The company anticipates potential sales in excess of $1bn for the late-onset Pompe disease drug by the end of the decade.

Pink Sheet analysis finds big jump in the share of novel approvals that received PDUFA extensions, largely driven by clinical and safety data submissions.

Chiesi-owned Amryt and Krystal Biotech have approved treatments for epidermolysis bullosa but they only treat one subtype - dystrophic - and a single wound. Paradigm CEO Robert Ryan tells Scrip that SD-101, a topical cream he helped develop over 20 years ago, has the potential to be offer a far more extensive and efficacious solution for many more sufferers.   

Amicus is launching its Pompe disease treatment Pombiliti/Opfolda in the UK after announcing on the same day that it had secured reimbursement and marketing approval for the combination treatment. Meanwhile, Immunocore says it disagrees with NICE's rejection of its advanced uveal melanoma drug, Kimmtrak.