Selected EMA news

Most of EHA’s regulatory activities focus on collaboration with the European Medicines Agency (EMA). EMA is the European Union agency that's responsible for:

  • Evaluating the efficacy and safety of medicines submitted for EU marketing authorization
  • Monitoring safety across the lifecycle of medicines

We've built a strong, mutually beneficial relationship with EMA that encompasses:

  • The exchange of information and advice
  • Joint educational activities
  • The nomination of experts for regulatory assessment and scientific consultations

Our relationship also helps to encourage dialogue between regulators and medical experts on a range of issues, including:

  • Emerging trends and issues arising from scientific and technological innovation
  • The implications of new EU legislation

EHA actively participates in a number of EMA groups and platforms, including the:

  • Health Care Professionals Working Party (HCPWP)
  • Scientific Advisory Group on Oncology (SAG-O)
  • Policy Officers Group (HCP POG)
  • Cancer Medicines Forum

On this page, we share monthly news and updates on:

  • EMA scientific committees' decisions
  • Hematological medicines

January 2024

Positive CHMP opinions on new medicines

  • Casgevy (exagamglogene autotemcel) – orphan medicine – conditional approval
    Treatment of transfusion dependent β-thalassemia and sickle cell disease

  • Pomalidomide Viatris (pomalidomide) – generic of Imnovid
    Treatment of multiple myeloma

New information on authorised medicines

  • VeraSeal (human fibrinogen / human thrombin) – extension of indication
    Treatment for stopping bleeding during surgeries 

Negative CHMP opinions on renewal of authorised medicine

  • Blenrep (belantamab mafodotin
    Intended for treatment of multiple myeloma

November–December 2023

Positive CHMP opinions on new medicines

  • Elrexfio (elranatamab) – conditional approval
    Treatment of multiple myeloma

New medicines authorised

  • Enrylaze (crisantaspase)
    Treatment of lymphoblastic leukaemia and lymphoblastic lymphoma

  • Inaqovi (cedazuridine / decitabine)
    Treatment of myeloid leukaemia

  • Tepkinly (epcoritamab) – orphan medicine – conditional approval
    Treatment of blood cancer called diffuse large B-cell lymphoma

  • Vanflyta (quizartinib)
    Treatment of myeloid leukaemia

New information on authorised medicines

  • Veyvondi (vonicog alfa) – orphan medicine – extension of indication
    Treatment of von Willebrand disease

Special update, November 2023

Electronic Product Information (ePI) for selected human medicines published for first time

EMA, along with the Heads of Medicines Agencies (HMA) and the European Commission (EC) have published for the first time electronic product information (ePI) for selected human medicines harmonized across the European Union.

This forms part of the ePI initiative, a one-year pilot by HMA, EMA, and the EC to enable the transition to an electronic system for medicines evaluated both nationally and at European level.

October 2023

Positive CHMP opinions on new medicines

  • Vanflyta (quizartinib) orphan medicine
    Treatment of acute myeloid leukaemia (blood cancer)
  • Elrexfio (elranatamab)orphan medicine – conditional approval
    Treatment of multiple myeloma (a type of white blood cell cancer)

New medicines authorized

  • Talvey (talquetamab)orphan medicine – conditional approval
    Treatment of multiple myeloma (a type of white blood cell cancer)

New information on authorized medicines

  • Adcetris (brentuximab vedotin) orphan medicineextension of indication
    Treatment of stage III Hodgkin lymphoma (a cancer of lymphocytes)

Withdrawal of authorized medicines

Withdrawal of applications for extension of indication

  • Iclusig (ponatinib)
    Intended for the treatment of adults newly diagnosed with Philadelphia chromosome-positive acute lymphoblastic leukaemia (blood cancer)

Negative CHMP opinions on renewal of conditional marketing authorisation

September 2023

Direct Healthcare Professional Communication (DHPC)

August 2023

Positive CHMP opinions on new medicines

  • Enrylaze (crisantaspase)
    Treatment of acute lymphoblastic leukaemia (a type of blood cancer)
  • Inaqovi (cedazuridine / decitabine)orphan medicine
    Treatment of myeloid leukaemia (a type of blood cancer)
  • Talvey (talquetamab)orphan medicine – conditional approval
    Treatment of multiple myeloma, a type of white blood cell cancer
  • Tepkinly (epcoritamab) – conditional approval
    Treatment of large B-cell lymphoma (a type of blood cancer)

New medicines authorised

  • Columvi (glofitamab) orphan medicine
    Treatment of blood cancer called diffuse large B-cell lymphoma (a type of blood cancer)

Withdrawal of applications for extension of indication

  • Gazyvaro (obinutuzumab)
    Intended to prevent a serious side effect (cytokine release syndrome) that occurs with the blood cancer medicine Columvi

Withdrawal of applications for new medicines

  • Jesduvroq (daprodustat)
    Treatment of symptomatic anaemia in patients with chronic kidney disease

Direct Healthcare Professional Communication (DHPC)

July 2023

Positive CHMP opinions on new medicines

  • Jesduvroq (daprodustat)
    Treatment of anaemia in adults with chronic kidney disease

New information on authorised medicines

  • Mircera (methoxy polyethylene glycol-epoetin beta) - extension of indication
    Treatment of anaemia associated with chronic kidney disease in children
  • Refixia (nonacog beta pegol) - extension of indication
    Treatment of haemophilia B

Withdrawal of applications for new medicines

  • Dyrupeg (pegfilgrastim)
    Intended to reduce the duration of neutropenia (low levels of neutrophils, a type of white blood cells in cancer patients)
  • Zefylti (filgrastim)
    Intended to stimulate the production of white blood cells

Direct Healthcare Professional Communication (DHPC)

  • Adakveo (crizanlizumab) - orphan medicine
    Prevention of painful crises in patients with sickle cell disease

June 2023

New medicines authorised

  • Epysqli (Eculizumab)
    Treatment of paroxysmal nocturnal haemoglobinuria, a disease in which excessive breakdown of red blood cells results in various medical complications
  • Tibsovo (ivosidenib)
    Treatment of acute myeloid leukaemia (a blood cancer) and biliary tract cancer (cancer of the bile ducts)
  • Vafseo (vadadustat)
    Treatment of anaemia (low levels of red blood cells) associated with chronic kidney disease

Supply shortages

  • Actilyse (alteplase)
    Treatment of acute pulmonary embolism (blood clot in blood vessels supplying the lung)

Safety update

  • Review of Adakveo (crizanlizumab) - CHMP Opinion
    Prevention of painful crises in patients with sickle cell disease

May 2023

New information on authorised medicines

  • Adempas (riociguat) – New indication
    Treatment of high blood pressure in lungs in children

Safety update

  • Review of Adakveo (crizanlizumab) review started
    Treatment of painful crises in patients with sickle cell disease

April 2023

Positive CHMP opinions on new medicines

  • Epysqli (eculizumab) – Biosimilar
    Treatment of paroxysmal nocturnal heamoglobinuria

New information on authorised medicines

  • Ultomiris (ravulizumab) – New indication and new pharmaceutical form
    Treatment of paroxysmal nocturnal haemoglobinuria (PNH) and atypical haemolytic uraemic syndrome (aHUS)

Withdrawal of applications for new medicines

  • Feraheme (ferumoxytol)
    Intended for treatment of iron deficiency anaemia

Safety update

Direct Healthcare Professional Communication (DHPC)

  • Janus kinase inhibitors (JAKi) (abrocitinib, filgotinib, baricitinib, upadacitinib, tofacitinib) Updated recommendations to minimize risk of cancer, heart problems and blood clots

March 2023

Positive CHMP opinions on new medicines

  • Bekemv (eculizumab) – Biosimilar medicine
    Treatment of paroxysmal nocturnal haemoglobinuria
  • Tibsovo (ivosidenib) - Orphan medicine
    Treatment of myeloid leukaemia
  • Tidhesco (ivosidenib) - Orphan  medicine
    Treatment of myeloid leukaemia
  • Vafseo (vadadustat) Treatment of anaemia associated with chronic kidney disease

New medicines authorised

  • Plerixafor Accord (plerixafor) - generic of Mozobil
    Medicine used in cancer patients to obtain cells from the bone marrow for use in transplantation

Direct Healthcare Professional Communication (DHPC)

  • Adakveo (crizanlizumab)
    Treatment of anaemia
  • Neofordex (dexamethasone)
    Treatment of multiple myeloma

February 2023

New information on authorized medicines

  • Reblozyl (luspatercept) – Extension of indication – Orphan medicine
    New indication: Treatment of anemia in patients with beta-thalassaemia

Safety update

  • Review of Adakveo (crizanlizumab) - Review started (Scope of safety referral)
  • Prevention of painful crises in patients with sickle cell disease

January 2023

Positive CHMP opinions on new medicines

  • Hemgenix (etranacogene dezaparvovec) – Orphan Medicine, conditional approval
    Treatment of severe and moderately severe hemophilia B

New medicines authorized

  • Pyrukynd (mitapivat) – Orphan medicine
    Treatment of adults with pyruvate kinase deficiency

New information on authorized medicines

  • Hemlibra (emicizumab) – Extension of indication
    New indication: routine prophylaxis of bleeding episodes in patients with hemophilia A without factor VIII inhibitors who have moderate disease with severe bleeding phenotype

November 2022

Positive CHMP opinions on new medicines

  • Ebvallo (tabelecleucel) – Orphan Medicine, Exceptional circumstances
    Treatment of Epstein-Barr virus positive post-transplant lymphoproliferative disease
     
  • Plerixafor Accord (plerixafor) – Generic of Mozobil
    Treatment of blood cancer after bone marrow transplantation

New medicines authorized

New information on authorized medicines

  • Brukinsa (zanubrutinib) – Orphan Medicine
    Treatment of chronic lymphocytic leukemia

September 2022

Positive CHMP opinions on new medicines

  • Enjaymo (sutimlimab) – Orphan Medicine
    Treatment of hemolytic anaemia

  • Pyrukynd (mitapivat) – Orphan Medicine
    Treatment of pyruvate kinase deficiency

  • Zynlonta (loncastuximab tesirine) – Orphan Medicine
    Treatment of diffuse large B-cell lymphoma and high-grade B-cell lymphoma 

August 2022

New information on authorised medicines

  • Tecartus (Autologous peripheral blood T cells CD4 and CD8 selected and CD3 and CD28 activated transduced with retroviral vector expressing anti-CD19 CD28/CD3-zeta chimeric antigen receptor and cultured) – Orphan medicine, Conditional Approval
    New indication: Treatment of mantle cell lymphoma

Withdrawal of applications for new medicines

July 2022

New information on new medicines

  • Tecvayli (teclistamab) - recommended for conditional marketing authorisation
    Treatment of relapsed and refractory multiple myeloma

Positive CHMP opinions on new medicines

  • Pepaxti (melphalan flufenamide) – Orphan Medicine
    Treatment of multiple myeloma

  • Roctavian (valoctocogene roxaparvovec) – Orphan Medicine – Conditional approval
    Treatment of haemophilia
  • Scemblix (asciminib) – Orphan Medicine
    Treatment of Philadelphia chromosome positive chronic myeloid leukaemia

New medicine authorised

  • Lunsumio (mosunetuzumab) – Orphan Medicine – Conditional approval
    Treatment of follicular lymphoma

New information on authorised medicines

  • Imbruvica (ibrutinib) - Extension of indication
    Treatment of chronic lymphocytic leukaemia

June 2022

Positive CHMP opinions on new medicines

  • Cevenfacta (Eptacog beta (activated))
    Treatment of bleeding episodes

New medicines authorised

  • Breyanzi (lisocabtagene maraleucel)
    Treatment of different types of blood cancers

Withdrawal of applications for new medicines

  • HemAryo (eptacog alfa (activated))
    Intended for treating bleeding episodes and for preventing bleeding after surgical procedures in patients with clotting disorders

April 2022

Positive CHMP opinions on new medicines

  • Carvykti (ciltacabtagene autoleucel) – Conditional approval
    Treatment of multiple myeloma

New medicines authorised

New information on authorised medicines

  • Kymriah (tisagenlecleucel) – Orphan medicine
    New indication: Treatment of follicular lymphoma
  • Polivy (polatuzumab vedotin) – Orphan medicine
    New indication Treatment of large B-cell lymphoma

Withdrawal of applications for extension of indication

  • Vyxeos liposomal (daunorubicin / cytarabine) – Orphan medicine
    Intended for the treatment of patients aged 1 to 21 years with acute myeloid leukaemia

March 2022

Positive CHMP opinions on new medicines

  • Carvykti (ciltacabtagene autoleucel)
    Treatment of adult patients with relapsed and refractory multiple myeloma

New medicines authorised

  • Evrenzo (roxadustat)
    Treatment of the symptoms of anaemia caused by chronic kidney failure

  • Rivaroxaban Mylan (rivaroxaban) - generic of Xarelto
    Treatment that prevents blood clotting

February 2022

Positive CHMP opinions on new medicines

  • Breyanzi (lisocabtagene maraleucel) – Orphan medicine
    Treatment of lymphoma

  • Dasatinib Accord (dasatinib) –  Generic of Sprycel
    Treatment of leukemia

  • Dasatinib Accordpharma (dasatinib) –  Generic of Sprycel
    Treatment of leukemia

  • Stimufend (pegfilgrastim) – Biosimilar of Neulasta
    Treatment of neutropenia after chemotherapy 

New information on authorised medicines

  • Ayvakyt (avapritinib) – Orphan medicine
    New indication Treatment of gastrointestinal stromal tumour and systemic mastocytosis

Withdrawal of applications for new medicines

  • Aliqopa (copanlisib)Treatment of previously treated marginal zone lymphoma

January 2022

Positive CHMP opinions on new medicines

  • Oxbryta (Voxelotor) - Orphan Medicine
    Treatment of anemia due to sickle cell disease

New medicines authorised

  • Aspaveli (pegcetacoplan) - Orphan Medicine
    Treatment of paroxysmal nocturnal haemoglobinuria

December 2021

Positive CHMP opinions on new medicines

  • Tavneos (avacopan) - Orphan Medicine
    Treatment of granulomatosis with polyangiitis or microscopic polyangiitis

November 2021

Positive CHMP opinions on new medicines

  • Aspaveli (pegcetacoplan)
    Treatment of paroxysmal nocturnal haemoglobinuria

New medicines authorised

  • Imatinib Koanaa (imatinib) generic of Glivec
    Treatment of blood cancers and gastrointestinal stromal tumors

New information on authorised medicines

  • Hizentra (human normal immunoglobulin (SCIg) - Extension of indication
    Treatment of immunologic deficiency syndromes

October 2021

Positive CHMP opinions on new medicines

  • Brukinsa (zanubrutinib) - Orphan medicine
    Treatment of Waldenström’s macroglobulinaemia

September 2021

New medicine authorised

  • Evrenzo (roxadustat)
    Treatment of anaemia symptoms (low red blood cell counts) caused by chronic kidney failure

August 2021

Positive CHMP opinions on new medicines

  • Imatinib Koanaa (imatinib)
    Treatment of leukaemia and gastrointestinal stromal tumours

New information on authorised medicines

  • Ultomiris (ravulizumab) - extension of indication
    Treatment of paroxysmal nocturnal haemoglobinuria and atypical haemolytic uraemic syndrome

Negative CHMP opinions on extension of indication

  • Siklos (hydroxycarbamide)
    Intended to extend the use of Siklos to include the treatment of severe chronic (long-term)
    anaemia in patients suffering from sickle cell syndrome

Safety update

  • Review of Zynteglo (betibeglogene autotemcel) - CHMP Opinion
    Treatment of beta thalassaemia

July 2021

Positive CHMP opinions on new medicines

  • Evrenzo (roxadustat)
    Treatment of anaemia symptoms in patients with chronic kidney disease

New medicines authorised

  • Inrebic (fedratinib) Orphan Medicine
    Treatment of myelofibrosis
  • Onureg (azacitidine)
    Treatment of acute myeloid leukaemia

April 2021

Positive CHMP opinions on new medicines

  • Copiktra (duvelisib)
    Treatment of relapsed or refractory chronic lymphocytic leukaemia and refractory follicular lymphoma

New medicines authorised

Negative CHMP opinions on new medicines

Withdrawal of applications to change the marketing authorisation

Safety update

March 2021

Positive CHMP opinions on new medicines

  • Nexpovio (selinexor)
    Treatment of multiple myeloma (cancer of the bone marrow)

  • Thiotepa Riemser (thiotepa) generic of Tepadina
    Treatment prior to blood-stem cell transplantation

New medicines authorised

  • Lenalidomide KrKa (lenalidomide) generic of Revlimid
    Treatment of multiple myeloma and follicular lymphoma (blood cancers)

  • Lenalidomide KrKa d.d. (lenalidomide) generic of Revlimid
    Treatment of multiple myeloma, myelodysplastic syndromes, and follicular lymphoma (blood cancers)

  • Lenalidomide KrKa d.d. Novo mesto (lenalidomide) generic of Revlimid
    Treatment of multiple myeloma, myelodysplastic syndromes, mantle cell lymphoma and follicular lymphoma (blood cancers)

New information on authorised medicines

  • Sarclisa (isatuximab) - extension of indication
    Treatment of multiple myeloma (blood cancer)

Withdrawal of authorised medicines

  • Udenyca (pegfilgrastim)
    Treatment of neutropenia (low level of white blood cells)

Other information

Direct Healthcare Professional Communication (DHPC)

February 2021

New medicines authorised

  • Elzonris (tagraxofusp) Treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN)

  • Tecartus (Autologous peripheral blood T cells CD4 and CD8 selected and CD3 and CD28 activated transduced with retroviral vector expressing anti-CD19 CD28/CD3-zeta chimeric antigen receptor and cultured) Treatment of mantle cell lymphoma – Orphan Medicine

January 2021

Positive CHMP opinions on new medicines

  • Inrebic (fedratinib) – Orphan Medicine
    Treatment of of primary myelofibrosis and of myelofibrosis secondary to polycythaemia vera or essential thrombocythaemia

  • Lenalidomide KrKa (lenalidomide) - generic of Revlimid
    Treatment of multiple myeloma and follicular lymphoma

  • Lenalidomide KrKa d.d. (lenalidomide) - generic of Revlimid
    Treatment of multiple myeloma, myelodysplastic syndromes and follicular lymphoma

  • Lenalidomide KrKa d.d. Novo mesto (lenalidomide) - generic of Revlimid
    Treatment of multiple myeloma, myelodysplastic syndromes, mantle cell lymphoma and follicular lymphoma

  • Lumoxiti (moxetumomab pasudotox)
    Treatment of relapsed or refractory hairy cell leukaemia – Orphan Medicine, Exceptional Circumstances

New information on authorised medicines

  • Nplate (romiplostim) - extension of indication
    Treatment of patients with long-term immune thrombocytopenic purpura (ITP), a disease in which the patient’s immune system destroys platelets

New information on authorised medicines

  • Doptelet (avatrombopag) - new indication Treatment of severe thrombocytopenia in patients with chronic liver disease

December 2020

Positive CHMP opinions on new medicines-following re-examination

  • Elzonris (tagraxofusp) Treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN) – Exceptional Circumstances

New medicines authorised

  • Adakveo (crizanlizumab) Prevention of recurrent vaso-occlusive crises in patients with sickle cell disease

  • Calquence (acalabrutinib) Treatment of chronic lymphocytic leukaemia – Orphan medicine

  • Nyvepria (pegfilgrastim) Treatment of neutropenia in cancer patients - Biosimilar of Neulasta

  • Phelinun (melphalan ) Treatment of blood and bone marrow cancers and preparing patients for blood stem cell transplants

  • Rivaroxaban Accord (rivaroxaban) Treatment and prevention of blood clots - Generic

New information on authorised medicines

  • Kyprolis (carfilzomib) - extension of indication Treatment of adult patients with multiple myeloma

Safety update

November 2020

Positive CHMP opinions on new medicines

  • Lenalidomide Mylan (lenalidomide) - Generic
    Treatment of multiple myeloma and follicular lymphoma (blood cancers)

New medicines authorised

  • Arsenic trioxide medac (arsenic trioxide) - Generic
    Treatment of acute promyelocytic leukaemia (blood cancer)

New information on authorised medicines

  • Blincyto (blinatumomab) - extension of indication – Orphan medicine
    Treatment of B-precursor acute lymphoblastic leukaemia (a type of blood cancer)

Safety update

October 2020

Positive CHMP opinions on new medicines

  • Nyvepria (pegfilgrastim) - Biosimilar of Neulasta
    Treatment of neutropenia in cancer patients

  • Rivaroxaban Accord (rivaroxaban) - Generic
    Treatment and prevention of blood clots

New medicines authorised

  • Blenrep (belantamab mafodotin) – Conditional Approval – Orphan Medicine
    Treatment of multiple myeloma

Re-examination request following negative CHMP opinions on new medicines

  • Elzonris (tagraxofusp)
    Intended for treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN), a rare and aggressive type of blood cancer

August 2020

Positive CHMP opinions on new medicines

  • Adakveo (crizanlizumab)
    Prevention of recurrent vaso-occlusive crises in patients with sickle cell disease

  • Arsenic trioxide medac (arsenic trioxide)
    Treatment of acute promyelocytic leukaemia

  • Calquence (acalabrutinib) – Orphan Medicine
    Treatment of chronic lymphocytic leukaemia

New medicines authorised

  • Daurismo (glasdegib)
    Treatment of acute myeloid leukaemia (blood cancer)

  • Reblozyl (luspatercept) – Orphan Medicine
    Treatment of anaemia caused by myelodysplastic syndromes (MDS) or betathalassaemia

New information on authorised medicines

  • Crysvita (burosumab) - change and extension of indication – Orphan Medicine – Conditional Approval
    Treatment of X-linked hypophosphataemia

  • Imbruvica (ibrutinib) - extension of indication – Orphan Medicine
    Treatment of blood cancers

  • NovoThirteen (catridecacog)- extension of indication
    Prevention of bleeding in patients with inherited blood clotting disorder

Negative CHMP opinions on new medicines

  • Gamifant (emapalumab)
    Intended for the treatment of primary haemophagocytic lymphohistiocytosis (HLH)

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