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

Alexion gets EU nod for two rare genetic disease drugs

This article was originally published in Scrip

Executive Summary

The EU's CHMP has given Alexion the marketing go-ahead for two new enzyme replacement therapies for rare genetic diseases: Kanuma (sebelipase alfa) for the treatment of lysosomal acid lipase deficiency (LAL-d); and Strensiq (asfotase alfa), the first therapy for the bone disease hypophosphatasia (HPP) that started in childhood.

You may also be interested in...



DESTINY Calls For Daiichi Sankyo/AZ’s Enhertu In Other Indications

Daiichi Sankyo and AstraZeneca’s ambitions to expand the use of their HER-2 targeted antibody-drug conjugate product beyond breast cancer are looking realizable on the back of new results presented at ASCO, but toxicities remain an issue. 

ASCO 2020: AZ’s Tagrisso Sails Into Early-Stage NSCLC On ‘Momentous’ ADAURA Trial Data

A highly impressive improvement in disease-free survival in ADAURA is set to pitch AstraZeneca’s best-selling product into a new untapped market, early-stage EGFRm NSCLC, reaping a possible $3bn in extra sales.

China Makes Its Mark On R&D Pipeline And Novel Launches

The largest-ever rise in the number of active R&D companies, particularly in China, helped propel an unprecedented increase in the size of the biopharma pipeline in 2019. Meanwhile, the number of new active substances launched in their first market last year dropped as 2018’s superlative performance gave way to something more usual.

Topics

Related Companies

UsernamePublicRestriction

Register

CO180755

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