Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 25-28 June 2018

News 29/06/2018

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.

CHMP statistics

Key figures from the June 2018 CHMP meeting are represented in the graphic below.

CHMP June 2018
* 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 PDF iconSummary of opinion for Cablivi

 

Name of medicine Duzallo
INN lesinurad / allopurinol
Marketing-authorisation applicant Grunenthal GmbH
Therapeutic indication Treatment of hyperuricaemia in gout patients
More information PDF iconSummary of opinion for Duzallo

 

Name of medicine Kymriah
INN tisagenlecleucel
Marketing-authorisation applicant Novartis Europharm Limited
Therapeutic indication Treatment of acute lymphoblastic leukaemia (ALL) and diffuse large B-cell lymphoma (DLBCL)
More information

PDF iconSummary of opinion for Kymriah

Press release: First two CAR-T cell medicines recommended for approval in the European Union

 

Name of medicine Mepsevii
INN vestronidase alfa
Marketing-authorisation applicant Ultragenyx Germany GmbH
Therapeutic indication Treatment of mucopolysaccharidosis type VII
More information PDF iconSummary of opinion for Mepsevii

 

Name of medicine Veyvondi
INN vonicog alfa
Marketing-authorisation applicant Baxalta Innovations GmbH
Therapeutic indication Treatment of von Willebrand disease
More information PDF iconSummary 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 PDF iconSummary of opinion for Vyxeos

 

Name of medicine Yescarta
INN axicabtagene ciloleucel
Marketing-authorisation applicant Kite Pharma EU B.V.
Therapeutic indication Treatment of diffuse large cell lymphoma (DLBCL) and primary mediastinal B-cell lymphoma (PMBCL)
More information

PDF iconSummary 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
INN ulipristal acetate
Marketing-authorisation applicant Gedeon Richter Plc.
Therapeutic indication Pre-operative treatment of uterine fibroids
More information PDF iconSummary of opinion for Ulipristal Acetate Gedeon Richter



Positive recommendation for new medicine following re-examination

Name of medicine Nerlynx
INN neratinib
Marketing-authorisation holder Puma Biotechnology Limited
Therapeutic indication Adjuvant treatment of adult patients with breast cancer
More information

PDF iconSummary of opinion for Nerlynx

PDF iconQuestions 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
INN dexmedetomidine
Marketing-authorisation holder Orion Corporation
More information PDF iconSummary of opinion for Dexdor

 

Name of medicine Inovelon
INN rufinamide
Marketing-authorisation holder Eisai Ltd.
More information PDF iconSummary of opinion for Inovelon

 

Name of medicine Jinarc
INN tolvaptan
Marketing-authorisation holder Otsuka Pharmaceutical Europe Ltd
More information PDF iconSummary of opinion for Jinarc

 

Name of medicine Lenvima
INN lenvatinib
Marketing-authorisation holder Eisai Europe Ltd.
More information PDF iconSummary of opinion for Lenvima

 

Name of medicine Opdivo
INN nivolumab
Marketing-authorisation applicant Bristol-Myers Squibb Pharma EEIG
More information PDF iconSummary of opinion for Opdivo

 

Name of medicine Rapamune
INN sirolimus
Marketing-authorisation holder Pfizer Limited
More information PDF iconSummary of opinion for Rapamune

 

Name of medicine RoActemra
INN tocilizumab
Marketing-authorisation holder Roche Registration GmbH
More information PDF iconSummary of opinion for RoActemra



Withdrawal of initial application

Name of medicine Graspa
INN L-asparaginase
Marketing-authorisation applicant Erytech Pharma S.A.
More information PDF iconQuestions 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



Other updates

PDF iconRecommendations on eligibility to PRIME scheme
PDF iconOverview of (invented) names reviewed in May 2018 by the Name Review Group (NRG)
PDF iconStart of community reviews
PDF iconScientific advice and protocol assistance

How useful was this page?

Add your rating
Average
4 ratings
2 ratings
3 ratings
3 ratings