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CHMP backs European approval for six new medicines

Recommends drugs by PTC Therapeutics, Roche, Biogen Idec, Octapharma, Alcon and Veloxis Pharma

European Medicines Agency (EMA)The EMA's Committee for Medicinal Products for Human Use (CHMP) recommended half a dozen new drugs for indications including leukaemia, muscular dystrophy and multiple sclerosis (MS) last week.

Heading the list, the CHMP recommended condition approval for PTC Therapeutics' Translarna (ataluren), a first-in-class therapy for Duchenne muscular dystrophy (DMD) patients aged under five that had previously been turned down by the committee.

Translarna was tested in a phase II study involving 174 patients with DMD which showed "some evidence of efficacy" of the drug in slowing down the loss of walking ability in DMD patients, and PTC will have to file positive data from an ongoing confirmatory trial to secure full approval. DMD affects around 18,600 people in the EU, but Translarna is indicated only for a subset with nonsense mutations in the dystrophin gene.

The CHMP also issued a positive opinion for Roche's Gazyvaro (obinutuzumab) as a combination therapy alongside chlorambucil for adult patients with previously-untreated chronic lymphocytic leukaemia (CLL) unsuitable for treatment with fludarabine. CLL makes up around a third of all adult leukaemias.

Gazyvaro was approved in the US last year on the back of clinical data showing the combination doubled progression-free survival (PFS) versus chlorambucil alone, with analysts predicting that it could become a $1.5bn-plus product at peak if it is found to be superior to Roche's $7bn-a-year Rituxan (rituximab).

There was also good news for Biogen Idec with a positive verdict on its long-acting MS drug Plegridy (peginterferon beta-1a), keeping its follow-up to $2.9bn-a-year Avonex (interferon beta-1a) on track for approval in mid-2014.

Plegridy requires less frequent injections than Avonex and is administered subcutaneously rather than intramuscularly, so should be less painful for patients.

Switzerland's Octapharma secured a recommendation for its haemophilia therapy Nuwiq (simoctocog alfa) as a treatment for both paediatric and adult patients with congenital Factor VIII deficiency (haemophilia A). Nuwiq has also been submitted to authorities in Canada and Australia, with further worldwide submissions planned, according to the company.

Octapharma describes Nuwiq as a new generation Factor VIII replacement protein that is derived from a human cell line and so could be less prone to the stimulating neutralising antibodies that can limit the efficacy of current haemophilia A therapies.

Finally, Novartis' eyecare subsidiary Alcon got a green light for its combination glaucoma therapy Simbrinza (brinzolamide/brimonidine - first approved in the US last year - while Veloxis Pharma got the nod for Envarsus (tacrolimus), its once-daily drug for the prophylaxis and treatment of organ rejection in adult transplant patients.

Article by
Phil Taylor

27th May 2014

From: Sales, Regulatory

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