Meeting of the Committee for Medicinal Products for Human Use (CHMP), February 2024.

• Therapeutic indications:

  Cellemic is indicated for active immunization against the influenza A virus subtype H5N1 in adults and children from 6 months of age.

  Celldemic should be used in accordance with official recommendations.

Celldemic has been shown to induce an immune response in adults and children as young as 3 weeks of age following two doses of the vaccine given three weeks apart, as measured by hemagglutination inhibition titers against H5N1.

The most common adverse reactions in adults and children aged 6 to 18 years were pain at the injection site, fatigue, headache, malaise, myalgia and arthralgia. In children 6 months to 6 years of age, injection site pain, irritability, drowsiness, changes in eating habits, and fever have been reported.

• Therapeutic indications:

Filspari is indicated for the treatment of adults with primary immunoglobulin A nephropathy (IgAN) with proteinuria ≥1.0 g/day (or urine protein/creatinine ratio ≥0.75 g/g, see section 5.1).

In a randomized phase 3 clinical trial compared with irbesartan in adults with IgAN, Filspari was shown to reduce proteinuria and slow the progression of kidney disease.

The most common adverse reactions were hypotension, hyperkalemia, dizziness and edema. The most common serious adverse reaction was acute renal failure.

CHMP offers conditional approval for Filspari. This type of approval is granted in the interest of public health to drugs that satisfy an unmet medical need when the benefit of immediate availability outweighs the risk of having less data than would normally be required at the time the approval is granted. The marketing authorization holder undertakes to provide complete clinical data within a period of time previously agreed upon by the CHMP.

• Therapeutic indications:

  Incellipan is indicated for active immunization against influenza in the event of an officially declared pandemic.

  Incellipan should be used in accordance with official recommendations.

Incellipan has been shown to induce an immune response in adults and children three weeks after two doses of the vaccine given three weeks apart, as determined by hemagglutinin inhibition titers against H5N1.

The most common adverse reactions in adults and children aged 6 to 18 years were pain at the injection site, fatigue, headache, malaise, myalgia and arthralgia. In children from 6 months to 6 years of age, pain at the injection site, irritability, drowsiness, changes in diet, and fever were observed.

• Therapeutic indications:

  Qalsody is indicated for the treatment of adults with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene.

In a randomized, placebo-controlled clinical trial, Qalsody was shown to reduce cerebrospinal fluid SOD1 levels and plasma light chain neurofilament levels (a marker of neuronal damage), and also had a numerically beneficial effect on the ALSFRS-R score used for scoring. patients’ functional performance compared with placebo.

The most common adverse reactions were pain, fatigue, fever, arthralgia, myalgia, and increased levels of white blood cells and proteins in the cerebrospinal fluid.

Treatment with Qalsody should be prescribed by physicians experienced in treating ALS.

The CHMP proposed to allow Qalsody in exceptional circumstances. This type of approval is issued in the interest of public health when the applicant can demonstrate that it will not be able to provide complete efficacy and safety data at the post-authorization stage due to the rarity of the disease being treated. indicated, limitations of scientific knowledge in this area or ethical considerations associated with the collection of said data. The permit is subject to certain specific obligations that the laboratory must comply with and is reassessed annually.

• Therapeutic indications:

Non-small cell lung cancer (NSCLC)

Tizveni in combination with pemetrexed and platinum-based chemotherapy is indicated as first-line therapy in adult patients with non-squamous non-small cell lung cancer whose tumor expresses PD-L1 in ≥50% of tumor cells, without positive EGFR or ALK mutations, and who have :

• locally advanced NSCLC and which are not candidates for surgical resection or platinum-based chemoradiotherapy, or
• Metastatic NSCLC.

Tizveni in combination with carboplatin and paclitaxel or nab-paclitaxel is indicated for the first-line treatment of adult patients with squamous cell non-small cell lung cancer who have:

• locally advanced NSCLC and which are not candidates for surgical resection or platinum-based chemoradiotherapy, or
• Metastatic NSCLC.

Tizveni as monotherapy is indicated for the treatment of adult patients with locally advanced or metastatic non-small cell lung cancer after prior treatment with platinum agents. Patients with EGFR-mutated or ALK-positive NSCLC were also required to have received targeted therapy before receiving tislelizumab.

Tizvani demonstrated improvements in overall survival and progression-free survival in patients with locally advanced or metastatic non-small cell lung cancer in three randomized phase 3 clinical trials that compared Tizvani (as monotherapy or in combination) with chemotherapy.

The most common adverse reactions were anemia, fatigue and increased AST levels.

• Therapeutic indications:

  Voidea is indicated in addition to ravulizumab or eculizumab for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH) with residual hemolytic anemia (see section 5.1).

Voidea added to treatment with C5 inhibitors has been shown to prevent hemolysis and increase hemoglobin levels, as observed in a pivotal phase 3 randomized, placebo-controlled trial.

The most common adverse reactions were fever, headache, elevated liver enzymes, and pain in the extremities.

Treatment with Voidea should be prescribed by physicians experienced in the management of patients with hematological disorders.

• Therapeutic indications:

ZYNYZ monotherapy is indicated as first-line therapy in adult patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC) who are refractory to curative surgery or radiation therapy.

Ziniz demonstrated efficacy in terms of objective response rate and duration of response in patients with recurrent locally advanced or metastatic Merkel cell carcinoma refractory to surgery or radiation therapy.

Treatment with Ziniz should be initiated and supervised by a physician experienced in the treatment of cancer.