March 29, 2019 10:46 AM EDT

Rare disease gene therapy gains Prime status; FDA approves first drug for inflammatory arthritis nr-axSpA

John Carroll

Editor & Founder

→ Pittsburgh-based Krystal Biotech $KRYS has won the EMA’s Prime status for its gene therapy to treat dystrophic epidermolysis bullosa. The company says regulators …

Sign up to read this article for free.

Get free access to a limited number of articles, plus choose newsletters to get straight to your inbox.

BECOME A PREMIUM SUBSCRIBER

Rare disease gene therapy gains Prime status; FDA approves first drug for inflammatory arthritis nr-axSpA

John Carroll

Editor & Founder

Sign up to read this article for free.

Vertex gets 'vanza triple' approval and Trikafta expansion — with boxed warnings

Novo Nordisk’s once-daily hemophilia drug Alhemo approved in US

The Endpoints 2024 winners and losers list: Who was up and who was down in biopharma

Califf, on his way out at FDA, predicts agency tug-of-war between Ramaswamy and RFK Jr.

GSK's Zejula-Jemperli combo in ovarian cancer hits primary endpoint, misses on overall survival

Exclusive: Florida cancer biotech raises $100M Series A

Rare dis­ease gene ther­a­py gains Prime sta­tus; FDA ap­proves first drug for in­flam­ma­to­ry arthri­tis nr-axS­pA

John Carroll

Editor & Founder

Sign up to read this article for free.

more like this

Ver­tex gets 'van­za triple' ap­proval and Trikaf­ta ex­pan­sion — with boxed warn­ings

No­vo Nordisk’s once-dai­ly he­mo­phil­ia drug Al­he­mo ap­proved in US

The End­points 2024 win­ners and losers list: Who was up and who was down in bio­phar­ma

Califf, on his way out at FDA, pre­dicts agency tug-of-war be­tween Ra­maswamy and RFK Jr.

GSK's Ze­ju­la-Jem­per­li com­bo in ovar­i­an can­cer hits pri­ma­ry end­point, miss­es on over­all sur­vival

Ex­clu­sive: Flori­da can­cer biotech rais­es $100M Se­ries A

Rare disease gene therapy gains Prime status; FDA approves first drug for inflammatory arthritis nr-axSpA

Vertex gets 'vanza triple' approval and Trikafta expansion — with boxed warnings

Novo Nordisk’s once-daily hemophilia drug Alhemo approved in US

The Endpoints 2024 winners and losers list: Who was up and who was down in biopharma

Califf, on his way out at FDA, predicts agency tug-of-war between Ramaswamy and RFK Jr.

GSK's Zejula-Jemperli combo in ovarian cancer hits primary endpoint, misses on overall survival

Exclusive: Florida cancer biotech raises $100M Series A