamine, Daunorubicin/cytarabine, Sutimlimab, Authorization, Positive, SFL, Latanoprost, Obiltoxaximab, Mitapivat, European Medicines Agency, Janssen COVID-19 vaccine, Potassium, Ertapenem, Etranacogene dezaparvovec, Request, MVA, Brexucabtagene autoleucel, Ebola, Imipenem/cilastatin/relebactam, Relugolix/estradiol/norethisterone acetate, CHMP, Pfizer–BioNTech COVID-19 vaccine, Pitolisant, Naloxone, Bupivacaine, Atidarsagene autotemcel, Defibrotide, Rilpivirine, Moderna COVID-19 vaccine, Filo, Pertuzumab, Tafasitamab, Trastuzumab, AstraZeneca, Potassium citrate, COVID-19, INN, JCOVDEN, N/A, Alfa, Assessment, Setmelanotide, Casirivimab/imdevimab, Fair

18 April 2024

Key Points: 
    • 18 April 2024
      EMA/934543/2022 Rev.3
      Human Medicines Division

      Table of decisions of labelling exemption requests falling under article 63 of Directive 2001/83/EC examined by the Quality Review of Documents (QRD) Group
      Related information
      Exemptions to labelling and package-leaflet obligations | European Medicines Agency
      Recommendations for the implementation of the exemptions to the labelling and package leaflet obligations in the centralised procedure

      MA status

      Product number

      Product name

      Article 63(1) request (orphan
      products)2

      Outcome 63(1)
      orphan products2

      Article 63(3) request for omission of
      certain particulars

      Outcome 63(3) omission of
      certain particulars

      Outcome date

      Product information

      Authorised

      EMEA/H/C/005352

      Upstaza

      Translation exemption (outer and
      immediate labelling)

      Positive (partial)

      Omission of particulars (immediate labelling)

      Positive (partial)

      07/Mar/24

      Upstaza (eladocagene exuparvovec) - N/A
      Product information

      Authorised

      EMEA/H/C/005269/X/0033

      Kaftrio

      Translation exemption (outer,
      intermediate and immediate
      labelling)

      Positive (partial)

      21/Nov/23

      Kaftrio
      (elexacaftor/tezacaftor/ivacaftor) Product information
      Rezzayo (rezafungin) - Product
      information

      N/A

      Authorised

      EMEA/H/C/005900

      Rezzayo

      Omission of particulars (immediate labelling)

      Positive

      30/Aug/23

      Authorised

      EMEA/H/C/005933

      Catiolanze

      Omission of particulars (immediate labelling)

      Positive

      13/Jul/23

      Catiolanze (latanoprost) - Product
      information

      N/A

      Authorised

      EMEA/H/C/005756

      Apretude

      Omission of particulars (immediate labelling)

      Positive (partial)

      03/Jul/23

      Apretude, (cabotegravir) - Product
      information

      Apretude (cabotegravir) Assessment report

      Authorised

      EMEA/H/C/005985

      Tepkinly

      Positive (partial)

      14/Jun/23

      Tepkinly (epcoritamab) - Product
      information

      Tepkinly (epcoritamab) Assessment report

      Authorised

      EMEA/H/C/005849/X/0003

      Vyvgart

      Positive

      10/Mar/23

      Vyvgart (efgartigimod alfa) - Product Vyvgart (efgartigimod alfa) information
      Assessment report

      Authorised

      EMEA/H/C/005122

      Omvoh

      Omission of particulars (immediate labelling)

      Positive

      04/Jan/23

      Omvoh (mirikizumab) - Product
      information

      Omvoh (mirikizumab) Assessment report

      Authorised

      EMEA/H/C/004258

      Alofisel

      Omission of particulars (outer labelling)

      Positive (temporary)

      17/Nov/22

      Alofisel (darvadstrocel) - Product
      information

      N/A

      Authorised

      EMEA/H/C/005343

      Mvabea

      Omission of particulars (outer labelling)

      Positive (partial)

      Translation exemption (outer and immediate Positive
      labelling)

      26/Oct/22

      N/A

      EMEA/H/C/005337

      Zabdeno

      Omission of particulars (outer labelling)

      Positive (partial)

      Translation exemption (outer and immediate Positive
      labelling)

      26/Oct/22

      Authorised

      EMEA/H/C/005343

      Mvabea

      Omission of particulars (outer labelling)

      Positive (partial)

      Translation exemption (outer and immediate Positive (partial)
      labelling)

      13/Oct/22

      Authorised

      EMEA/H/C/005337

      Zabdeno

      Omission of particulars (outer labelling)

      Positive (partial)

      Translation exemption (outer and immediate Positive (partial)
      labelling)

      13/Oct/22

      Authorised

      EMEA/H/C/005484

      Loargys

      Mvabea (Ebola vaccine (MVA BN Filo
      [recombinant]) - Product
      information
      Zabdeno (Ad26.ZEBOV-GP
      [recombinant]) - Product
      information
      Mvabea (Ebola vaccine (MVA BN Filo
      [recombinant]) - Product
      information
      Zabdeno (Ad26.ZEBOV-GP
      [recombinant]) - Product
      information
      Loargys (pegzilarginase) - Product
      information

      Authorised

      Authorised

      EMEA/H/C/005703

      Pombiliti

      Omission of particulars (immediate labelling)

      Positive

      13/Oct/22

      Pombiliti (cipaglucosidase alfa) Product information

      Pombiliti (cipaglucosidase alfa) Assessment report

      Authorised

      EMEA/H/C/005695

      Opfolda

      Omission of particulars (immediate labelling)

      Positive

      13/Oct/22

      Opfolda (miglustat) - Product
      information

      N/A

      Authorised

      EMEA/H/C/006016

      Imjudo

      Omission of particulars (immediate labelling)

      Positive

      13/Oct/22

      IMJUDO - Product information

      Imjudo - Assessment report

      Authorised

      EMEA/H/C/004650

      Tremelimumab
      AstraZeneca

      Omission of particulars (immediate labelling)

      Positive

      13/Oct/22

      Tremelimumab AstraZeneca Product information

      Tremelimumab AstraZeneca Assessment report

      Authorised

      EMEA/H/C/005776

      Enjaymo

      13/Sep/22

      Enjaymo (Sutimlimab) - Product
      information

      N/A

      Authorised

      EMEA/H/C/005035

      Filsuvez

      Authorised

      EMEA/H/C/004827

      Hemgenix

      Authorised

      EMEA/H/C/005751

      Columvi

      Translation exemption (outer,
      immediate labelling and package
      leaflet)
      Translation exemption (outer
      labelling)

      Translation exemption (outer,
      immediate labelling and package
      leaflet)

      Article 63(3) request for translation
      exemption

      Positive (partial)

      13/Oct/22

      Translation exemption (outer, immediate
      labelling and package leaflet)

      Translation exemption (outer,
      immediate labelling and package
      leaflet)

      Outcome 63(3) request for
      translation exemption

      Positive (partial)

      Positive

      Assessment report

      N/A

      N/A

      N/A

      N/A

      Loargys (pegzilarginase) Assessment report

      Omission of particulars (immediate labelling)

      Positive

      08/Aug/22

      Filsuvez (birch bark extract) Product information

      N/A

      Omission of particulars (outer labelling)

      Negative

      14/Jun/22

      Hemgenix - Product information

      Hemgenix - Assessment report

      Omission of particulars (immediate labelling)

      Positive

      14/Jun/22

      Columvi, INN-glofitamab
      (europa.eu)

      N/A

      Authorised

      EMEA/H/C/005848

      Pemetrexed Baxter

      Omission of particulars (immediate labelling)

      Positive

      14/Jun/22

      Pemetrexed Baxter - Product
      information

      Pemetrexed Baxter - Assessment
      report

      Authorised

      EMEA/H/C/005246/X/0002

      Byfavo

      Omission of particulars (immediate labelling)

      Negative

      14/Jun/22

      Byfavo - Product information

      N/A

      Authorised

      EMEA/H/C/005815

      Ertapenem SUN

      Omission of particulars (immediate labelling)

      Positive

      11/May/22

      Ertapenem SUN (ertapenem) Product information

      Ertapenem SUN (ertapenem) Assessment report

      Authorised

      EMEA/H/C/004850

      Xenpozyme

      12/Apr/22

      Xenpozyme (olipudase alfa) Product information

      Xenpozyme (olipudase alfa) Assessment report

      Authorised

      EMEA/H/C/005769

      Herwenda

      10/Mar/22

      Herwenda (trastuzumab) - Product
      information

      N/A

      Authorised

      EMEA/H/C/005540

      Pyrukynd

      10/Mar/22

      Pyrukynd - Product information

      N/A

      Authorised

      EMEA/H/C/005680

      Lunsumio

      10/Mar/22

      Lunsumio (mosunetuzumab) Product information

      N/A

      Authorised

      EMA/H/C/004577

      Ebvallo

      Translation exemption (outer,
      immediate labelling and package
      leaflet)

      Positive (partial)

      10/Mar/22

      Ebvallo - Product information

      Ebvallo - Assessment report

      Authorised

      EMEA/H/C/004929

      Kimmtrak

      Translation exemption (outer and
      immediate labelling)

      Positive

      16/Nov/21

      Kimmtrak (tebentafusp) - Product
      information

      Kimmtrak (tebentafusp) Assessment report

      Authorised

      EMEA/H/C/005378

      Nulibry

      Translation exemption (outer and
      immediate labelling)

      Positive (partial)

      16/Nov/21

      Nulibry - Product information

      N/A

      Authorised

      EMEA/H/C/005849

      Vyvgart

      Omission of particulars (outer and immediate Positive (partial)
      labelling)

      16/Nov/21

      Vyvgart (efgartigimod alfa) - Product Vyvgart (efgartigimod alfa) information
      Assessment report

      Authorised

      EMEA/H/C/005973

      Paxlovid

      Omission of particulars (outer labelling)

      Positive

      Translation exemption (outer, immediate
      labelling and package leaflet)

      Positive (partial)

      16/Nov/21

      Paxlovid (nirmatrelvir + ritonavir) Product information

      Authorised

      EMEA/H/C/005788

      Evusheld

      Omission of particulars (outer labelling)

      Positive

      Translation exemption (outer, immediate
      labelling and package leaflet)

      Positive (partial)

      13/Oct/21

      Evusheld (ixagevimab, cilgavimab) - N/A
      Product information

      Authorised

      EMEA/H/C/005553

      Aspaveli

      Omission of particulars (immediate labelling)

      Positive

      08/Oct/21

      Aspaveli (pegcetacoplan) - Product
      information

      Aspaveli (pegcetacoplan) Assessment report

      Authorised

      EMEA/H/C/005814

      Ronapreve

      Omission of particulars (immediate labelling)

      Positive

      24/Sep/21

      Ronapreve (casirivimab and
      imdevimab) - Produt information

      Ronapreve (casirivimab and
      imdevimab) - Assessment report

      Authorised

      EMEA/H/C/005550

      Artesunate Amivas

      14/Sep/21

      Artesunate Amivas (artesunate) Product information

      Artesunate Amivas (Artesunate) Assessment report

      Authorised

      EMEA/H/C/005854

      Regkirona

      Omission of particulars (outer and immediate Positive
      labelling)

      Translation exemption (outer, immediate
      labelling and package leaflet)

      Positive (partial)

      28/Jul/21

      Regkirona (regdanvimab) - Product
      information

      Regkirona (regdanvimab) Assessment report

      Authorised

      EMEA/H/C/005676

      Xevudy

      Omission of particulars (outer labelling)

      Positive

      Translation exemption (outer, immediate
      labelling and package leaflet)

      Positive (partial)

      28/Jul/21

      Xevudy (sotrovimab) - Product
      information

      Xevudy (sotrovimab) Assessment report

      Authorised

      EMEA/H/C/005182

      Trodelvy

      Omission of particulars (immediate labelling)

      Positive

      17/Jun/21

      Trodelvy (sacituzumab govitecan) Product information

      Trodelvy (sacituzumab govitecan)
      - Assessment report

      Authorised

      EMEA/H/C/005681

      Pepaxti

      Omission of particulars (immediate labelling)

      Positive

      17/Jun/21

      Pepaxti (melphalan flufenamide) Product information

      N/A

      Authorised

      EMEA/H/C/005035

      Filsuvez

      Translation exemption (outer,
      intermediate, immediate labelling
      and package leaflet)

      Positive (partial)

      17/Jun/21

      Filsuvez (birch bark extract) Product information

      N/A

      Authorised

      EMEA/H/C/005095

      Carvykti

      Translation exemption (outer and
      immediate labelling)

      Positive

      17/Jun/21

      Carvykti (Ciltacaptagene autoleucel) - Carvykti (ciltacabtagene
      Product information
      autoleucel) - Assessment report

      Authorised

      EMEA/H/C/005467

      Voraxaze

      Translation exemption (outer,
      intermediate, immediate labelling
      and package leaflet)

      Positive (partial)

      17/Jun/21

      Voraxaze (glucarpidase) - Product
      information

      Authorised

      EMEA/H/C/005501

      Nexviadyme

      Omission of particulars (immediate labelling)

      17/Jun/21

      Nexviadyme (avalglucosidase alfa) - Nexviadyme (avalglucosidase
      Product information
      alfa) - Assessment report

      Authorised

      EMEA/H/C/004662

      Abecma

      Omission of particulars (outer and immediate Positive (partial)
      labelling)

      03/Jun/21

      Abecma (idecabtagene vicleucel) Product information

      Abecma (idecabtagene vicleucel) Assessment report

      Authorised

      EMEA/H/C/004275/II/0021

      Crysvita

      Positive

      06/May/21

      Crysvita (burosumab) - Product
      information

      N/A

      Authorised

      EMEA/H/C/005475

      Voxzogo

      Positive (partial)

      04/Mar/21

      Voxzogo (vosoritide) - Product
      information

      Voxzogo (vosoritide) - CHMP
      assessment report

      Authorised

      EMEA/H/C/005737

      Jcovden

      Omission of particulars (outer, immediate
      labelling and package leaflet)

      Positive

      01/Mar/21

      Authorised

      EMEA/H/C/005327

      Abevmy

      Omission of particulars (immediate labelling)

      Positive

      JCOVDEN [COVID-19 vaccine
      (Ad26.COV2-S [recombinant]) Product information
      Abevmy (bevacizumab) - Product
      information

      JCOVDEN [COVID-19 Vaccine
      Janssen (Ad26.COV2-S,
      recombinant)] - Assessment
      Abevmy (bevacizumab) Assessment report

      Translation exemption (outer,
      immediate labelling and package
      leaflet)

      Positive

      Omission of particulars (immediate labelling)

      Translation exemption (outer,
      intermediate and immediate
      labelling)

      Translation exemption (outer,
      immediate labelling and package
      leaflet)

      Translation exemption (outer,
      immediate labelling and package
      leaflet)
      Translation exemption (outer and
      immediate labelling)

      Positive

      Positive (partial)

      Omission of particulars (immediate labelling)

      Positive

      Omission of particulars (immediate labelling)

      Positive (partial)

      Translation exemption (outer labelling)

      Positive

      Positive (partial)

      Positive

      Translation exemption (outer, immediate
      labelling and package leaflet)

      Positive

      13/Jan/21

      Paxlovid (nimatrelvir + ritonavir) Assessment report

      Voraxaze (glucarpidase) Assessment report

      Authorised

      EMEA/H/C/005791

      Spikevax

      Omission of particulars (outer, immediate
      labelling and package leaflet)

      Translation exemption (outer, immediate
      labelling and package leaflet)

      Positive

      01/Jan/21

      Spikevax - Product information

      Spikevax - Assessment report

      Authorised

      EMEA/H/C/005735

      Comirnaty

      Omission of particulars (outer and immediate Positive
      labelling)

      Translation exemption (outer and immediate
      labelling)

      Positive

      17/Dec/20

      Comirnaty (tozinameran,
      tozinameran/riltozinameran,
      tozinameran/famtozinameran) Alymsys (bevacizumab) - Product
      information

      Comirnaty (tozinameran,
      tozinameran/riltozinamearn,
      tozinamearn/famtozinameran) N/A

      Authorised

      EMEA/H/C/005286

      Alymsis

      Omission of particulars (immediate labelling)

      Positive

      20/Oct/20

      Authorised

      EMEA/H/C/005556

      Oyavas

      Omission of particulars (immediate labelling)

      Positive

      20/Oct/20

      Oyavas (bevacizumab) - Product
      information

      N/A

      Authorised

      EMEA/H/C/005145

      Evrysdi

      Omission of particulars (immediate labelling)

      Negative

      20/Oct/20

      Evrysdy (risdiplam) - Product
      information

      Evrysdi (risdiplam) - Assessment
      report

      Authorised

      EMEA/H/C/005089

      Imcivree

      20/Oct/20

      Imcivree (setmelanotide) - Product
      information

      Imcivree (setmelanotide) Assessment report

      Authorised

      EMEA/H/C/004662

      Abecma

      Omission of particulars (outer and
      intermediate labelling)

      Positive (partial)

      20/Oct/20

      Abecma (idecabtagene vicleucel) Product information

      Abecma (decabtagene vicleucel) Assessment report

      Authorised

      EMEA/H/C/004731

      Breyanzi

      Omission of particulars (outer and
      intermediate labelling)

      Positive (partial)

      20/Oct/20

      Breyanzi (lisocabtagene maraleucel) - N/A
      Product information

      Authorised

      EMEA/H/C/005386

      Phesgo

      Omission of particulars (immediate labelling)

      Positive

      13/Oct/20

      EMEA/H/C/005102

      Tecartus

      Omission of particulars (immediate labelling)

      Positive

      05/Oct/20

      Authorised

      EMEA/H/C/005675

      Vaxzevria

      Omission of particulars (outer and immediate Positive
      labelling)

      Phesgo (pertuzumab/trastuzumab) - Phesgo (pertuzumab /
      Product information
      trastuzumab) - Assessment
      report
      Tecartus (autologous anti-CD19Tecartus (autologous anti-CD19transduced CD3+ cells) - Product
      transduced CD3+ cells) information
      Assessment report
      Vaxzevria - Product information
      Vaxzevria - Assessment report

      Authorised

      Authorised

      EMEA/H/C/005246

      Byfavo

      Omission of particulars (immediate labelling)

      Positive

      16/Jun/20

      Byfavo (remimazolam) - Product
      information

      Byfavo (remimazolam) Assessment report

      Authorised

      EMEA/H/C/004586

      Exparel liposomal

      Omission of particulars (immediate labelling)

      Positive

      16/Jun/20

      Exparel liposomal (bupivacaine) Product information

      Exparel liposomal (bupivacaine) Assessment report

      Authorised

      EMEA/H/C/004954/X/0004/G

      Ultomiris

      Omission of particulars (immediate labelling)

      Positive

      16/Jun/20

      Ultomiris (ravulizumab) - Product
      information

      N/A

      Authorised

      EMEA/H/C/004976

      Vocabria

      Omission of particulars (immediate labelling)

      Positive

      16/Jun/20

      Vocabria (cabotegravir) - Product
      information

      N/A

      Authorised

      EMEA/H/C/005060

      Rekambys

      Omission of particulars (immediate labelling)

      Positive

      16/Jun/20

      Rekambys (rilpivirine) - Product
      information

      N/A

      Authorised

      EMEA/H/C/005436

      Minjuvi

      Omission of particulars (immediate labelling)

      Positive

      16/Jun/20

      Minjuvi (tafasitamab) - Product
      information

      N/A

      Authorised

      EMEA/H/C/005681

      Pepaxti

      Omission of particulars (immediate labelling)

      Positive

      16/Jun/20

      Pepaxti (melphalan flufenamide) Product information

      N/A

      Authorised

      EMEA/H/C/004379

      Amglidia

      Translation exemption (outer,
      immediate labelling and package
      leaflet)

      Negative

      16/Jun/20

      Amglidia (glibenclamide) - Product
      information

      N/A

      Authorised

      EMEA/H/C/005271

      Zokinvy

      Translation exemption (outer,
      immediate labelling and package
      leaflet)

      Positive

      16/Jun/20

      Zokinvy (lonafarnib) - Product
      information

      N/A

      Authorised

      EMEA/H/C/002393

      Defitelio

      Translation exemption (outer,
      immediate labelling and package
      leaflet)

      Positive (partial)

      16/Jun/20

      Defitelio (defibrotide) - Product
      information

      N/A

      Authorised

      EMEA/H/C/005622

      Veklury

      15/May/20

      Veklury (remdesivir) - Product
      information

      Veklury (remdesivir) Assessment report

      Authorised

      EMEA/H/C/005269

      Kaftrio

      Translation exemption (outer,
      intermediate, immediate labelling
      and package leaflet)

      Positive (partial)

      22/May/20

      Kaftrio
      (elexacaftor/tezacaftor/ivacaftor) Product information

      N/A

      Authorised

      EMEA/H/C/005167

      Dovprela

      Translation exemption (outer and
      immediate labelling)

      Positive

      27/Mar/20

      Dovprela (pretomanid) - Product
      information

      Dovprela (pretomanid) Assessment report

      Authorised

      EMEA/H/C/004077/X/0032

      Darzalex

      20/Mar/20

      Darzalex (daratumumab) - Product
      information

      N/A

      Authorised

      EMEA/H/C/002614

      Sirturo

      Translation exemption (outer and
      immediate labelling)

      Positive

      03/Mar/20

      Sirturo (bedaquiline) - Product
      information

      N/A

      Authorised

      EMEA/H/C/005321

      Libmeldy

      Translation exemption (outer,
      intermediate, immediate labelling
      and lot information sheet)

      Positive

      03/Mar/20

      Libmeldy (atidarsagene autotemcel) - Libmeldy (atidarsagene
      Product information
      autotemcel) - Assessment report

      Authorised

      EMEA/H/C/004849

      Idefirix

      Translation exemption (outer and
      immediate labelling)

      Positive

      03/Mar/20

      Idefirix (imlifidase) - Product
      information

      Idefirix (imlifidase) - Assessment
      report

      Authorised

      EMEA/H/C/000829/X/0122/G

      Pradaxa

      03/Mar/20

      Pradaxa (dabigatran etexilate) Product information

      Pradaxa (dabigatran etexilate) Assessment report

      Translation exemption (outer and
      immediate labelling)

      Positive

      Positive

      Omission of particulars (immediate labelling)

      Omission of particulars (immediate labelling)

      Omission of particulars (immediate labelling)

      Omission of particulars (immediate labelling)

      Positive

      Positive

      Positive

      Positive

      Translation exemption (outer and immediate
      labelling)

      Positive

      07/Jul/20

      Authorised

      EMEA/H/C/003855

      Coagadex

      Translation exemption (outer and
      immediate labelling)

      Positive

      Authorised

      EMEA/H/C/005352

      Upstaza

      Translation exemption (outer and
      immediate labelling)

      Positive (partial)

      Authorised

      EMEA/H/C/003855

      Coagadex

      Translation exemption (outer and
      immediate labelling)

      Positive

      Authorised

      EMEA/H/C/004829

      Fetcroja

      Authorised

      EMEA/H/C/005169

      Obiltoxaximab SFL

      Translation exemption (outer
      labelling and package leaflet)

      Authorised

      EMEA/H/C/005830

      Roctavian

      Translation exemption (outer
      labelling and package leaflet)

      Authorised

      EMEA/H/C/005205

      Zynrelef

      Omission of particulars (immediate labelling)

      Positive

      08/Oct/19

      Authorised

      EMEA/H/C/003860/N/0027

      Nucala

      Omission of particulars (immediate labelling)

      Positive

      08/Oct/19

      Nucala (mepolizumab) - Product
      information

      N/A

      Authorised

      EMEA/H/C/004808

      Recarbrio

      Omission of particulars (immediate labelling)

      Positive

      08/Oct/19

      Authorised

      EMEA/H/C/004750

      Zolgensma

      Translation exemption (outer and
      immediate labelling)

      Positive (partial)

      26/Mar/19

      Recarbrio
      (imipenem/cilastatin/relebactam) Product information
      Zolgensma (Onasemnogene
      abeparvovec) - Product information

      Authorised

      EMEA/H/C/005407

      Sibnayal

      Translation exemption (outer and
      immediate labelling)

      Positive

      26/Mar/19

      Sibnayal (potassium citrate and
      potassium hydrogen carbonate) Product information

      Recarbrio
      (imipenem/cilastatin/relebactam) Assessment report
      Zolgensma (Onasemnogene
      abeparvovec) - Assessment
      report
      Sibnayal (potassium citrate and
      potassium hydrogen carbonate) Assessment report

      Authorised

      EMEA/H/C/005830

      Roctavian

      Translation exemption (outer and
      immediate labelling)

      Positive (partial)

      26/Mar/19

      Authorised

      EMEA/H/C/004282

      Vyxeos liposomal

      Translation exemption (outer,
      immediate labelling and package
      leaflet)

      Positive (partial)

      09/Oct/18

      ROCTAVIAN (valoctocogene
      Roctavian (valoctocogene
      roxaparvovec) - Product information roxaparvovec) - Assessment
      report
      Vyxeos liposomal
      N/A
      (daunorubicin/cytarabine) - Product
      information

      Authorised

      EMEA/H/C/005407

      Sibnayal

      Translation exemption (outer and
      immediate labelling)

      Positive

      09/Oct/18

      Sibnayal (potassium citrate and
      potassium hydrogen carbonate) Product information

      Sibnayal (potassium citrate and
      potassium hydrogen carbonate) Assessment report

      Authorised

      EMEA/H/C/004275

      Crysvita

      Translation exemption (outer,
      immediate labelling and package
      leaflet)

      Positive (partial)

      Oct/17

      Crysvita (burosumab) - Product
      information

      Crysvita (busorumab) Assessment report

      Omission of particulars (immediate labelling)

      Positive (partial)

      03/Mar/20

      Coagadex (human coagulation factor N/A
      X) - Product information

      03/Mar/20

      Upstaza (eladocagene exuparvovec) - Upstaza (eladocagene
      Product information
      exuparvovec) - Assessment
      report
      Coagadex (human coagulation factor N/A
      X) - Product information

      13/Jan/20

      Omission of particulars (immediate labelling)

      19/Nov/19

      Fetcroja (cefiderocol) - Product
      information

      Positive (partial)

      08/Oct/19

      Positive (partial)

      08/Oct/19

      Obiltoxaximab SFL (obiltoxaximab) - Obiltoxaximab SFL
      Product information
      (obiltoxaximab) - Assessment
      report
      ROCTAVIAN (valoctocogene
      Roctavian (valoctocogene
      roxaparvovec) - Product information roxaparvovec) - Assessment
      report
      Zynrelef (bupivacaine/meloxicam) - Zynrelef (bupivacaine/meloxicam)
      Product information
      - Assessment report

      Omission of particulars (immediate labelling)

      Omission of particulars (outer labelling)

      Positive

      Negative

      Positive

      Fetcroja (cefiderocol) Assessment report

      18 April 2024
      EMA/934543/2022 Rev.3
      Human Medicines Division

      Table of decisions of labelling exemption requested with the aim to create multilingual packages and examined by the Quality Review of Documents (QRD) Group
      Related information
      Exemptions to labelling and package-leaflet obligations | European Medicines Agency
      Recommendations for the implementation of the exemptions to the labelling and package leaflet obligations in the centralised procedure

      MA status

      Product number

      Product name

      Request for omission of particulars

      Outcome request for Request for translation exemption
      omission of
      particulars

      Authorised

      EMEA/H/C/005107/N/0022

      Roclanda

      Omission of particulars (immediate labelling)

      Positive

      Authorised

      EMEA/H/C/004452/X/0028

      Erleada

      Omission of particulars (immediate labelling)

      Authorised

      EMEA/H/C/000697

      Suboxone

      Authorised

      EMEA/H/C/005843

      Authorised

      Product information

      Assessment report

      21/Nov/23

      Roclanda (netarsudil
      mesylate/latanoprost) - Product
      information

      N/A

      Positive

      10/Mar/23

      Erleada, INN - apalutamide
      (europa.eu)

      Erleada (apalutamide) Assessment report

      Omission of particulars (immediate labelling)

      Positive

      13/Oct/22

      Suboxone (naloxone) - Product
      information

      N/A

      Opzelura

      Omission of particulars (immediate labelling)

      Positive (partial)

      13/Oct/22

      Opzelura (ruxolitinib) - Product
      information

      Opzelura (Ruxolitinib ) Assessment report

      EMEA/H/C/005361

      Akeega

      Omission of particulars (immediate labelling)

      Positive

      14/Jun/22

      Akeega (niraparib/abiraterone
      acetate) - Product information

      N/A

      Authorised

      EMEA/H/C/002465/X/0035

      Procysbi

      Omission of particulars (immediate labelling)

      Positive

      16/Nov/21

      Procysbi (mercaptamine
      bitartrate) - Product information

      N/A

      Authorised

      EMEA/H/C/005367

      Skytrofa

      Omission of particulars (immediate labelling)

      Positive (partial)

      17/Jun/21

      Skytrofa (lonapegsomatropin) Product information

      Skytrofa (lonapegsomatropin) Assessment report

      Authorised

      EMEA/H/C/000610/X/0063

      Noxafil

      Omission of particulars (immediate labelling)

      Positive

      17/Jun/21

      Noxafil (posaconazole) - Product
      information

      N/A

      Authorised

      EMEA/H/C/005267

      Ryeqo

      Omission of particulars (immediate labelling)

      Positive

      04/Mar/21

      Ryeqo
      N/A
      (Relugolix/Estradiol/Norethistero
      ne acetate) - Product information

      Authorised

      EMEA/H/C/005117

      Ozawade

      Omission of particulars (outer and immediate labelling)

      Positive

      04/Mar/21

      Ozawade (Pitolisant) - Product
      information

      Ozawade (Pitolisant) Assessment report

      Authorised

      EMEA/H/C/005208

      Ayvakyt

      Omission of particulars (immediate labelling)

      Negative

      20/Jun/20

      AYVAKYT (avapritinib) - Product
      information

      N/A

      Authorised

      EMEA/H/C/004835

      Zeposia

      Omission of particulars (immediate labelling)

      Positive (partial)

      08/Oct/19

      Zeposia (ozanimod) - Product
      information

      Zeposia (ozanimod) - Public
      assessment report

      Authorised

      EMEA/H/C/005243

      Lacosamide UCB

      Omission of particulars (immediate labelling)

      Positive

      08/Oct/19

      Lacosamide UCB (lacosamide) Product information

      N/A

      Authorised

      EMEA/H/C/004452

      Erleada

      Omission of particulars (immediate labelling)

      Positive

      09/Oct/18

      Erleada (apalutamide) - Product
      information

      Erleada (apalutamide) - Public
      assessment report

      Translation exemption (immediate labelling)

      Outcome translation Outcome date
      exemption

      Positive

FDA Approves First Gene Therapy for Children with Metachromatic Leukodystrophy

Retrieved on: 
måndag, mars 18, 2024
Encephalitis, Locomotion, Brain, B cell, Bone marrow, Cell, Dietary supplement, Nervous system, Risk, Patient, Public, Urinary tract infection, Animal, Metachromasia, DSM-IV codes, Peripheral nervous system, Viral, Human services, Hope, Death, Quality of life, Atidarsagene autotemcel, Rash, Therapy, Metachromatic leukodystrophy, Natural history, Fever, MLD, Heated tobacco product, Safety, Disability studies, Cosmetics, Health, Vaccine, Food, Swelling, FDA, Liver, Multimedia, ARSA, Pharmaceutical industry

Metachromatic leukodystrophy is a debilitating, rare genetic disease affecting the brain and nervous system.

Key Points: 
  • Metachromatic leukodystrophy is a debilitating, rare genetic disease affecting the brain and nervous system.
  • The stem cells are collected from the patient and modified by adding a functional copy of the ARSA gene.
  • In children with MLD, treatment with Lenmeldy significantly reduced the risk of severe motor impairment or death compared with untreated children.
  • Eighty five percent of the children treated had normal language and performance IQ scores, which has not been reported in untreated children.

Orchard Therapeutics Receives Swissmedic Approval for Libmeldy in Early-onset MLD

Retrieved on: 
måndag, december 11, 2023
Nerve, MLD, Traffic barrier, European Commission, Food, Agency, Priority review, Health care, Death, FDA, PDUFA, Civil service commission, HSC, MHRA, Therapy, Busulfan, ARSA, Mutation, Atidarsagene autotemcel, Genome, Central nervous system, Patient, Health, Pharmaceutical industry

BOSTON and LONDON, Dec. 11, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced the Swiss Agency for Therapeutic Products (Swissmedic) has approved Libmeldy® (atidarsagene autotemcel), a hematopoietic stem cell (HSC) gene therapy, for the treatment of early-onset metachromatic leukodystrophy (MLD).

Key Points: 
  • BOSTON and LONDON, Dec. 11, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced the Swiss Agency for Therapeutic Products (Swissmedic) has approved Libmeldy® (atidarsagene autotemcel), a hematopoietic stem cell (HSC) gene therapy, for the treatment of early-onset metachromatic leukodystrophy (MLD).
  • Libmeldy aims to correct the underlying genetic cause of MLD by inserting a working copy of the ARSA gene into the genome of a patients’ own HSCs.
  • With more than a cumulative 250 patient-years of follow-up, Libmeldy was generally well-tolerated, with no treatment-related serious adverse events or deaths.
  • “Today’s approval of Libmeldy in Switzerland opens up tremendous new possibilities for eligible children with MLD who previously had no approved treatment options beyond supportive care,” said Leslie Meltzer, Ph.D., chief medical officer of Orchard Therapeutics.

Orchard Therapeutics Reports Third Quarter 2023 Financial Results and Highlights Recent Business Accomplishments

Retrieved on: 
måndag, november 13, 2023
Infant, Acquisition, HSC, Investment, Patient, Cell, ADS, ESGCT, U.S. FDA, FTD, Marketing, ICER, Diagnosis, R, Congress, Priority review, CVR, Food, Public, MLD, Abstract, Public health, Biomarker, MPS, Therapy, BLA, Gene, Health, HAE, Frontotemporal dementia, Hurler, Research, Disease, FDA, American depositary receipt, File, Hereditary angioedema, CTA, PDUFA, Society, Kyowa Kirin, Pharmaceutical industry, Security (finance), Fine chemical, Mobile phone, Vaccine, Dietary supplement, Cryptocurrency, Metabolism, Orchard, Atidarsagene autotemcel

Biologics License Application for OTL-200 in MLD accepted by U.S. FDA under Priority Review; PDUFA date set for March 18, 2024

Key Points: 
  • Biologics License Application for OTL-200 in MLD accepted by U.S. FDA under Priority Review; PDUFA date set for March 18, 2024
    BOSTON and LONDON, Nov. 13, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced several business accomplishments along with its financial results for the quarter ended September 30, 2023.
  • Following the completion of the acquisition, Orchard Therapeutics will become a wholly-owned subsidiary of Kyowa Kirin.
  • The company had approximately 227.3 million ordinary shares, equivalent to 22.7 million American Depositary Shares, outstanding as of September 30, 2023.
  • As a result of the anticipated acquisition by Kyowa Kirin, Orchard Therapeutics has removed its financial guidance around Libmeldy revenue and anticipated cash runway.

Kyowa Kirin to Acquire Orchard Therapeutics

Retrieved on: 
torsdag, oktober 5, 2023
Acquisition, HSC, Therapy, Stem cell, Vision, Health, Research, Priority review, Morrison, Sanfilippo syndrome, Food and Drug Administration Amendments Act of 2007, DSM-IV codes, Kyowa Kirin, Frontotemporal dementia, EC, JST, MLD, Civil service commission, Health care, Food, European Commission, Biotechnology, Transaction, FDA, CVR, FTD, MPS, Guggenheim Partners, Kirin, Sanfilippo, TSE, ADS, MHRA, Morrison & Foerster, Science, PDUFA, Life, Patient, Communications satellite, Pharmaceutical industry, Vaccine, Security (finance), Orchard, Atidarsagene autotemcel

TOKYO and LONDON and BOSTON, Oct. 05, 2023 (GLOBE NEWSWIRE) -- Kyowa Kirin Co., Ltd. (Kyowa Kirin, TSE: 4151), a Japan-based global specialty pharmaceutical company (J-GSP) creating innovative medical solutions utilizing the latest biotechnology, and Orchard Therapeutics plc (Orchard Therapeutics, Nasdaq: ORTX), a global gene therapy leader, today announced the companies have entered into a definitive agreement under which Kyowa Kirin will acquire Orchard Therapeutics for $16.00 per American Depositary Share (ADS) in cash (approximately $387.4 million, or ¥57.3 billion), under which Orchard shareholders will hold an additional contingent value right (CVR) of $1.00 per ADS. An additional $1.00 CVR will be paid for a total of $17.00 per ADS, or approximately $477.6 million (¥70.7 billion) if the conditions are met.

Key Points: 
  • Kyowa Kirin has established a 2030 Vision to consistently create and deliver medicines with life-changing value that ultimately makes people smile, as a J-GSP.
  • “We are excited to announce that we have signed the Transaction Agreement to acquire Orchard Therapeutics, one of the leading providers of HSC gene therapy,” said Takeyoshi Yamashita, Ph.D., Director of the Board, chief medical officer, senior managing executive officer of Kyowa Kirin.
  • Under the terms of the agreement, Kyowa Kirin will initiate a scheme of arrangement to acquire all Orchard Therapeutics’ ADSs at a price of $16.00 per ADS in cash (or aggregated value of approximately $387.4 million, or approximately ¥57.3 billion) at closing, which represents a premium of 144% to Orchard Therapeutics’ volume-weighted average price per ADS over the previous 30 days ended October 4.
  • Following the completion of the acquisition, Orchard Therapeutics will become a wholly-owned subsidiary of Kyowa Kirin.

Orchard Therapeutics Announces Second Closing of Strategic Financing, Resulting in $34 Million of Additional Capital

Retrieved on: 
måndag, juni 26, 2023
Exercise, MLD, Sale, American depositary receipt, Prospectus, Investment, Atidarsagene autotemcel, HSC, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, BLA, Guggenheim Partners, Food, Pharmaceutical industry, Security (finance), Orchard

BOSTON and LONDON, June 26, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that it has completed the second closing of its previously announced securities purchase agreement, resulting in $34 million of new capital before placement agent and transaction fees.

Key Points: 
  • In March 2023, the company completed the first closing of an innovative financing arrangement , led by RA Capital Management with participation from other leading healthcare investors that resulted in $34 million of new capital before placement agent and transaction fees.
  • The second closing comprises the sale of units at a purchase price of $8.00 per unit for aggregate proceeds of $34 million, including the issuance of 4.25 million American Depository Shares (ADSs) as well as warrants.
  • “The initiation of our BLA submission marks a significant de-risking event for Orchard and our OTL-200 program ahead of a potential U.S. approval in MLD,” said Frank Thomas, president and chief operating officer of Orchard Therapeutics.
  • Any offering of the securities under the resale registration statement will only be by means of a prospectus.

Orchard Therapeutics Announces Positive Clinical and Preclinical Data in Programs Targeting Neurometabolic and CNS Disorders at ASGCT

Retrieved on: 
fredag, maj 19, 2023
Kaufman, UNIPD, Prefrontal cortex, Cerebrospinal fluid, Plasma, Motion, Frasier, Protein, Thalamus, Acquisition, Patient, Atidarsagene autotemcel, Randomized controlled trial, The New England Journal of Medicine, Cell, Disease, IDUA, UOM, Enzyme replacement therapy, Phenotype, DSM-IV codes, Tissue, University, Hip, Gene, Orchard, GRN, Dementia, San Raffaele Hospital, Growth, HSC, Cognition, Natural history, Kyphosis, Biomarker, MPS, Șaeș, Frontotemporal dementia, Webcast, Mortality, Therapy, Central nervous system, Doctor of Philosophy, Speech, HSCT, CNS, EDT, Health, MRI, SGSH, Dysplasia, Society, FTD, Finnish heritage disease, Hippocampus, Medical imaging, Pharmaceutical industry, Vaccine, Medical device, Nursing, Dentistry

BOSTON and LONDON, May 19, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced multiple clinical and preclinical updates from its portfolio of investigational hematopoietic stem cell (HSC) gene therapies in neurometabolic and neurodegenerative disorders. The data are being featured in several oral presentations at the ongoing 26th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) in Los Angeles.

Key Points: 
  • The data are being featured in several oral presentations at the ongoing 26th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) in Los Angeles.
  • At baseline, most patients presented with severe joint range of motion impairment, severe acetabular (hip) dysplasia and varying degrees of dorso-lumbar kyphosis.
  • Treatment with OTL-203 was generally well tolerated and demonstrated extensive metabolic correction over four years after treatment.
  • Data from in vivo studies indicate effective GRN protein delivery to the CNS of knockout mice transplanted with gene-modified HSCs.

Orchard Therapeutics Reports First Quarter 2023 Financial Results and Announces Initiation of Rolling Submission for Biologics License Application of OTL-200 for Metachromatic Leukodystrophy to U.S. FDA

Retrieved on: 
måndag, maj 15, 2023
POC, RA, Gene, Civil service commission, MLD, Orchard, Health care, CMC, Cohort (statistics), Hereditary angioedema, American depositary receipt, Rady, Medication, G&A, Research, Genetic distance, Cell, Science, HAE, Sanfilippo, Patient, Investment, Annual general meeting, Frontotemporal dementia, Medicines and Healthcare products Regulatory Agency, HSC, FDA, Therapy, EC, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, BLA, FTD, Society, Food, European Commission, MHRA, IND, Hurler, Pharmaceutical industry, Health insurance, Medical device, Security (finance), Cryptocurrency, Atidarsagene autotemcel

BOSTON and LONDON, May 15, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced various business accomplishments along with its financial results for the quarter ended March 31, 2023.

Key Points: 
  • OTL-200 is approved as Libmeldy® (atidarsagene autotemcel) by the European Commission (EC) and Medicines and Healthcare products Regulatory Agency (MHRA).
  • The company has secured reimbursement agreements for Libmeldy in three new markets so far in 2023.
  • Orchard Therapeutics has outlined the following key milestones expected for 2023:
    Libmeldy: Establish qualified treatment centers in Sweden, Spain and Saudi Arabia.
  • The company had approximately 184.3 million ordinary shares, equivalent to 18.4 million American Depositary Shares, outstanding as of March 31, 2023.

Orchard Therapeutics Provides Business Update and Reports 2022 Financial Results

Retrieved on: 
måndag, mars 6, 2023
Civil service commission, Research, Health care, ADS, Patient, POC, American depositary receipt, BLA, HAE, Webcast, IND, Incidence, HSC, GAAP, MPS, NBS, Hereditary angioedema, Type, Medicines and Healthcare products Regulatory Agency, European Commission, Investment, FDA, Diagnosis, CGMP, Aircraft, MLD, SG, Newborn screening, Bank statement, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Therapy, Food, MHRA, Medication, EC, Orchard, Pharmaceutical industry, Cryptocurrency, Medical device, Bank, Commercial property, Atidarsagene autotemcel, G&A

ET

Key Points: 
  • ET
    BOSTON and LONDON, March 06, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced business updates along with its financial results for the quarter and year ended December 31, 2022.
  • Based on the outcome of the Type B meeting, Orchard Therapeutics subsequently requested a pre-BLA meeting which has been scheduled for the second quarter of 2023.
  • Management believes this non-GAAP information is useful for investors, taken in conjunction with Orchard’s GAAP financial statements, because it provides greater transparency regarding Orchard’s operating performance.
  • Management uses these measures, among others, to assess and analyze operational results and trends and to make financial and operational decisions.