Scrip is part of Pharma Intelligence UK Limited

This site is operated by Pharma Intelligence UK Limited, a company registered in England and Wales with company number 13787459 whose registered office is 5 Howick Place, London SW1P 1WG. The Pharma Intelligence group is owned by Caerus Topco S.à r.l. and all copyright resides with the group.

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

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

Latest Headlines
See All
UsernamePublicRestriction

Register

SC076213

Ask The Analyst

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

Thank you for submitting your question. We will respond to you within 2 business days. 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