In the month of June 2024, medicinal products for the following indications have received a positive opinion:

• Urothelial carcinoma
• Emergency treatment of allergic reactions (anaphylaxis)
• Prevention of lower respiratory tract disease (LRTD) caused by Respiratory Syncytial Virus
• Follicular lymphoma and diffuse large B-cell lymphoma
• Paroxysmal nocturnal haemoglobinuria (PNH)
• Positron emission tomography (PET) imaging of the brain
• Pulmonary arterial hypertension

New medicines recommended for approval

Balversa (erdafitinib) has received a positive opinion, as monotherapy, for the treatment of adult patients with unresectable or metastatic urothelial carcinoma (UC), harbouring susceptible FGFR3 genetic alterations who have previously received at least one line of therapy containing a PD-1 or PD-L1 inhibitor in the unresectable or metastatic treatment setting.

UC is an aggressive cancer of the bladder or other parts of the urinary tract. Risk factors include smoking, exposure to toxins, and Schistosomiasis infection (mostly in Africa and the Middle East). Metastatic UC is incurable and has a poor prognosis.

Alterations in the gene encoding for fibroblast growth factor receptor (FGFR) are common in UC and thus, FGFR emerged as a novel therapeutic target for the treatment of metastatic UC. Erdafitinib is the first inhibitor targeting all isoforms of FGFR.

Eurneffy (epinephrine) has received a positive opinion for the emergency treatment of allergic reactions (anaphylaxis) due to insect stings or bites, foods, medicinal products and other allergens as well as idiopathic or exercise induced anaphylaxis. Treatment is indicated for adults and children with a body weight ≥ 30 kg.

Anaphylaxis is a severe and life-threatening allergic reaction. It requires an immediate medical intervention. Epinephrine, also known as adrenaline, counteracts the decrease in blood pressure and the obstruction of the blood vessels in the lung, which both happens during an anaphylactic reaction. There are several authorised epinephrine-containing products, which need to be injected in the case of emergency. Eurneffy is the first nasal epinephrine spray which has been authorised. The EMA has published a press release on Eurneffy.

mResvia (Respiratory Syncytial Virus (RSV) mRNA vaccine) has received a positive opinion for the active immunisation for the prevention of lower respiratory tract disease (LRTD) caused by Respiratory Syncytial Virus in adults 60 years of age and older.

Respiratory Syncytial Virus (RSV) is a single-stranded RNA virus that causes the majority of cases of respiratory hospitalisation in infants. In the elderly population, approximately 15% of acute respiratory infections due to RSV lead to hospitalisation. Lower respiratory tract disease is characterised by bronchiolitis and pneumonia, and is potentially life-threatening. mResvia is a mRNA-based vaccine that encodes for RSV glycoprotein F. This protein is the target of neutralising antibodies that mediate protection against RSV-associated respiratory tract disease.

Ordspono (odronextamab) has received a positive opinion for a conditional marketing authorisation (CMA) for the following indications:

Ordspono as monotherapy is indicated for the treatment of adult patients with relapsed or refractory follicular lymphoma (r/r FL) after two or more lines of systemic therapy.

Ordspono as monotherapy is indicated for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (r/r DLBCL) after two or more lines of systemic therapy.

Follicular lymphoma (FL) and diffuse large B-cell lymphoma (DLBCL) are two subtypes of non-Hodgkin lymphoma (NHL), a heterogeneous group of lymphoid malignancies deriving from B-cells or T-cells. FL and DLBCL are B-cell lymphomas.

Like many mature B-cell lymphomas, FL and DLBCL express the surface antigen CD20. Odronextamab is a bispecific antibody that targets CD20 and CD3, a subunit of the T-cell receptor complex. Odronextamab binds CD3, thereby eliciting recruitment of effector T-cells. Upon concurrent binding with CD20, T-cell activation occurs, resulting in T-cell-mediated tumour cell lysis.

Piasky (crovalimab) has received a positive opinion, as monotherapy, for the treatment of adult and paediatric patients 12 years of age or older with a weight of 40 kg and above with paroxysmal nocturnal haemoglobinuria (PNH):

• In patients with haemolysis with clinical symptom(s) indicative of high disease activity.
• In patients who are clinically stable after having been treated with a complement component 5 (C5) inhibitor for at least the past 6 months.

PNH is a life-threatening blood disorder characterised by the destruction of red blood cells by the complement system, a component of the immune system. This occurs most commonly as a result of a somatic mutation that alters the exterior surface proteins of red blood cells, rendering these susceptible to be attacked by the complement.

Crovalimab is a monoclonal antibody targeting the complement protein C5, thereby protecting it from cleavage. Thus, the complement system initiation is blocked and intravascular haemolysis is reduced.

Tauvid (flortaucipir (¹⁸F)) has received a positive opinion for the following indication:

This medicinal product is for diagnostic use only.

Flortaucipir (¹⁸F) is a radiopharmaceutical indicated for positron emission tomography (PET) imaging of the brain to assess the neocortical distribution of aggregated tau neurofibrillary tangles (NFTs) in adult patients with cognitive impairment who are being evaluated for Alzheimer’s disease (AD). Flortaucipir (¹⁸F) is an adjunct to clinical and other diagnostic evaluations.

PET imaging is a diagnostic procedure which is used, e.g., for the detection of Alzheimer’s disease. To this end, certain Alzheimer-specific structures in the brain need to be labelled with radioactive, positron-emitting molecules. Tauvid contains radioactive fluor (¹⁸F) and binds to these structures, enabling the diagnostic evaluation.

Winrevair (sotatercept) has received a positive opinion, in combination with other pulmonary arterial hypertension (PAH) therapies, for the treatment of PAH in adult patients with WHO Functional Class (FC) II to III, to improve exercise capacity.

Pulmonary arterial hypertension (PAH) is high blood pressure in the blood vessels which lead from the heart to the lung. PAH is a rare condition and is associated with difficulty to breathe, chest pain and dizziness. As the heart needs to work harder to pump blood into the lung, it may eventually be affected. PAH can be a life-threatening disease.

Sotatercept is a recombinant protein and an activin inhibitor. Activin is overexpressed in PAH and promotes the growth of new blood vessels in the lungs, which results in narrowing and thickening of vessel walls. The treatment with sotatercept is expected to improve exercise capacity of patients. Winrevair is a first-in-class product and the EMA has published a press release.

Recommendations on extensions of therapeutic indication

Betmiga (mirabegron): extension of indication to include the treatment of neurogenic detrusor overactivity (NDO) in paediatric patients aged 3 to less than 18 years.

Betmiga is already authorised for adults with overactive bladder syndrome.

Beyfortus (nirsevimab): extension of indication to include the prevention of Respiratory Syncytial Virus (RSV) lower respiratory tract disease in children up to 24 months of age who remain vulnerable to severe RSV disease through their second RSV season.

Beyfortus is already authorised for neonates and infants during their first RSV season.

Cresemba (isavuconazole): extension of indication to include the treatment of patients from 1 year of age and older (pharmaceutical form: powder for concentrate for solution for infusion) and of adults and paediatric patients from 6 years of age (pharmaceutical form: hard capsules) for the treatment of

• invasive aspergillosis
• mucormycosis in patients for whom amphotericin B is inappropriate

Cresemba is already authorised for the treatment of both infections in adults, as powder for concentrate for solution for infusion.

Imcivree (setmelanotide): extension of indication to include the treatment of obesity and the control of hunger associated with genetically confirmed Bardet-Biedl syndrome (BBS), loss-of-function biallelic proopiomelanocortin (POMC), including PCSK1, deficiency or biallelic leptin receptor (LEPR) deficiency in adults and children 2 years of age and above.

Imcivree is already authorised in children from 6 years of age.

Imfinzi (durvalumab): extension of indication to include the following indication:

Imfinzi in combination with carboplatin and paclitaxel is indicated for the first-line treatment of adults with primary advanced or recurrent endometrial cancer who are candidates for systemic therapy, followed by maintenance treatment with:

• Imfinzi as monotherapy in endometrial cancer that is mismatch repair deficient (dMMR)
• Imfinzi in combination with olaparib in endometrial cancer that is mismatch repair proficient (pMMR).

Imfinzi is already authorised for non-small cell lung cancer, small cell lung cancer, biliary tract cancer and hepatocellular carcinoma.

Infanrix hexa (diphtheria (D), tetanus (T), pertussis (acellular, component) (Pa), hepatitis B (rDNA) (HBV), poliomyelitis (inactivated) (IPV) and Haemophilus influenzae type b (Hib) conjugate vaccine (adsorbed)): extension of indication to include the use as primary and booster vaccination of infants from the age of 6 weeks and toddlers against diphtheria, tetanus, pertussis, hepatitis B, poliomyelitis and disease caused by Haemophilus influenzae type b.

Infanrix hexa was already authorised without a specified lower age limit.

Lynparza (olaparib): extension of indication to include, in combination with durvalumab, the maintenance treatment of adult patients with primary advanced or recurrent endometrial cancer that is mismatch repair proficient (pMMR) whose disease has not progressed on first-line treatment with durvalumab in combination with carboplatin and paclitaxel.

Lynparza is already authorised for ovarian cancer, breast cancer, adenocarcinoma of the pancreas and prostate cancer.

Pegasys (peginterferon alfa-2a): extension of indication to include the following indications:

• Pegasys is indicated as monotherapy in adults for the treatment of polycythaemia vera.
• Pegasys is indicated as monotherapy in adults for the treatment of essential thrombocythaemia.

Pegasys is already authorised for the treatment of chronic hepatitis B.

Tepkinly (epcoritamab): extension of indication to include the monotherapy treatment of adult patients with relapsed or refractory follicular lymphoma (FL) after two or more lines of systemic therapy.

Tepkinly is already authorised for the treatment of diffuse large B-cell lymphoma (DLBCL).

Vabysmo (faricimab): extension of indication to include the treatment of visual impairment due to macular oedema secondary to retinal vein occlusion (branch RVO or central RVO) in adult patients.

Vabysmo is already authorised for neovascular (wet) age-related macular degeneration and visual impairment due to diabetic macular oedema.

Xalkori (crizotinib): extension of indication to include the following indications:

The treatment of paediatric patients (age ≥1 to <6 years) with relapsed or refractory systemic anaplastic lymphoma kinase (ALK)-positive anaplastic large cell lymphoma (ALCL)

The treatment of paediatric patients (age ≥1 to <6 years) with recurrent or refractory anaplastic lymphoma kinase (ALK)-positive unresectable inflammatory myofibroblastic tumour (IMT)

Xalkori is already authorised in children from ≥6 to <18 years in the same indications as listed above, as well as in adults with ALK- and ROS1-positive non-small cell lung cancer (NSCLC).

Newly published EPARs

The European public assessment report (EPAR) 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:

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

EPAR Qalsody

Fabhalta (iptacopan) is indicated as monotherapy in the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who have haemolytic anaemia.

EPAR Fabhalta

Recently started procedures

Every month, new marketing authorization applications are submitted to EMA under the centralised authorisation procedure. The CHMP carries out a scientific assessment of the applications and gives a recommendation. The CHMP has started the assessment of the following submitted applications:

Initial marketing authorisation applications:

• Datopotamab - Treatment of adult patients with inoperable or metastatic HR-positive / HER2-negative breast cancer with disease progression following chemotherapy in the metastatic setting
• Datopotamab - Treatment of adult patients with locally advanced or metastatic non squamous non-small cell lung cancer (NSCLC)
• Resminostat - Treatment of patients with advanced stage mycosis fungoides (MF) and Sézary syndrome (SS)
• Seladelpar lysine dihydrate - Treatment of primary biliary cholangitis (PBC) including pruritus in adults without cirrhosis or with compensated cirrhosis (Child-Pugh A) in combination with ursodeoxycholic acid (UDCA) who have an inadequate response to UDCA alone, or as monotherapy in those unable to tolerate UDCA
• Nirogacestat - Treatment of desmoid tumours
• Resmetirom - Treatment of adults with non-alcoholic steatohepatitis (NASH)/metabolic dysfunctionassociated steatohepatitis (MASH) with liver fibrosis

More Information

Previous issue of the CHMP Highlights

EMA Information: Agenda, Minutes und Highlights of the CHMP