Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 20 - 23 February 2023

News 24/02/2023

Eight new medicines recommended for approval

EMA’s human medicines committee (CHMP) recommended granting a marketing authorisation for Akeega (niraparib/abiraterone acetate) for the treatment of metastatic castration-resistant prostate cancer with BRCA 1/BRCA 2 mutations.

The committee adopted a positive opinion for Elfabrio* (pegunigalsidase alfa) for the treatment of Fabry disease, a rare genetic disorder that results from the build-up of a type of fat in the body cells.

Hyftor* (sirolimus) received a positive opinion from the CHMP for the treatment of facial angiofibroma, a benign vascular skin lesion composed of dermal fibrous tissue and blood vessels. Hyftor is indicated for the treatment of adults and children above six years of age.

The CHMP adopted a positive opinion for Opzelura (ruxolitinib) for the treatment of non-segmental vitiligo, a skin disorder characterised by depigmented patches of skin. Opzelura is indicated for the treatment of adults and adolescents from 12 years of age.

The committee adopted a positive opinion for Tibsovo* (ivosidenib) for the treatment of newly diagnosed acute myeloid leukaemia, a cancer of the blood and bone marrow, and for the treatment of locally advanced or metastatic cholangiocarcinoma, a type of cancer that forms in the bile ducts. The committee also adopted a positive opinion for its duplicate Tidhesco* (ivosidenib) for the treatment of newly diagnosed acute myeloid leukaemia.

The CHMP gave a positive opinion to Vafseo (vadadustat) for the treatment of symptomatic anaemia in adults with chronic kidney disease who are on maintenance dialysis.

The biosimilar medicine Bekemv (eculizumab) received a positive opinion for the treatment of paroxysmal nocturnal haemoglobinuria in adults and children. Paroxysmal nocturnal haemoglobinuria is a life-threatening genetic disease that causes the breakdown of red blood cells resulting in various medical complications. Bekemv is the first biosimilar approved for this active substance. Unlike the reference product, Bekemv is contraindicated in patients with hereditary fructose intolerance and in children below two years of age.

Recommendations on extensions of therapeutic indication for four medicines

The committee recommended four extensions of indication for medicines that are already authorised in the European Union (EU): Esbriet, Libtayo, Rinvoq and TachoSil.

Withdrawals of applications

The application to extend the therapeutic indication of Buvidal to treat chronic (long-term) pain in people with dependence on opioids was withdrawn. A question-and-answer document on the withdrawal is available in the grid below.

Re-examination of recommendation

The applicant for Sohonos* has requested a re-examination of EMA’s opinion of January 2023. Upon receipt of the grounds of the request, the Agency will re-examine its opinion and issue a final recommendation.

COVID-19 updates

The committee recommended authorising the use of COVID-19 Vaccine Valneva (inactivated, adjuvanted) as a booster dose for adults 18 to 50 years of age.

An overview of all the COVID-19 vaccines authorised in the EU is available on EMA’s website.

The committee recommended the refusal of a marketing authorisation for Lagevrio (molnupiravir) for the treatment of COVID-19 in adults.

The review of the marketing authorisation application for Lagevrio had started on 23 November 2021, following advice intended to support decisions at Member State level to enable possible early use of the medicine prior to marketing authorisation. Having evaluated the interim data available at the time of the advice, and all additional data provided by the company since then, the CHMP concluded that the clinical benefit of Lagevrio in the treatment of adults with COVID-19 who are not receiving supplemental oxygen and who are at increased risk of developing severe COVID-19 could not be demonstrated. Based on the totality of data, it was not possible to conclude that Lagevrio can reduce the risk of hospitalisation or death or shorten the duration of illness or time to recovery in adults at risk of severe disease. Furthermore, it was not possible to identify a specific group of patients in whom a clinically relevant benefit of Lagevrio could be demonstrated. For more information on this negative opinion, see the question-and-answer document in the grid below.

Agenda and minutes

The agenda of the February 2023 CHMP meeting is published on EMA's website. Minutes of the January 2023 CHMP meeting will be published in the coming weeks.

CHMP statistics

Key figures from the February 2023 CHMP meeting are represented in the graphic below.

CHMP statistics for February 2023, 8 positive opinions for new medicines and 1 negative opinion. 4 positive opinions on extensions of therapeutic indications and 0 withdrawn applications for new medicines.

*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 medicineAkeega
International non-proprietary name (INN)niraparib / abiraterone acetate
Marketing-authorisation applicantJanssen-Cilag International N.V.
Therapeutic indicationTreatment of metastatic castration-resistant prostate cancer with BRCA 1/BRCA 2 mutations
More informationAkeega: Pending EC decision


Name of medicineElfabrio
INNpegunigalsidase alfa
Marketing-authorisation applicantChiesi Farmaceutici S.p.A.
Therapeutic indicationTreatment of Fabry disease
More informationElfabrio: Pending EC decision


Name of medicineHyftor
Marketing-authorisation applicantPlusultra pharma GmbH
Therapeutic indicationTreatment of facial angiofibroma
More informationHyftor: Pending EC decision


Name of medicineOpzelura
Marketing-authorisation applicantIncyte Biosciences Distribution B.V.
Therapeutic indicationTreatment of non-segmental vitiligo
More informationOpzelura: Pending EC decision


Name of medicineTibsovo
Marketing-authorisation holderLes Laboratoires Servier
Therapeutic indicationTreatment of adult patients with newly diagnosed acute myeloid leukaemia (AML) and for the treatment of locally advanced or metastatic cholangiocarcinoma
More informationTibsovo: Pending EC decision


Name of medicineTidhesco
Marketing-authorisation holderLes Laboratoires Servier
Therapeutic indicationTreatment of newly diagnosed acute myeloid leukaemia
More informationTidhesco: Pending EC decision


Name of medicineVafseo 
Marketing-authorisation holderAKEBIA EUROPE Limited
Therapeutic indicationTreatment of symptomatic anaemia in adults with chronic kidney disease who are on maintenance dialysis
More informationVafseo: Pending EC decision


Positive recommendation on new biosimilar medicine

Name of medicineBekemv
Marketing-authorisation holderAmgen Technology (Ireland) Unlimited Company
Therapeutic indicationTreatment of paroxysmal nocturnal haemoglobinuria
More informationBekemv: Pending EC decision


Positive recommendations on extensions of indications

Name of medicineEsbriet
Marketing-authorisation holderRoche Registration GmbH
More informationEsbriet: Pending EC decision


Name of medicineLibtayo
Marketing-authorisation holderRegeneron Ireland Designated Activity Company (DAC)
More informationLibtayo: Pending EC decision


Name of medicineRinvoq
Marketing-authorisation holderAbbVie Deutschland GmbH & Co. KG
More informationRinvoq: Pending EC decision


Name of medicineTachoSil
INNhuman thrombin / human fibrinogen
Marketing-authorisation holderCorza Medical GmbH
More informationTachoSil: Pending EC decision


Withdrawal of post-authorisation marketing authorisation application

Name of medicineBuvidal
Marketing-authorisation applicantCamurus AB
More informationBuvidal: Questions and answers


Re-examination of recommendation for new medicine

Name of medicineSohonos
Marketing-authorisation applicantIpsen Pharma
More informationSohonos: Questions and answers


Negative recommendation on new medicine

Name of medicineLagevrio
Marketing-authorisation applicantMerck Sharp & Dohme B.V.
More informationLagevrio: Questions and answers


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