CHMP Meeting Highlights July 2021
In the month of July 2021, 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.
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. EPAR for Deltyba.
Ultomiris (ravulizumab) extension of indication to include the treatment of paediatric patients with a body weight of 10 kg or above. EPAR for Ultomiris.
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. EPAR for Volibris.
Vosevi (sofosbuvir / velpatasvir / voxilaprevi) extension of indication to include the treatment of patients aged 12 years and older and weighing at least 30 kg. EPAR for Vosevi.
Newly published European Public Assessment Reports (EPAR)
The European Public Assessment Report (EPAR) is the main document where the European Medicines Agency (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 (ioflupane - 123I) to help in the diagnosis of movement disorders and dementia. EPAR Celsunax.
Recently started procedures
Every month, new marketing authorization applications are submitted to EMA under the centralized authorization 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 authorization applications:
- Amifampridine - Treatment of Lambert-Eaton Myasthenic Syndrome.
- Capmatinib - Treatment of non-small cell lung cancer.
- Ciltacabtagene autoleucel - Treatment of multiple myeloma.
- 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.
- Ranibizumab - Treatment of neovascular age-related macular degeneration in adults.
Referral procedures
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.