Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 25-28 June 2018
Nine medicines recommended for approval, including the first two CAR-T cell therapies in the EU
The European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) recommended nine medicines for approval, including six orphan medicines1, at its June 2018 meeting.
The CHMP recommended granting marketing authorisations for the first two chimeric antigen receptors (CAR) T-cell therapies in the European Union: Kymriah (tisagenlecleucel) and Yescarta (axicabtagene ciloleucel) are both advanced therapy medicinal products (ATMPs) intended for the treatment of certain blood cancers. Kymriah and Yescarta were designated as orphan medicines during their development. They are also the first medicines supported through EMA's PRIority MEdicines (PRIME) scheme to receive a positive opinion from the Committee.
Together with the approval recommendation for the first CAR-T cell therapies, the Committee also recommended an extension to the therapeutic indication for RoActemra (tocilizumab) to include the treatment of CAR-T-cell-induced cytokine release syndrome (CRS), a known serious side effect of CAR-T cell therapies. For more information on these medicines please see the press release in the grid below.
Four other orphan medicines were recommended for approval by the Committee: Cablivi (caplacizumab) for the treatment of acquired thrombotic thrombocytopenic purpura; Mepsevii (vestronidase alfa) for the treatment of mucopolysaccharidosis type VII; Veyvondi (vonicog alfa) for the treatment of von Willebrand disease and Vyxeos (daunorubicin / cytarabine) for the treatment of acute myeloid leukaemia.
Duzallo (lesinurad / allopurinol) received a positive opinion for the treatment of hyperuricaemia in patients with gout.
The CHMP granted a positive opinion for Ulipristal Acetate Gedeon Richter (ulipristal acetate), an informed consent application for the pre-operative treatment of uterine fibroids. An informed consent application makes use of data from the dossier of a previously authorised medicine, with the marketing authorisation holder of that medicine giving consent for the use of their data in the application.
Positive recommendation on a new medicine following re-examination
The CHMP recommended granting a marketing authorisation for Nerlynx (neratinib), for the adjuvant treatment of adult patients with breast cancer, after re-examining its negative opinion for this medicine adopted in February 2018.
For more information on this positive opinion following re-examination, please see the question-and-answer document in the grid below.
Seven recommendations on extensions of therapeutic indication
The Committee recommended extensions of indications for Dexdor, Inovelon, Jinarc, Lenvima, Opdivo, Rapamune and RoActemra (see above).
Start of referrals
The CHMP started a review of the effectiveness of bacterial lysate medicines in reducing the number and severity of respiratory infections in adults and children who experience repeated infections. For more information, please see the start-of-referral document in the grid below.
The Committee also started a review of Septanest (articaine / adrenaline solutions for injection) to harmonise the way the medicine is used in the countries where it is available. Septanest is a local anaesthetic used to prevent pain and discomfort in the mouth during dental procedures. For more information, please see the start-of-referral document in the grid below.
Withdrawal of application
The application for an initial marketing authorisation for Graspa (L-asparaginase) was withdrawn by the company. This medicine was intended to be used for the treatment of acute lymphoblastic leukaemia. A question-and-answer document on this withdrawal is available in the grid below.
Agenda and minutes
The agenda of the June 2018 meeting is published on EMA's website. Minutes of the May 2018 CHMP meeting will be published in the coming weeks.
Key figures from the June 2018 CHMP meeting are represented in the graphic below.
* The total number of negative opinions has been amended from six to five to reflect the positive opinion adopted by CHMP on Nerlynx following re-examination. Nerlynx initially received a negative opinion from the CHMP in February 2018.
More information on all other outcomes of the CHMP's June 2018 meeting is available in the grid below.
1 As always at time of approval, these orphan designations will now be reviewed by EMA's Committee for Orphan Medicinal Products (COMP) to determine whether the information available to date allows maintaining the medicines' orphan status and granting the medicines ten years of market exclusivity.
Positive recommendations on new medicines
|Name of medicine||Cablivi|
|International non-proprietary name (INN)||caplacizumab|
|Marketing-authorisation applicant||Ablynx NV|
|Therapeutic indication||Treatment of acquired thrombotic thrombocytopenic purpura (aTTP)|
|More information||Summary of opinion for Cablivi|
|Name of medicine||Duzallo|
|INN||lesinurad / allopurinol|
|Marketing-authorisation applicant||Grunenthal GmbH|
|Therapeutic indication||Treatment of hyperuricaemia in gout patients|
|Name of medicine||Kymriah|
|Marketing-authorisation applicant||Novartis Europharm Limited|
|Therapeutic indication||Treatment of acute lymphoblastic leukaemia (ALL) and diffuse large B-cell lymphoma (DLBCL)|
Summary of opinion for Kymriah
Press release: First two CAR-T cell medicines recommended for approval in the European Union
|Name of medicine||Mepsevii|
|Marketing-authorisation applicant||Ultragenyx Germany GmbH|
|Therapeutic indication||Treatment of mucopolysaccharidosis type VII|
|More information||Summary of opinion for Mepsevii|
|Name of medicine||Veyvondi|
|Marketing-authorisation applicant||Baxalta Innovations GmbH|
|Therapeutic indication||Treatment of von Willebrand disease|
|More information||Summary of opinion for Veyvondi|
|Name of medicine||Vyxeos|
|INN||daunorubicin / cytarabine|
|Marketing-authorisation applicant||Jazz Pharmaceuticals Ireland Limited|
|Therapeutic indication||Treatment of acute myeloid leukaemia|
|More information||Summary of opinion for Vyxeos|
|Name of medicine||Yescarta|
|Marketing-authorisation applicant||Kite Pharma EU B.V.|
|Therapeutic indication||Treatment of diffuse large cell lymphoma (DLBCL) and primary mediastinal B-cell lymphoma (PMBCL)|
Summary of opinion for Yescarta
Press release: First two CAR-T cell medicines recommended for approval in the European Union
Positive recommendation on new informed-consent application
|Name of medicine||Ulipristal Acetate Gedeon Richter|
|Marketing-authorisation applicant||Gedeon Richter Plc.|
|Therapeutic indication||Pre-operative treatment of uterine fibroids|
|More information||Summary of opinion for Ulipristal Acetate Gedeon Richter|
Positive recommendation for new medicine following re-examination
|Name of medicine||Nerlynx|
|Marketing-authorisation holder||Puma Biotechnology Limited|
|Therapeutic indication||Adjuvant treatment of adult patients with breast cancer|
Summary of opinion for Nerlynx
Questions and answers on the positive opinion on the marketing authorisation for Nerlynx - Outcome of re-examination
Positive recommendations on extensions of indications
|Name of medicine||Dexdor|
|Marketing-authorisation holder||Orion Corporation|
|More information||Summary of opinion for Dexdor|
|Name of medicine||Inovelon|
|Marketing-authorisation holder||Eisai Ltd.|
|More information||Summary of opinion for Inovelon|
|Name of medicine||Jinarc|
|Marketing-authorisation holder||Otsuka Pharmaceutical Europe Ltd|
|More information||Summary of opinion for Jinarc|
|Name of medicine||Lenvima|
|Marketing-authorisation holder||Eisai Europe Ltd.|
|More information||Summary of opinion for Lenvima|
|Name of medicine||Opdivo|
|Marketing-authorisation applicant||Bristol-Myers Squibb Pharma EEIG|
|More information||Summary of opinion for Opdivo|
|Name of medicine||Rapamune|
|Marketing-authorisation holder||Pfizer Limited|
|More information||Summary of opinion for Rapamune|
|Name of medicine||RoActemra|
|Marketing-authorisation holder||Roche Registration GmbH|
|More information||Summary of opinion for RoActemra|
Withdrawal of initial application
|Name of medicine||Graspa|
|Marketing-authorisation applicant||Erytech Pharma S.A.|
|More information||Questions and answers on withdrawal of the marketing authorisation application for Graspa|
Start of referral
|Name of medicine||Bacterial lysate medicines|
|More information||EMA reviewing bacterial lysate medicines for respiratory conditions|
|Name of medicine||Septanest and associated names|
|INN||articaine / adrenaline|
|More information||EMA starts review of Septanest (articaine / adrenaline solutions for injection) and associated names|
|Recommendations on eligibility to PRIME scheme|
|Overview of (invented) names reviewed in May 2018 by the Name Review Group (NRG)|
|Start of community reviews|
|Scientific advice and protocol assistance|
- Cablivi: EPAR
- Dexdor: EPAR
- Duzallo: EPAR
- Inovelon: EPAR
- Jinarc: EPAR
- Kymriah: EPAR
- Lenvima: EPAR
- Mepsevii: EPAR
- Nerlynx: EPAR
- Opdivo: EPAR
- Rapamune: EPAR
- RoActemra: EPAR
- Ulipristal Acetate Gedeon Richter: EPAR
- Veyvondi: EPAR
- Vyxeos liposomal (previously known as Vyxeos): EPAR
- Yescarta: EPAR
- Bacterial lysates-containing medicinal products indicated for respiratory conditions: Article 31 referrals
- Septanest and associated names: Article 30 referrals