Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 9-12 November 2020
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Five new medicines recommended for approval
EMA’s human medicines committee (CHMP) recommended five medicines for approval at its November 2020 meeting.
The Committee recommended granting a marketing authorisation for Phesgo (pertuzumab / trastuzumab) for the treatment of early and metastatic breast cancer.
The CHMP adopted a positive opinion for Roclanda (latanoprost / netarsudil) for the reduction of elevated intraocular pressure (IOP) in adult patients with primary open-angle glaucoma or ocular hypertension for whom monotherapy with a prostaglandin or netarsudil provides insufficient IOP reduction.
Xofluza (baloxavir marboxil) received a positive opinion from the CHMP for the treatment and post-exposure prophylaxis of uncomplicated influenza.
The biosimilar medicine Onbevzi (bevacizumab) received a positive opinion for the treatment of carcinoma of the colon or rectum, breast cancer, non-small cell lung cancer, renal cell cancer, epithelial ovarian, fallopian tube or primary peritoneal cancer and carcinoma of the cervix.
Positive recommendation on a new medicine following re-examination
The CHMP recommended approving the marketing authorisation for Elzonris* (tagraxofusp), for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN), a rare and aggressive type of acute myeloid leukaemia (blood cancer). The Committee had initially refused the application in July 2020 for Elzonris to be used for the treatment of BPDCN regardless of whether patients had been previously treated with other medicines or not. After re-examination, the CHMP recommended that marketing authorisation under exceptional circumstances could be granted but for a restricted indication in patients who had not yet received any treatment for BPDCN (first-line treatment).
For more information on this positive opinion following re-examination, see the question-and-answer document in the grid below.
Negative recommendation on a new medicine following re-examination
The applicant for Gamifant (emapalumab) requested a re-examination of the Committee's negative opinion adopted at the July 2020 meeting. After considering the grounds for this request, the CHMP re-examined the initial opinion and confirmed its previous recommendation to refuse the granting of a marketing authorisation for this medicine, which was intended for the treatment of primary haemophagocytic lymphohistiocytosis (a genetic disease characterised by an overactive immune system) in children under 18 years of age.
For more information on this negative opinion, see the question-and-answer document in the grid below.
Six recommendations on extensions of therapeutic indication
The Committee recommended extensions of indication for Kyprolis, Pradaxa, Tivicay, Trimbow, Xarelto and Xyrem.
Nitrosamines: EMA aligns recommendations for sartans with those for other medicines
The CHMP has aligned recommendations for limiting nitrosamine impurities in sartan medicines with recent Nitrosamine impurities - Final outcome of Article 5(3) it issued for other classes of medicines. The main change concerns the limits for nitrosamines, which previously applied to the active ingredients but will now apply instead to the finished products (e.g. tablets). These limits, based on internationally agreed standards (ICH M7(R1)), should ensure that the excess risk of cancer from nitrosamines in any sartan medicines is below 1 in 100,000 for a person taking the medicine for lifelong treatment.
For more information, see the public health recommendation in the grid below.
Outcome of review on medicines containing ulipristal acetate 5 mg
The Committee recommended restricting use of medicines containing ulipristal acetate 5 mg (Esmya and generic medicines) as a result of cases of serious liver injury. The medicines can now only be used to treat uterine fibroids in premenopausal women for whom surgical procedures (including uterine fibroid embolisation) are not appropriate or have not worked. The medicines must not be used for controlling symptoms of uterine fibroids while awaiting surgical treatment.
For more information, see the public health recommendation in the grid below.
Withdrawals of applications
Three applications for initial marketing authorisations were withdrawn: Puldysa (idebenone), for the treatment of Duchenne muscular dystrophy; Roctavian (valoctocogene roxaparvovec), for the treatment of severe haemophilia A; and Tibsovo (ivosidenib), for the treatment of acute myeloid leukaemia, a cancer of white blood cells.
Question-and-answer documents on these withdrawals are available in the grid below.
Agenda and minutes
The agenda of the November 2020 meeting is published on EMA's website. Minutes of the October 2020 CHMP meeting will be published in the coming weeks.
CHMP statistics
Key figures from the November 2020 CHMP meeting are represented in the graphic below.
* This product was designated as an orphan medicine during its development. Orphan designations are reviewed by EMA's Committee for Orphan Medicinal Products (COMP) at the time of approval to determine whether the information available to date allows maintaining the medicine’s orphan status and granting the medicine ten years of market exclusivity.
Positive recommendations on new medicines
| Name of medicine | Phesgo |
| INN | pertuzumab / trastuzumab |
| Marketing-authorisation applicant | Roche Registration GmbH |
| Therapeutic indication | Treatment of early and metastatic breast cancer. |
| More information |
| Name of medicine | Roclanda |
| INN | latanoprost / netarsudil |
| Marketing-authorisation applicant | Aerie Pharmaceuticals Ireland Limited |
| Therapeutic indication | Reduction of elevated intraocular pressure (IOP) in adult patients with primary open-angle glaucoma or ocular hypertension for whom monotherapy with a prostaglandin or netarsudil provides insufficient IOP reduction. |
| More information |
| Name of medicine | Xofluza |
| INN | baloxavir marboxil |
| Marketing-authorisation applicant | Roche Registration GmbH |
| Therapeutic indication | Treatment and post-exposure prophylaxis of uncomplicated influenza. |
| More information | Xofluza: Pending EC decision |
Positive recommendation on new biosimilar medicine
| Name of medicine | Onbevzi |
| INN | bevacizumab |
| Marketing-authorisation applicant | Samsung Bioepis NL B.V. |
| Therapeutic indication | Treatment of carcinoma of the colon or rectum, breast cancer, non-small cell lung cancer, renal cell cancer, epithelial ovarian, fallopian tube or primary peritoneal cancer, and carcinoma of the cervix. |
| More information | Onbevzi: Pending EC decision |
Positive recommendation for new medicine following re-examination
| Name of medicine | Elzonris |
| INN | tagraxofusp |
| Marketing-authorisation applicant | Stemline Therapeutics B.V. |
| Therapeutic indication | Treatment of blastic plasmacytoid dendritic cell neoplasm. |
| More information | Questions and answers on the approval of the marketing authorisation for Elzonris (tagraxofusp) |
Negative recommendation for new medicine following re-examination
| Name of medicine | Gamifant |
| INN | emapalumab |
| Marketing-authorisation applicant | Swedish Orphan Biovitrum AB (publ) |
| Therapeutic indication | Treatment of primary haemophagocytic lymphohistiocytosis in children under 18 years of age. |
| More information | Gamifant: Pending EC decision |
Positive recommendations on extensions of indications
| Name of medicine | Kyprolis |
| INN | carfilzomib |
| Marketing-authorisation holder | Amgen Europe B.V. |
| More information | Kyprolis: Pending EC decision |
| Name of medicine | Pradaxa |
| INN | dabigatran etexilate |
| Marketing-authorisation holder | Boehringer Ingelheim International GmbH |
| More information | Pradaxa: Pending EC decision |
| Name of medicine | Tivicay |
| INN | dolutegravir |
| Marketing-authorisation holder | ViiV Healthcare B.V. |
| More information | Tivicay: Pending EC decision |
| Name of medicine | Trimbow |
| INN | beclometasone / formoterol / glycopyrronium bromide |
| Marketing-authorisation holder | Chiesi Farmaceutici S.p.A |
| More information | Trimbow: Pending EC decision |
| Name of medicine | Xarelto |
| INN | rivaroxaban |
| Marketing-authorisation holder | Bayer AG |
| More information | Xarelto: Pending EC decision |
| Name of medicine | Xyrem |
| INN | sodium oxybate |
| Marketing-authorisation holder | UCB Pharma S.A. |
| More information | Xyrem: Pending EC decision |
Public-health recommendations
| Name of medicine | Angiotensin-II-receptor antagonists (sartans) containing a tetrazole group |
| INN | valsartan, candesartan, irbesartan, losartan and olmesartan |
| More information | Angiotensin-II-receptor antagonists (sartans) containing a tetrazole group Article-31 referral: Nitrosamines - EMA aligns recommendations for sartans with those for other medicines |
| Name of medicine | Ulipristal acetate 5mg medicinal products |
| INN | ulipristal acetate |
| More information | Ulipristal acetate 5mg medicinal products Article-31 referral: Ulipristal acetate for uterine fibroids: EMA recommends restricting use |
| Name of medicine | Puldysa |
| INN | idebenone |
| Marketing-authorisation applicant | Santhera Pharmaceuticals (Deutschland) GmbH |
| More information | Puldysa: Withdrawn application |
| Name of medicine | Roctavian |
| INN | valoctocogene roxaparvovec |
| Marketing-authorisation applicant | BioMarin International Limited |
| More information | Roctavian: Withdrawn application |
| Name of medicine | Tibsovo |
| INN | ivosidenib |
| Marketing-authorisation applicant | Agios Netherlands B.V. |
| More information | Tibsovo: Withdrawn application |