PTC CEO Stuart Peltz tells Scrip that cost watchdogs are likely to look favorably on the firm’s transformational gene therapy for a distressing rare brain disorder in children who could not even lift their heads before treatment.

Regulators want to help smooth the process of bringing a gene therapy to market, hoping to iron out ‘surprises’ and even address reimbursement issues for the sake of patients.

Embattled gene therapy companies will take comfort from desire of FDA and EMA help smooth the process of bringing the novel treatments to market.

Vertex is likely to have the cystic fibrosis market to itself for years to come, but investors are banking on it repeating that success in several new first-in-class therapy areas.