CHMP Meeting Highlights April 2025 New
This month, medicinal products for the following indications have received a positive opinion:
- Cystic fibrosis
- Hereditary transthyretin-mediated amyloidosis
- Duchenne muscular dystrophy
- Phenylketonuria
- Thyroid eye disease
- Biliary tract cancer
- Acromegaly
New medicines recommended for approval:
Alyftrek (deutivacaftor, tezacaftor, vanzacaftor): has received a positive opinion for the treatment of cystic fibrosis (CF) in people aged 6 years and older who have at least one non-class I mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. An orphan designation was granted for the treatment of this disease.
Cystic fibrosis is a genetic disorder that leads to a thicker than normal mucus. The thick mucus can’t be cleared normally from the lungs, which increases the probability of bacterial infections. Other affected organs are the pancreas, liver, kidney and intestine. For more information please consult the product for Alyftrek on the EMA website.
Attrogy (diflunisal): has received a positive opinion for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adult patients with stage 1 or stage 2 polyneuropathy. An orphan designation was granted for the treatment of this disease.
hATTR amyloidosis is a rare and severe disease, characterised by multisystem amyloid depositions. The most common clinical presentations include polyneuropathy and cardiomyopathy. ATTRv is caused by mutations in the TTR gene (encoding the transport protein transthyretin), which cause protein misfolding, aggregation and subsequent deposition. For more information please consult the product for Attrogy on the EMA website.
Duvyzat (givinostat): has received a positive opinion for the conditional marketing authorisation for the treatment of Duchenne muscular dystrophy (DMD) in ambulant patients, aged 6 years and older, and with concomitant corticosteroid treatment. An orphan designation was granted for the treatment of this disease.
DMD is a rare, severe muscle wasting disease, which affects boys from birth and is invariably fatal due to the lack of dystrophin, a structural and regulatory protein at the membranes of muscle fibres. It is caused by errors in the gene encoding dystrophin, an essential cohesive protein which connects muscle fibers with the surrounding extracellular matrix. The dystrophin gene is located on the X-chromosome, therefore DMD occurs almost exclusively in males (X-linked recessive disorder). A separate news was released by the EMA. For more information please consult the product for Duvyzat on the EMA website.
Oczyesa (octreotide): has received a positive opinion for the maintenance treatment in adult patients with acromegaly who have responded to and tolerated treatment with somatostatin analogues. An orphan designation was granted for the treatment of this disease.
Acromegaly is a rare life-threatening disease characterised by increased growth hormone (GH) secretion after the growth plates have closed, which results in excessive growth of body tissues. This over-production of GH is most often caused by an adenoma of the anterior pituitary gland. For more information please consult the product for Oczyesa on the EMA website.
Sephience (sepiapterin): has received a positive opinion for the treatment of hyperphenylalaninaemia (HPA) in adult and paediatric patients with phenylketonuria (PKU). An orphan designation was granted for the treatment of this disease.
PKU is a genetic disorder that leads to a decreased transformation of phenylalanine to tyrosine and increased phenylanlanine levels in the blood. Non treated PKU leads to intellectual disability and a decreased psychomotor development. For more information please consult the product for Sephience on the EMA website.
Tepezza (teprotumumab): has received a positive opinion for the treatment of moderate to severe thyroid eye disease (TED).
TED is an autoimmune disease which causing the inflammation of the tissue around and behind the eyes. Teprotumumab is the first pharmacological treatment despite corticosteroids for TED and a separate news was published by the EMA. For more information please consult the product for Tepezza on the EMA website.
Ziihera (zanidatamab): has received a positive opinion for the conditional marketing authorisation for the treatment of adults with unresectable locally advanced or metastatic HER2-positive (IHC3+) biliary tract cancer (BTC) previously treated with at least one prior line of systemic therapy. An orphan designation was granted for the treatment of this disease. For more information please consult the product for Ziihera on the EMA website.
Recommendations on extensions of therapeutic indication:
Adcetris (brentuximab vedotin): extension of indication for Adcetris to include the treatment of adult patients with previously untreated CD30+ Stage IIB with risk factors, Stage III or Stage IV Hodgkin lymphoma in combination with etoposide, cyclophosphamide, doxorubicin, dacarbazine, dexamethasone (BrECADD). Adcetris is already approved for the treatment of Hodkin lymphoma in different settings. For more information please consult the product for Adcetris on the EMA website.
Adempas (riociguat): extension of indication for Adempas to include the treatment of pulmonary arterial hypertension in paediatric patients aged 6 to less than 18 years with WHO Functional Class (FC) II to III in combination with endothelin receptor antagonists. Previously paediatric patients had to weigh over 50 kg. For more information please consult the product for Adempas on the EMA website.
Amvuttra (vutrisiran): extension of indication for Amvuttra to include the treatment of wild-type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM). Amvutta is already approved for the treatment of hereditary transthyretin amyloidosis with polyneuropathy. For more information please consult the product for Amvuttra on the EMA website.
Calquence (acalabrutinib): extension of indication for Calquence to include in combination with venetoclax with or without obinutuzumab the treatment of adult patients with previously untreated chronic lymphocytic leukaemia (CLL). Calquence is already approved for the treatment of CLL in different settings. For more information please consult the product for Calquence on the EMA website.
Cystadrops (mercaptamine): extension of indication for Cystadrops to include the treatment of corneal cystine crystal deposits in adults and children from 6 months of age with cystinosis. Previously the age limit was 2 years. For more information please consult the product for Cystadrops on the EMA website.
Jivi (damoctocog alfa pegol): extension of indication for Jivi to include the treatment and prophylaxis of bleeding in previously treated patients ≥7 years of age with haemophilia A (congenital factor VIII deficiency). Previously the age limit was 12 years. For more information please consult the product for Jivi on the EMA website.
Vyvgart (efgartigimod alfa): extension of indication for Vyvgart to include the treatment of adult patients with progressive or relapsing active chronic inflammatory demyelinating polyneuropathy (CIDP) after prior treatment with corticosteroids or immunoglobulins. For more information please consult the product for Vyvgart on the EMA website.
Xofluza (baloxavir marboxil): extension of indication for Xofluza to include the treatment of uncomplicated influenza in patients aged 3 weeks and above and the post‑exposure prophylaxis of influenza in individuals aged 3 weeks and above. Previously the age limit was 1 year of age. For more information please consult the product for Xofluza on the EMA website.
Zoonotic Influenza Vaccine Seqirus (zoonotic influenza vaccine (H5N8) (surface antigen, inactivated, adjuvanted)): extension of indication for the active immunisation against H5 subtype influenza A viruses in individuals 6 months of age and above. Previously the age limit was 18 years of age. For more information please consult the product for Zoonotic 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:
Capvaxive: is indicated for active immunisation for the prevention of invasive disease andpneumonia caused by Streptococcus pneumoniae in individuals 18 years of age and older.
EPAR Capvaxive.
Lynozyfic: is indicated as monotherapy for the treatment of adult patients with relapsed and refractory multiple myeloma who have received at least 3 prior therapies, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody, and have demonstrated disease progression on the last therapy.
EPAR Lynozyfic.
Tivdak: as monotherapy is indicated for the treatment of adult patients with recurrent or metastatic cervical cancer with disease progression on or after systemic therapy.
EPAR Tivdak.
