Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 22-25 May 2023
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Two new medicines recommended for approval
EMA’s human medicines committee (CHMP) recommended two medicines for approval at its May 2023 meeting.
The committee recommended granting a marketing authorisation for Pylclari (piflufolastat (18F)), intended for the diagnosis of prostate cancer. The benefit of this medicinal product is its potential to diagnose prostate cancer during primary staging of patients at high risk and in the staging of patients with a suspected recurrence.
Ztalmy* (ganaxolone) received a positive opinion from the CHMP for the treatment of epileptic seizures associated with cyclin-dependent kinase-like 5 deficiency disorder, a genetic disorder defined by seizures beginning in infancy.
Recommendations on extensions of therapeutic indication for two medicines
The committee recommended two extensions of indication for medicines that are already authorised in the EU: Opdivo and Sogroya*.
Re-examinations
Following a re-examination, the CHMP confirmed its original recommendation to refuse the granting of a marketing authorisation for Sohonos* (palovarotene). This medicine was intended to treat fibrodysplasia ossificans progressiva, a rare genetic disease that causes extra bone to form in places outside the skeleton, such as in joints, muscles, tendons and ligaments, leading to progressively decreasing mobility and other severe impairments. For more information on this re-examination opinion, see the question-and-answer document in the grid below.
Withdrawals of applications
Two applications for marketing authorisation were withdrawn: Asimtufii, as a maintenance treatment of schizophrenia, and Susvimo, for the treatment of neovascular age-related macular degeneration in adults.
Question-and-answer documents on the withdrawals are available in the grid below.
Outcome of referral
The committee has recommended that Adakveo (crizanlizumab) should no longer be used to prevent painful crises in patients aged 16 years and older with sickle cell disease, a genetic condition in which the red blood cells become rigid and sticky and change from being disc-shaped to being crescent-shaped (like a sickle). This follows a review by the CHMP, which concluded that the benefits of the medicine did not outweigh its risks. The review looked at results of a clinical trial study, which compared the effectiveness and safety of Adakveo with placebo (a dummy treatment) in patients who had previously had painful crises leading to a healthcare visit. The study showed that Adakveo did not lead to a decrease when compared to the placebo. For more information, see the public health communication in the grid below.
Agenda and minutes
The agenda of the May 2023 CHMP meeting is published on EMA's website. Minutes of the April 2023 CHMP meeting will be published in the coming weeks.
CHMP statistics
Key figures from the May 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 | Pylclari |
|---|---|
| INN | piflufolastat (18F) |
| Marketing-authorisation applicant | Curium Pet France |
| Therapeutic indication | Imaging in patients undergoing oncologic diagnostic procedures when increased expression of prostate specific membrane antigen is a diagnostic target (new active substance) |
| More information | Pylclari: Pending EC decision |
| Name of medicine | Ztalmy |
|---|---|
| INN | ganaxolone |
| Marketing-authorisation applicant | Marinus Pharmaceuticals Emerald Limited |
| Therapeutic indication | Treatment of epileptic seizures associated with cyclindependent kinase-like 5 deficiency disorder (CDD) (new active substance) |
| More information | Ztalmy: Pending EC decision |
Positive recommendations on extensions of indications
| Name of medicine | Opdivo |
|---|---|
| INN | nivolumab |
| Marketing-authorisation holder | Bristol-Myers Squibb Pharma EEIG |
| More information | Opdivo: Pending EC decision |
| Name of medicine | Sogroya |
|---|---|
| INN | somapacitan |
| Marketing-authorisation holder | Novo Nordisk A/S |
| More information | Sogroya: Pending EC decision |
Outcome of re-examination
| Name of medicine | Sohonos |
|---|---|
| INN | palovarotene |
| Marketing-authorisation holder | Ipsen Pharma |
| More information | Sohonos: Pending EC decision |
| Name of medicine | Asimtufii |
|---|---|
| INN | aripiprazole |
| Marketing-authorisation holder | Otsuka Pharmaceutical Netherlands B.V. |
| More information | Asimtufii: Withdrawn application |
| Name of medicine | Susvimo |
|---|---|
| INN | ranibizumab |
| Marketing-authorisation holder | Roche Registration GmbH |
| More information | Susvimo: Withdrawn application |
Conclusion of referral
| Name of medicine | Adakveo |
|---|---|
| INN | crizanlizumab |
| Marketing-authorisation applicant | Novartis Europharm Limited |
| More information | Adakveo |