Proactive Investors - Run By Investors For Investors

AstraZeneca granted Breakthrough Therapy Designation for rare genetic condition treatment

A breakthrough therapy designation is designed to expedite the development and review of medicines that are intended to treat a serious condition and have shown promise in early-stage clinical trials
laboratory
NF1 is a rare genetic condition that people are born with and can lead to learning difficulties

AstraZeneca PLC’s (LON:AZN) potential treatment for a rare genetic condition called neurofibromatosis type 1 (NF1) has been granted breakthrough therapy designation (BTD) by US regulators.

The move from the US Food and Drug Administration effectively fast-tracks the regulatory review of the medicine, called selumetinib, which Astra is working on with fellow pharma giant Merck & Co Inc (NYSE:MRK).

READ: AstraZeneca launches UK's biggest cash call this year

NF1 is a rare genetic condition that causes tumours to grow along a person’s nerves which can lead to various skin issues and learning difficulties, as well as problems with the bones, eyes and nervous system.

People with NF1 also have a much higher rate of cancer and cardiovascular disease.

The BTD is based on data from a Phase II monotherapy trial last summer which showed that selumetinib can reduce pain and motor impairment.

“Selumetinib shows promise in the treatment of NF1-related plexiform neurofibromas, a rare and debilitating disease with no approved medications to date,” said Astra’s oncology R&D boss José Baselga.

“The Breakthrough Therapy Designation acknowledges the significant unmet need of these patients and the potential benefit of selumetinib in this setting.”

View full AZN profile View Profile

AstraZeneca Timeline

Related Articles

copd radiograph
April 02 2019
City broker Peel Hunt reckons the recent approvals “support the top-line forecasts in our model that should see Circassia achieve EBITDA positivity in 2020”
antibodies
May 02 2019
Avacta is using Affimers to develop its own cancer therapy, but it also licenses the technology to other companies, including major pharma group ModernaTX and LG Chem Life Sciences
consumer health segment overview
January 11 2019
The company sells clinically validated anti-ageing products for hair, skin and body.

© Proactive Investors 2019

Proactive Investors Limited, trading as “Proactiveinvestors United Kingdom”, is Authorised and regulated by the Financial Conduct Authority.
Registered in England with Company Registration number 05639690. Group VAT registration number 872070825 FCA Registration number 559082. You can contact us here.

Market Indices, Commodities and Regulatory News Headlines copyright © Morningstar. Data delayed 15 minutes unless otherwise indicated. Terms of use