Scrip is part of the Business Intelligence Division of Informa PLC

This site is operated by a business or businesses owned by Informa PLC and all copyright resides with them. Informa PLC’s registered office is 5 Howick Place, London SW1P 1WG. Registered in England and Wales. Number 8860726.

This copy is for your personal, non-commercial use. For high-quality copies or electronic reprints for distribution to colleagues or customers, please call +44 (0) 20 3377 3183

Printed By

Alexion Snags 2nd FDA Voucher On Kanuma Win

This article was originally published in Scrip

Executive Summary

The FDA's approval on Dec. 8 of Alexion Pharmaceuticals Inc.'s enzyme replacement therapy (ERT) Kanuma (sebelipase alfa) was significant for several reasons – mostly, because it provides patients with the first treatment in the US for a deadly, progressive ultra-rare genetic disease, known as lysosomal acid lipase deficiency (LAL-D), which causes multi-organ damage, resulting in premature death.






Ask The Analyst

Ask the Analyst is free for subscribers.  Submit your question and one of our analysts will be in touch.

All set! This Question has been sent to my@email.address.

All fields are required.

Please make sure all fields are completed.

Please make sure you have filled out all fields

Please make sure you have filled out all fields

Please enter a valid e-mail address

Please enter a valid Phone Number

Add a personalized Question to your Analyst