Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 9-12 October 2023
NewsHumanMedicinesReferrals
Seven new medicines recommended for approval
EMA’s human medicines committee (CHMP) recommended seven medicines for approval at its October 2023 meeting.
The CHMP adopted a positive opinion for Agamree* (vamorolone), for the treatment of Duchenne muscular dystrophy, a genetic disorder characterised by the progressive loss of muscle function.
The committee recommended granting a conditional marketing authorisation for Elrexfio* (elranatamab), for the treatment of adult patients with relapsed or refractory multiple myeloma, a rare cancer of the bone marrow that affects plasma cells, a type of white blood cell that produces antibodies. This medicine was supported through EMA’s Priority Medicines (PRIME) scheme, which provides early and enhanced scientific and regulatory support for promising medicines with a potential to address unmet medical needs. See more details in the news announcement in the grid below.
The committee granted a positive opinion for Elucirem (gadopiclenol) and its duplicate Vueway, two diagnostic medicines to improve detection, visualisation and assist in the characterisation of lesions in the central nervous system and in other body regions, including the breast, the liver and the prostate.
Loargys* (pegzilarginase), received a positive opinion under exceptional circumstances for the treatment of hyperargininemia, a rare disease with neurological clinical signs including spasticity, ataxia, hyperreflexia, incoordination, and seizures.
The CHMP recommended granting a marketing authorisation for Rezzayo* (rezafungin), for the treatment of invasive candidiasis, a serious fungal infection caused by Candida.
The committee adopted a positive opinion for Veoza (fezolinetant), for the treatment of moderate-to-severe vasomotor symptoms (hot flushes) associated with menopause.
Recommendations on extensions of therapeutic indication for 8 medicines
The committee recommended 8 extensions of indication for medicines that are already authorised in the European Union (EU): Brukinsa, Imfinzi, Jemperli, Keytruda, Praluent, Prevymis*, Rubraca, and Veyvondi.
Withdrawals of applications
Two applications for marketing authorisation were withdrawn during their assessment: Jivadco, for the treatment of breast cancer, and Sugammadex Lorien, for the reversal of neuromuscular blockade induced by rocuronium or vecuronium. Question-and-answer documents on these withdrawals are available in the grid below.
One application to extend the therapeutic indications was withdrawn:
- RoActemra, for the use of this medicine in the treatment of interstitial lung disease, disorders causing scarring in the lungs, associated with systemic sclerosis.
Question-and-answer documents on these withdrawals to extend therapeutic indications are available in the grid below.
Start of re-examinations
The marketing authorisation holders for Translarna* and Blenrep* have requested a re-examination of EMA’s opinions of September 2023. Upon receipt of the grounds of the requests, the Agency will re-examine its opinions and issue final recommendations.
Outcome of re-examination
Following a re-examination, the CHMP confirmed its original recommendation to refuse the granting of a marketing authorisation for Albrioza* (sodium phenylbutyrate/ursodoxicoltaurine). This medicine was intended for the treatment of amyotrophic lateral sclerosis, a rare neurological disease affecting nerve cells in the brain and spinal cord that control voluntary muscle movement. For more information on this re-examination opinion, see the question-and-answer document in the grid below.
Start of referral
The CHMP started a review of all available information on the benefits and risks of Ocaliva* (obeticholic acid), a medicine used to treat adults with primary biliary cholangitis, an autoimmune condition that causes gradual destruction of the small bile ducts in the liver, which can lead to liver failure and increase the risk of liver cancer.
The review is carried out under Article 20 of Regulation (EC) No 726/2004. For more information, see the public health communication in the grid below.
Agenda and minutes
The agenda of the October 2023 CHMP meeting is published on EMA's website. Minutes of the meeting will be published in the coming weeks.
CHMP statistics
Key figures from the October 2023 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 | Agamree |
|---|---|
| International non-proprietary name (INN) | vamorolone |
| Marketing-authorisation applicant | Santhera Pharmaceuticals (Deutschland) GmbH |
| Therapeutic indication | Treatment of Duchenne muscular dystrophy (DMD) |
| More information | Agamree: pending EC decision |
| Name of medicine | Elrexfio |
|---|---|
| INN | elranatamab |
| Marketing-authorisation applicant | Pfizer Europe MA EEIG |
| Therapeutic indication | Treatment of adult patients with relapsed or refractory multiple myeloma |
| More information | Elrexfio: pending EC decision |
| Name of medicine | Elucirem |
|---|---|
| INN | gadopiclenol |
| Marketing-authorisation applicant | Guerbet |
| Therapeutic indication | For diagnostic: contrast-enhanced magnetic resonance imaging (MRI) to improve detection, visualization and assist in characterization of lesions in the central nervous system and in other body regions (including breast, liver and prostate). |
| More information | Elucirem: pending EC decision |
| Name of medicine | Loargys |
|---|---|
| INN | pegzilarginase |
| Marketing-authorisation applicant | Immedica Pharma AB |
| Therapeutic indication | Treatment of hyperargininemia |
| More information | Loargys: pending EC decision |
| Name of medicine | Rezzayo |
|---|---|
| INN | rezafungin |
| Marketing-authorisation applicant | Mundipharma GmbH |
| Therapeutic indication | Treatment of invasive candidiasis |
| More information | Rezzayo: pending EC decision |
| Name of medicine | Veoza |
|---|---|
| INN | fezolinetant |
| Marketing-authorisation applicant | Astellas Pharma Europe B.V. |
| Therapeutic indication | Treatment of moderate to severe vasomotor symptoms (VMS) associated with menopause |
| More information | Veoza: pending EC decision |
| Name of medicine | Vueway |
|---|---|
| INN | gadopiclenol |
| Marketing-authorisation applicant | Bracco Imaging S.p.A. |
| Therapeutic indication | For diagnostic: contrast-enhanced magnetic resonance imaging (MRI) to improve detection, visualization and assist in characterization of lesions in the central nervous system and in other body regions (including breast, liver and prostate) |
| More information | Vueway: pending EC decision |
| Name of medicine | Jivadco |
|---|---|
| INN | trastuzumab duocarmazine |
| Marketing-authorisation holder | medac Gesellschaft fur klinische Spezialpraparate mbH |
| More information | Jivadco: withdrawn application |
| Name of medicine | Sugammadex Lorien |
|---|---|
| INN | sugammadex |
| Marketing-authorisation holder | LABORATORIOS LORIEN, S.L. |
| More information | Sugammadex Lorien: withdrawn application |
Outcome of re-examination
| Name of medicine | Albrioza |
|---|---|
| INN | sodium phenylbutyrate / ursodoxicoltaurine |
| Marketing-authorisation applicant | Amylyx Pharmaceuticals EMEA B.V. |
| Therapeutic indication | Treatment of amyotrophic lateral sclerosis (ALS) |
| More information | Albrioza: pending EC decision |
| Name of medicine | Translarna |
|---|---|
| INN | ataluren |
| Marketing-authorisation holder | PTC Therapeutics International Limited |
| More information | EMA recommends non-renewal of authorisation of Duchenne muscular dystrophy medicine Translarna |
| Name of medicine | Blenrep |
|---|---|
| INN | belantamab mafodotin |
| Marketing-authorisation holder | GlaxoSmithKline (Ireland) Limited |
| More information | EMA recommends non-renewal of authorisation of multiple myeloma medicine Blenrep |
Positive recommendations on extensions of indications
| Name of medicine | Brukinsa |
|---|---|
| INN | zanubrutinib |
| Marketing-authorisation holder | BeiGene Ireland Ltd |
| More information | Brukinsa: pending EC decision |
| Name of medicine | Imfinzi |
|---|---|
| INN | durvalumab |
| Marketing-authorisation holder | AstraZeneca AB |
| More information | Imfinzi: pending EC decision |
| Name of medicine | Jemperli |
|---|---|
| INN | dostarlimab |
| Marketing-authorisation holder | GlaxoSmithKline (Ireland) Limited |
| More information | Jemperli: pending EC decision |
| Name of medicine | Keytruda |
|---|---|
| INN | pembrolizumab |
| Marketing-authorisation holder | Merck Sharp & Dohme B.V. |
| More information | Keytruda: pending EC decision |
| Name of medicine | Praluent |
|---|---|
| INN | alirocumab |
| Marketing-authorisation applicant | Sanofi Winthrop Industrie |
| More information | Praluent: pending EC decision |
| Name of medicine | Prevymis |
|---|---|
| INN | letermovir |
| Marketing-authorisation applicant | Merck Sharp & Dohme B.V. |
| More information | Prevymis: pending EC decision |
| Name of medicine | Rubraca |
|---|---|
| INN | rucaparib |
| Marketing-authorisation holder | Zr Pharma& GmbH |
| More information | Rubraca: pending EC decision |
| Name of medicine | Veyvondi |
|---|---|
| INN | vonicog alfa |
| Marketing-authorisation applicant | Baxalta Innovations GmbH |
| More information | Veyvondi: pending EC decision |
| Name of medicine | RoActemra |
|---|---|
| INN | tocilizumab |
| Marketing-authorisation holder | Roche Registration GmbH |
| More information | RoActemra: Withdrawal of the application to change the marketing authorisation |
Start of referral
| Name of medicine | Ocaliva |
|---|---|
| INN | Obeticholic acid |
| Marketing-authorisation holder | ADVANZ PHARMA Limited |
| More information | Ocaliva: Article 20 referral |