CHMP Meeting Highlights May 2023

This month, medicinal products for the following indications have received a positive opinion:

• Detection of prostate-specific membrane antigen in prostate cancer
• Epileptic seizures associated with CDKL5 deficiency disorder

New medicines recommended for approval:

Pylclari (piflufolastat (18F)): received a positive opinion for the detection of prostate-specific membrane antigen (PSMA) positive lesions with positron emission tomography (PET) in adults with prostate cancer (PCa) in the following clinical settings:

• Primary staging of patients with high-risk PCa prior to initial curative therapy
• To localize recurrence of PCa in patients with a suspected recurrence based on increasing serum prostate-specific antigen (PSA) levels after primary treatment with curative intent

Prostate cancer is the second most common cancer in men. The membrane protein PSMA is expressed in the prostate in high levels and further overexpressed in prostate cancer. Pylclari is a radioactive diagnostic agent that binds to PSMA and is labelled with a positron-emitting isotope (18F) used for imaging by PET. Therefore, by binding to PSMA, the radiopharmaceutical will be able to detect prostate cancer cells.

Pylclari is the second PSMA-targeted radiopharmaceutical for prostate cancer detection via PET authorised in the EU (after Locametz (gozetotide), authorised in the EU since December 2022). For more information please consult the product for Pylclari on the EMA website.

Ztalmy (ganaxolone): received a positive opinion for the adjunctive treatment of epileptic seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients 2 to 17 years of age. Ztalmy may be continued in patients 18 years of age and older.

CDD is a rare X-linked dominant genetic disease caused by loss-of-function mutations in the CDKL5 gene. CDKL5 is involved in brain development and function and patients with CDD present with epilepsy and severe developmental delay, including cognitive and motor impairment.

Ztalmy is a synthetic analogue of allopregnanolone, a naturally-occurring neurosteroid that positively modulates the gamma-aminobutyric acid type A (GABAA) receptor.

Ztalmy exerts its anti-seizure effects by allosterically modulating GABAA receptors. For more information please consult the product for Ztalmy on the EMA website.

Recommendations on extensions of therapeutic indication:

Opdivo (nivolumab): extension of indication to the neoadjuvant treatment of resectable non-small cell lung cancer at high risk of recurrence in adult patients whose tumours have PD-L1 expression ≥ 1%, in combination with platinum-based chemotherapy. Opdivo was already authorised for the treatment of melanoma, non-small cell lung cancer, malignant pleural mesothelioma, renal cell carcinoma, classical Hodgkin lymphoma, squamous cell cancer of the head and neck, urothelial carcinoma, colorectal cancer, oesophageal squamous cell carcinoma, and gastric, gastro-oesophageal junction and oesophageal adenocarcinoma. For more information please consult the product for Opdivo on the EMA website.

Sogroya (somapacitan): extension of indication for the replacement of endogenous growth hormone (GH) in children aged 3 years and above and adolescents with growth failure due to GH deficiency (paediatric GHD). Sogroya was already authorised in adults. For more information please consult the product for Sogroya on the EMA website.

Newly published EPARs:

The EPAR (European public assessment report) is the main document where the EMA publishes detailed information on the medicines assessed by the CHMP. Below is a list of the EPARs for recently approved products that have been made available on the EMA homepage:

Elfabrio (pegunigalsidase alfa): is indicated for long-term enzyme replacement therapy in adult patients with a confirmed diagnosis of Fabry disease (deficiency of alpha-galactosidase). EPAR Elfabrio.

Pombiliti (cipaglucosidase alfa): is a long-term enzyme replacement therapy used in combination with the enzyme stabiliser miglustat for the treatment of adults with late-onset Pompe disease (acid α-glucosidase deficiency).  EPAR Pombiliti.

Tibsovo (ivosidenib): in combination with azacitidine is indicated for the treatment of adult patients with newly diagnosed acute myeloid leukaemia with an isocitrate dehydrogenase-1 (IDH1) R132 mutation who are not eligible to receive standard induction chemotherapy.

Tibsovo monotherapy is indicated for the treatment of adult patients with locally advanced or metastatic cholangiocarcinoma with an IDH1 R132 mutation who were previously treated by at least one prior line of systemic therapy. EPAR Tibsovo.

Vafseo (vadadustat): is indicated for the treatment of symptomatic anaemia associated with chronic kidney disease in adults on chronic maintenance dialysis. EPAR Vafseo.

Recently started procedures:

• Aprocitentan - Treatment of resistant hypertension
• Buprenorphine - Treatment of opioid drug dependence
• In vitro diagnostic medical device - To determine Human Epidermal Growth Factor Receptor 2 (HER2) oncoprotein status
• Methylphenidate hydrochloride - PUMA - Treatment of Attention Deficit Hyperactivity Disorder (ADHD) in children aged 6 years of age and over
• rdESAT-6 + rCFP-10 - Diagnosis of infection with Mycobacterium tuberculosis
• Sugemalimab - Treatment of adults with metastatic non-small cell lung cancer

Further inquiry note

basg_anfragen@basg.gv.at