Alexion Gets Second Priority Review Voucher With Approval Of Rare Enzyme Disorder Drug
This article was originally published in The Pink Sheet Daily
Executive Summary
Kanuma, the first approved treatment for lysosomal acid lipase deficiency, will launch in January at an average annual treatment cost of $310,000.
You may also be interested in...
Priority Review Voucher Not For Sale, Alexion Says After Strensiq Approval
Alexion plans to use voucher for one of its own products and might be targeting its next-generation version of blockbuster Soliris.
Breakthrough's Achilles Heel: Alexion’s Newly Acquired Kanuma Delayed By Manufacturing
FDA extends the PDUFA date for enzyme replacement therapy by three months so agency can review chemistry, manufacturing and controls information; another drug, Strensiq, is also pending at FDA without any action.
Companies Reveal Hurdles In Providing Drugs Via Expanded Access Programs
GSK, Stealth BioTherapeutics and Blueprint Medicines discuss the difficulties getting participation of physicians, the excessive cost of expanded access, and whether physicians should report research data.