IONS/AKCA receive thumbs down on Waylivra for FCS

“We are extremely disappointed with the FDA’s decision”, writes the Ionis Pharma press release in describing the Complete Response Letter (CRL) they received regarding their latest medicine, WAYLIVRA. They continue, “we feel strongly that WAYLIVRA demonstrates a favorable benefit/risk profile in people with FCS as was reflected in the positive outcome from our Advisory Committee […]

I was wrong about Zogenix! FDA updates guidelines for gene therapy.

Note: I meant to say that sub-1% factor activity is SEVERE hemophilia. Normal is from 50% to 150%. Today I talk again about Zogenix because I was wrong about Study 2 being priced in! Congrats to the ZGNX holders. I also talk about the new FDA guidelines on gene therapy for hemophilia and its implications […]