The latest set of opinions from advisors to the European Medicines Agency include recommendations to approve six new medicines, including the first ever drug for Duchenne muscular dystrophy, and treatments for leukaemia and multiple sclerosis.

First up, the agency's Committee for Medicinal Products for Human Use has backed granting a conditional marketing authorisation on PTC Therapeutics' Translarna (ataluren) for DMD, a rare genetic disease affecting 18,600 people in the European Union, that gradually causes weakness and loss of muscle function. The recommendation, which is for use in the subgroup of boys (about 10%-15% of cases) aged five and over whose disease is due to certain defects called nonsense mutations in the dystrophin gene, follows the re-examination of a negative opinion issued in January.

Under the terms of the authorisation, PTC has to provide comprehensive data from an ongoing Phase III study to the EMA which has provided free scientific advice. Unsurprisingly, the announcement has gone down well with the DMD community and chief executive of the UK's Muscular Dystrophy Campaign, Robert Meadowcroft, said the EMA decision is "fantastic news".

He was especially positive given the "recent setbacks involving other potential treatments for DMD [which] have been disappointing for everyone racing against the clock to develop treatments that could protect muscles from further damage". This "step forward for Translarna offers much needed hope", he added.

The CHMP has also issued a positive opinion on Roche's Gazyvaro (obinutuzumab) in combination with chlorambucil for the treatment of chronic lymphocytic leukaemia. The drug, already marketed in the USA, is for patients who have comorbidities making them unsuitable for full-dose fludarabine chemotherapy and Sandra Horning, Roche’s chief medical officer, said the firm is one step closer to making Gazyvaro "a new foundation of treatment for their disease".

The EMA's advisors have also recommended approval for Biogen Idec's Plegridy (peginterferon beta-1a) for relapsing remitting multiple sclerosis in adults and Octapharma's Nuwiq (simoctogog alfa) for bleeding in patients with haemophilia A. Other highlights included recommendations for Novartis' Simbrinza (brinzolamide/brimonidine) for open-angle glaucoma or ocular hypertension and Veloxis/Chiesi's Envarsus (tacrolimus) for kidney and liver transplants.

The CHMP also recommended extensions of indications for GlaxoSmithKline's Arzerra (ofatumumab), Eisai's Halaven (eribulin) and Pfizer's Vfend (voriconazole).