Sobi's Orfadin oral suspension for HT-1 approved in USA

26 April 2016
2019_biotech_test_vial_discovery_big

Swedish Orphan Biovitrum (STO: SOBI) has gained approval from the US Food and Drug Administration for Orfadin (nitisinone) oral suspension to treat hereditary tyrosinaemia type-1 (HT-1), in combination with dietary restriction of tyrosine and phenylanine.

HT-1 is a rare genetic disease that affects infants and children. It can result in liver and kidney complications, and is potentially fatal if untreated. Some 20 years ago, before pharmacological treatment was available, fewer than one third of infants diagnosed with HT-1 before two months of age, lived past their second birthday.

Today, treatment with Orfadin as an adjunct to dietary restriction as well as early diagnosis have improved outcomes for HT-1 patients.

This article is accessible to registered users, to continue reading please register for free.  A free trial will give you access to exclusive features, interviews, round-ups and commentary from the sharpest minds in the pharmaceutical and biotechnology space for a week. If you are already a registered user please login. If your trial has come to an end, you can subscribe here.

Login to your account

Become a subscriber

 

£820

Or £77 per month

Subscribe Now
  • Unfettered access to industry-leading news, commentary and analysis in pharma and biotech.
  • Updates from clinical trials, conferences, M&A, licensing, financing, regulation, patents & legal, executive appointments, commercial strategy and financial results.
  • Daily roundup of key events in pharma and biotech.
  • Monthly in-depth briefings on Boardroom appointments and M&A news.
  • Choose from a cost-effective annual package or a flexible monthly subscription
The Pharma Letter is an extremely useful and valuable Life Sciences service that brings together a daily update on performance people and products. It’s part of the key information for keeping me informed

Chairman, Sanofi Aventis UK

Companies featured in this story

More ones to watch >


Today's issue

Company Spotlight





More Features in Biotechnology