Selected EMA news
Most of EHA’s regulatory activities focus on collaboration with the European Medicines Agency (EMA), the European Union agency tasked with evaluating the efficacy and safety of medicines submitted for EU marketing authorization, and for monitoring safety across the lifecycle of medicines. EHA has built a strong, mutually beneficial relationship with EMA which encompasses the exchange of information and advice, joint educational activities, nomination of experts for regulatory assessment and scientific consultations, and dialogue between regulator and medical experts about emerging trends and issues arising from scientific and technological innovation or 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), the Scientific Advisory Group on Oncology (SAG-O), the Policy Officers Group (HCP POG) and the Cancer Medicines Forum.
Monthly news on EMA Scientific Committees decisions and updates on hematological medicines.
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
- Thalidomide Lipomed (thalidomide) – Biosimilar
Treatment of multiple myeloma
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
- Parsaclisib Incyte Biosciences Distribution B.V. (parsaclisib)
Intended for the treatment of marginal zone lymphoma
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
EMA News April 2022
Positive CHMP opinions on new medicines
- Carvykti (ciltacabtagene autoleucel) – Conditional approval
Treatment of multiple myeloma
- Zolsketil pegylated liposomal (doxorubicin)
Treatment of breast and ovarian cancers, multiple myeloma and AIDS-related Kaposi's sarcoma
New medicines authorised
- Dasatinib Accord and Dasatinib Accordpharma (dasatinib) - Generic of Sprycel
Treatment of leukemia - Oxbryta (Voxelotor) – Orphan medicine
Treatment of haemolytic anaemia in patients with sickle cell disease
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
- Inrebic (fedratinib) – Orphan Medicine
Treatment of myelofibrosis
Negative CHMP opinions on new medicines
- Gamifant (emapalumab)
Intended for the treatment of primary haemophagocytic lymphohistiocytosis (HLH)
Withdrawal of applications to change the marketing authorisation
- Brilique (ticagrelor)
Intended to be used with aspirin to prevent problems caused by blood clots
Safety update
- Review of Zynteglo (betibeglogene autotemcel) - review started (Art. 20)
Treatment of a blood disorder known as beta thalassaemia
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
- Precautionary marketing suspension of thalassaemia medicine Zynteglo (betibeglogene autotemcel)
Direct Healthcare Professional Communication (DHPC)
- Lojuxta (lomitapide): Reminder to monitor the liver function of patients treated with Lojuxta and to
avoid use in pregnancy
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
- Direct healthcare professional communication: Ondexxya (andexanet alfa): Avoid use of andexanet prior to heparinization
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
- Direct healthcare professional communication (DHPC): Esbriet (pirfenidone): Important safety update and new recommendations to prevent Drug-Induced Liver Injury (DILI)
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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