EU Approval Set For Three New Orphans And Lilly’s Emgality; Exondys Appeal Fails

 
• By Maureen Kenny
  
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The CHMP has upheld its previous negative opinion on Sarepta’s DMD treatment Exondys, but has given a raft of positive opinions on other novel products, including Lilly’s new migraine prophylactic Emgality, Spark Therapeutics’ gene therapy Luxturna for retinal dystrophy, and Vabomere, The Medicines Co/Rempex Pharmaceuticals’ new antibiotic for severe infections.

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Not all products get the OK from the EMA's CHMP • Source: Shutterstock

Sarepta Therapeutics’ chances of marketing its Duchenne muscular dystrophy treatment, Exondys, in the EU any time soon are zero. EU approval is now essentially a given, though, for Eli Lilly’s new prophylactic migraine product, Emgality, and for a swathe of other new products, including three orphan medicines, one of which is Spark Therapeutics’ Luxturna gene therapy for the treatment of adults and children with inherited retinal dystrophy caused by RPE65 gene mutations, a rare genetic disorder that causes vision loss and usually leads to blindness.

Vabomere, The Medicines Co./Rempex Pharmaceuticals Inc.'s new antibiotic for the treatment of certain severe infections in adults, is...

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