Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 20 - 23 February 2023
NewsHumanCOVID-19Medicines
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.
*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 | Akeega |
| International non-proprietary name (INN) | niraparib / abiraterone acetate |
| Marketing-authorisation applicant | Janssen-Cilag International N.V. |
| Therapeutic indication | Treatment of metastatic castration-resistant prostate cancer with BRCA 1/BRCA 2 mutations |
| More information | Akeega: Pending EC decision |
| Name of medicine | Elfabrio |
| INN | pegunigalsidase alfa |
| Marketing-authorisation applicant | Chiesi Farmaceutici S.p.A. |
| Therapeutic indication | Treatment of Fabry disease |
| More information | Elfabrio: Pending EC decision |
| Name of medicine | Hyftor |
| INN | sirolimus |
| Marketing-authorisation applicant | Plusultra pharma GmbH |
| Therapeutic indication | Treatment of facial angiofibroma |
| More information | Hyftor: Pending EC decision |
| Name of medicine | Opzelura |
| INN | ruxolitinib |
| Marketing-authorisation applicant | Incyte Biosciences Distribution B.V. |
| Therapeutic indication | Treatment of non-segmental vitiligo |
| More information | Opzelura: Pending EC decision |
| Name of medicine | Tibsovo |
| INN | ivosidenib |
| Marketing-authorisation holder | Les Laboratoires Servier |
| Therapeutic indication | Treatment of adult patients with newly diagnosed acute myeloid leukaemia (AML) and for the treatment of locally advanced or metastatic cholangiocarcinoma |
| More information | Tibsovo: Pending EC decision |
| Name of medicine | Tidhesco |
| INN | ivosidenib |
| Marketing-authorisation holder | Les Laboratoires Servier |
| Therapeutic indication | Treatment of newly diagnosed acute myeloid leukaemia |
| More information | Tidhesco: Pending EC decision |
| Name of medicine | Vafseo |
| INN | vadadustat |
| Marketing-authorisation holder | AKEBIA EUROPE Limited |
| Therapeutic indication | Treatment of symptomatic anaemia in adults with chronic kidney disease who are on maintenance dialysis |
| More information | Vafseo: Pending EC decision |
Positive recommendation on new biosimilar medicine
| Name of medicine | Bekemv |
| INN | eculizumab |
| Marketing-authorisation holder | Amgen Technology (Ireland) Unlimited Company |
| Therapeutic indication | Treatment of paroxysmal nocturnal haemoglobinuria |
| More information | Bekemv: Pending EC decision |
Positive recommendations on extensions of indications
| Name of medicine | Esbriet |
| INN | pirfenidone |
| Marketing-authorisation holder | Roche Registration GmbH |
| More information | Esbriet: Pending EC decision |
| Name of medicine | Libtayo |
| INN | cemiplimab |
| Marketing-authorisation holder | Regeneron Ireland Designated Activity Company (DAC) |
| More information | Libtayo: Pending EC decision |
| Name of medicine | Rinvoq |
| INN | upadacitinib |
| Marketing-authorisation holder | AbbVie Deutschland GmbH & Co. KG |
| More information | Rinvoq: Pending EC decision |
| Name of medicine | TachoSil |
| INN | human thrombin / human fibrinogen |
| Marketing-authorisation holder | Corza Medical GmbH |
| More information | TachoSil: Pending EC decision |
| Name of medicine | Buvidal |
| INN | buprenorphine |
| Marketing-authorisation applicant | Camurus AB |
| More information | Buvidal: Questions and answers |
Re-examination of recommendation for new medicine
| Name of medicine | Sohonos |
| INN | palovarotene |
| Marketing-authorisation applicant | Ipsen Pharma |
| More information | Sohonos: Questions and answers |
Negative recommendation on new medicine
| Name of medicine | Lagevrio |
| INN | molnupiravir |
| Marketing-authorisation applicant | Merck Sharp & Dohme B.V. |
| More information | Lagevrio: Questions and answers |