Medtech Insight 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


In Brief: Orphan Medical's Cystadane

Executive Summary

Orphan Medical's Cystadane: FDA approves betaine anhydrous oral solution Oct. 25 for "the treatment of homocystinuria to decrease elevated homocysteine blood levels," agency approval letter states. Included within the category of homocystinuria are deficiencies or defects in cystathionine beta synthase (CBS); 5,10-methylenetetrahydrofolate reductase (MTHFR); and cobalamin cofactor metabolism (cbl). Cystadane is classified as a "1PV" drug, denoting a new molecular entity with an orphan designation receiving a priority review...



Ask The Analyst

Please Note: You can also Click below Link for Ask the Analyst
Ask The Analyst

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