CHMP Meeting Highlights July 2021
Information about the Austrian representation on the CHMP and monthly updates can be found at this Link.
This month, medicinal products for the following indications have received a positive opinion:
- Glycogen storage disease type II or acid α-glucosidase deficiency (Pompe disease)
New medicines recommended for approval:
Nexviadyme (avalglucosidase alfa): is indicated for long-term enzyme replacement therapy for the treatment of patients with Pompe disease (acid α-glucosidase deficiency). Pompe disease is an autosomal recessive metabolic disease caused by a reduced or absent activity of the lysosomal alpha-glucosidase. As a consequence, glycogen accumulates in the lysosome, causing damage in the muscle and nerve cells throughout the body. Nexviadyme is a recombinant human alpha-glucosidase intended for enzyme replacement and it is conjugated with synthetic glycans to enhance receptor targeting. For more information please consult the product page for Nexviadyme on the EMA website.
Recommendations on extensions of therapeutic indication:
Deltyba (delamanid): extension of indication to include the treatment of infant patients with a body weight of at least 10 kg. For more information please consult the product page for Deltyba on the EMA website.
Ultomiris (ravulizumab): extension of indication to include the treatment of paediatric patients with a body weight of 10 kg or above. For more information please consult the product page for Ultomiris on the EMA website.
Volibris (ambrisentan): extension of indication to include the treatment of pulmonary arterial hypertension (PAH) in adolescents and children (aged 8 to less than 18 years) of WHO Functional Class (FC) II to III including use in combination treatment. For more information please consult the product page for Volibris on the EMA website.
Vosevi (sofosbuvir / velpatasvir / voxilaprevir): extension of indication to include the treatment of patients aged 12 years and older and weighing at least 30 kg. For more information please consult the product page for Vosevi on the EMA website.
Newly published EPARs:
The EPAR (European public assessment report) is the main document where the EMA publishes the detailed information from the medicines assessed by the CHMP. Below is a list of the EPARs for recently approved products that have been made available at the EMA homepage between June 25th and July 22nd 2021:
Celsunax (Ioflupan - 123I): to help in the diagnosis of movement disorders and dementia. EPAR Celsunax.
Recently started procedures:
Every month, new medicines are submitted to the EMA for approval to provide patients with new treatment options for various diseases. The CHMP conducts a scientific assessment of the applications and makes a recommendation. The CHMP has started the evaluation of the following submitted applications:
- Amifampridine - Treatment of Lambert-Eaton Myasthenic Syndrome
- Capmatinib - Treatment of non-small cell lung cancer (NSCLC)
- Ciltacabtagene autoleucel - Treatment of multiple myeloma
- Insulin aspart - Treatment of diabetes mellitus
- Melphalan flufenamide - Treatment of multiple myeloma
- Omburtamab I-131 - Treatment of neuroblastoma
- Palovarotene - Treatment of fibrodysplasia ossificans progressiva
- Pirfenidone - Treatment of mild to moderate idiopathic pulmonary fibrosis (IPF)
A referral is a procedure in which EMA is asked to evaluate scientifically a medicine or class of medicines on behalf of the European Union. These type of procedures are intended to resolve certain safety or benefit-risk balance concerns. Learn more about referral procedures.
Conclusion of the referral on Zynteglo: CHMP has endorsed a review that found no link between the viral vector and acute myeloid leukaemia (AML):
- Although two patients who received the same viral vector in another medicine had AML, there are more plausible explanations than insertional oncogenesis, including the conditioning treatment the patients received and the higher risk of haematological cancer in people with sickle cell disease.
- Healthcare professionals should explicitly inform patients receiving Zynteglo of the increased risk of blood cancers from medicines used in conditioning treatments.
- Healthcare professionals should continue monitoring patients for cancer. EMA has updated its recommendation for cancer screening, changing the requirement from once a year to at least once a year.
Learn more about the conclusion of the referral on Zynteglo.
|CHMP_highlight_2021-07_nexviadyme||CHMP summary of positive opinion for Nexviadyme||https://www.ema.europa.eu/en/medicines/human/summaries-opinion/nexviadyme|
|CHMP_highlight_2021-07_deltyba||CHMP post-authorisation summary of positive opinion for Deltyba||https://www.ema.europa.eu/en/medicines/human/summaries-opinion/deltyba-0|
|CHMP_highlight_2021-07_ultomiris||CHMP post-authorisation summary of positive opinion for Ultomiris||https://www.ema.europa.eu/en/medicines/human/summaries-opinion/ultomiris-1|
|CHMP_highlight_2021-07_volibris||CHMP post-authorisation summary of positive opinion for Volibris||https://www.ema.europa.eu/en/medicines/human/summaries-opinion/volibris|
|CHMP_highlight_2021-07_vosevi||CHMP post-authorisation summary of positive opinion for Vosevi||https://www.ema.europa.eu/en/medicines/human/summaries-opinion/vosevi|
|CHMP_highlight_2021-07_zynteglo||Conclusion of the referral on Zynteglo||https://www.ema.europa.eu/en/news/chmp-endorses-review-finding-no-link-between-viral-vector-zynteglo-blood-cancer|