Pink Sheet is part 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


Genzyme Myozyme BLA

This article was originally published in Pharmaceutical Approvals Monthly

Executive Summary

Genzyme expects a priority review for its Myozyme (alglucosidase alfa) BLA for treatment of Pompe disease. The company submitted the application July 28, setting a priority review action date at Jan. 28. Pompe disease, which the firm estimates affects fewer than 10,000 people worldwide, is part of the same family of lysosomal storage disorders as mucopolysaccharidosis, Fabry and Gaucher disease...

You may also be interested in...

Genzyme Myozyme revised PDUFA date

Genzyme's Pompe disease treatment Myozyme (alglucosidase alfa) has a revised user fee date of April 28 following FDA's 90-day extension. The extension is to provide time to review additional agency-requested information, Genzyme says. The company submitted the BLA July 28 (1Pharmaceutical Approvals Monthly August 2005, In Brief); the enzyme replacement therapy is receiving a priority review...

Pink Sheet Podcast: Woodcock To Be Acting US FDA Chief, Key Staff Depart, Political Donations Shift

Pink Sheet reporters and editor discuss President-Elect Joe Biden’s decision to name the CDER director as acting FDA commissioner and other staffing changes, as well as changes to industry trade groups’ political donations.

Liquid Biopsy Company Delfi Diagnostics Raised $100M For New Class of Early Cancer Detection Test

Delfi Diagnostics will use the new funds to expand its team of cancer researchers and machine-learning experts to validate its novel approach for early cancer detection through multiple prospective clinical trials. 





Ask The Analyst

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

Your question has been successfully sent to the email address below and we will get back as soon as possible. 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

Ask your question to our analysts