AstraZeneca rare disease therapy improves bone health in Phase III HPP study
Pharma major AstraZeneca (LSE: AZN) has reported positive Phase III data for efzimfotase alfa, showing that the investigational enzyme replacement therapy improved bone health in treatment-naive children with hypophosphatasia (HPP), a rare inherited metabolic disease.
Results from the MULBERRY trial, presented at the 12th International Conference on Children’s Bone Health in Montreal, showed a statistically significant and clinically meaningful improvement in radiographic global impression of change (RGI-C) score at week 25 compared with placebo.
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 accountTry before you buy
Free
7 day trial access
- All the news that moves the needle in pharma and biotech
- Exclusive features, podcasts, interviews, data analyses and commentary from our global network of life sciences reporters.
- Receive The Pharma Letter daily news bulletin, free forever.
Become a subscriber
£820
Or £77 per month
- 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
More on this story...
Today's issue
Company Spotlight
More Features in Biotechnology
| Headless Content Management with Blaze