The European Medicines Agency (EMA) human medicines committee (CHMP) made a number of key recommendations at its December 2023 meeting.
The committee recommended granting a conditional marketing authorisation for Casgevy (exagamglogene autotemcel), an advanced therapy medicinal product (ATMP) for the treatment of transfusion dependent beta-thalassemia and severe sickle cell disease.
This is the first gene therapy to treat these patients and the first medicine that uses CRISPR/Cas9 technology. It was also recently approved by the UK’s Medicines and Healthcare Regulatory Agency (MHRA) and the US Food and Drug Administration (FDA).
“This positive opinion is yet another important regulatory milestone underscoring the potentially transformative benefit of Casgevy for eligible patients with sickle cell and transfusion-dependent beta thalassemia,” said Nia Tatsis, Executive Vice President and Chief Regulatory and Quality Officer at Vertex, who co-developed the therapy with CRISPR Therapeutics.
Other recommendations from the CHMP
Skyclarys (omaveloxolone) received a positive opinion for the treatment of Friedreich’s ataxia, an inherited disease causing a range of symptoms, including difficulty walking, inability to co-ordinate movements, muscle weakness, speech problems, damage to the heart muscle and diabetes.
The drug was developed by Reata Pharmaceuticals, which was acquired by Biogen in July 2023.
The committee also adopted a positive opinion for Velsipity (etrasimod), for the treatment of patients with moderate to severe ulcerative colitis, an inflammation of the large intestine causing ulceration and bleeding.
Following a re-examination, the CHMP confirmed its initial recommendation to not renew the conditional marketing authorisation for Blenrep (belantamab mafodotin) for multiple myeloma.
The committee also recommended extensions of indication for four medicines that are already authorised in the EU: HyQvia, Metalyse, VeraSeal and Zinplava.
Medicines for use outside the EU
The CHMP adopted positive opinions for two medicines submitted under a regulatory procedure known as EU-Medicines for all (EU-M4All) that enables EMA to support global regulatory capacity building and contribute to the protection and promotion of public health beyond the EU.
The first was Arpraziquantel (arpraziquantel), a new treatment option for children with schistosomiasis, a neglected tropical disease caused by blood flukes (trematode worms).
The second drug, Fexinidazole Winthrop (fexinidazole) treats human African trypanosomiasis (sleeping sickness). It was recommended in 2018 for sleeping sickness caused by the parasite trypanosoma brucei gambiense, now the CHMP recommends extending the indication to include disease caused by trypanosoma rhodesiense.