The company, in its new profitable era, is bringing in new leadership, with Amgen VP of medical affairs for rare diseases Greg Friberg and recently retired Roche Partnering head James Sabry.

Scotland’s health technology assessment body has granted routine reimbursement to Novartis’ vision loss drug Luxturna after an interim funding period via its ultra-orphan pathway, while Chiesi’s Filsuvez is set to begin its journey on the same pathway.

In a major boost for the UK biotech sector, an impressive sum has been raised by Beacon Therapeutics to support its R&D activities in ophthalmic gene therapies.

Companies should talk to FDA staff in between formal meetings if they need clarity on agency advice that is several years old or development programs hit a snag, Office of Therapeutic Products Director Nicole Verdun says.