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.

CHMP statistics

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.

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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	Mepsevii
INN	vestronidase alfa
Marketing-authorisation applicant	Ultragenyx Germany GmbH
Therapeutic indication	Treatment of mucopolysaccharidosis type VII
More information	Summary 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	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

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	Summary of opinion for Ulipristal Acetate Gedeon Richter

Positive recommendation for new medicine following re-examination

Positive recommendations on extensions of indications

Name of medicine	Dexdor
INN	dexmedetomidine
Marketing-authorisation holder	Orion Corporation
More information	Summary of opinion for Dexdor

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

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

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

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

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

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

Withdrawal of initial application

Name of medicine	Graspa
INN	L-asparaginase
Marketing-authorisation applicant	Erytech Pharma S.A.
More information	Questions and answers on withdrawal of the marketing authorisation application for Graspa

Start of referral

Other updates

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