Scrip 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

UsernamePublicRestriction
UsernamePublicRestriction

China Approves Merck Serono Orphan Drug Kuvan; Paves Way For More Orphan Drug R&D

This article was originally published in PharmAsia News

Executive Summary

China's State FDA has approved Merck Serono's Kuvan (sapropterin) for orphan condition hyperphenylalaninemia in phenylketonuria or BH4 deficiency, a rare congenital metabolic disorder that increases blood phenylalanine levels. The approval underlines opportunities in the Chinese orphan drug market, says a leading Chinese expert

You may also be interested in...



Can Patients Move The Rare Disease Treatment Needle In China?

Innovative orphan drug makers have so far largely shunned China but things could be changing. Chinese patient communities tell the Pink Sheet how they are increasingly taking it upon themselves to lobby regulators and persuade drug developers to include China in their clinical development consideration.

China Orphan Drug Reimbursement: Swift Action Needed – Former Genzyme GM

Only 1% of 5,781 rare diseases in China have effective treatments. Facing reimbursement and infrastructure challenges, the market needs rapid action from government and industry, says Shirley Zhao, former Genzyme China general manager.

China Orphan Drug Reimbursement: Swift Action Needed – Former Genzyme GM

Only 1% of 5,781 rare diseases in China have effective treatments. Facing reimbursement and infrastructure challenges, the market needs rapid action from government and industry, says Shirley Zhao, former Genzyme China general manager.

Related Content

UsernamePublicRestriction

Register

SC076213

Ask The Analyst

Please Note: Click here for more information on the Ask the Analyst service.

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

Cancel