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Vertex’s triple CF therapy scores recommendation from CHMP

Other highlights from EMA’s CHMP’s June meeting include Idefirix and remdesivir


Vertex has won a positive recommendation from the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) for its triple combination cystic fibrosis therapy, alongside a host of other recommendations announced in the Committee’s June meeting.

The CHMP has recommended granting a conditional marketing authorisation for the triple therapy, comprised of elexacaftor/tezacaftor/ivacaftor, under the brand name Kaftrio. It is the first triple combination therapy for the treatment of CF in patients aged 12 years and older with one F508del mutation and one minimal function mutation and in patients with two F508del mutations.

The triple therapy was approved by the US Food and Drug Administration (FDA) in October last year, and scored that approval only a few months after filing the combination treatment with the agency. In the US, the drug was approved under the brand name Trikafta, and priced at $311,000 for a year of treatment.

The treatment provides a new treatment option for many CF patients, including for those with certain mutations where no treatment is currently available in the EU.

Other highlights from the June CHMP meeting include a positive recommendation for Hansa Biopharma’s Idefirix (imlifidase), the first treatment for adult patients waiting for a kidney transplant who are highly sensitised against tissue from the donor and who have a positive crossmatch test against an available kidney from a deceased donor.

The drug was assessed with support from the EMA’s PRIME scheme platform, which includes early and enhanced dialogue with developers of promising new medicines that dress unmet medical needs.

As previously reported, the CHMP also recommended approval of Gilead Science’s antiviral drug remdesivir, which has now been dubbed Veklury. The drug is the first COVID-19 treatment to receive a conditional marketing approval from the CHMP, and full EMA  approval is expected soon, with the CHMP saying it could come within a week.

The only drug to receive a negative opinion in this month’s meeting was Daiichi Sankyo’s Turalio (pexidartinib), which was seeking approval for the treatment of tenosynovial giant cell tumour (TGCT), a rare non-malignant tumour that affects small and large joints.

Although the agency agreed the drug shrank tumours in patients with TGCT, it presented concerns the there was only a small improvement in symptoms including pain and the ability to use the affected joints. There was also a serious concern surrounding the unpredictable and potentially life-threading effects of Turalio on the liver.

Turalio was previously approved by the FDA for the treatment of TGCT associated with severe morbidity or functional limitations, and which is not likely to improve with surgery.

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29th June 2020



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