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

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.”

Quick facts: AstraZeneca

Price: 8589 GBX

Market: LSE
Market Cap: £112.71 billion

Add related topics to MyProactive

Create your account: sign up and get ahead on news and events


The Company is a publisher. You understand and agree that no content published on the Site constitutes a recommendation that any particular security, portfolio of securities, transaction, or investment strategy is...



Redx Pharma PLC's Lisa Anson updates on lead asset RXC004 and increased...

Redx Pharma PLC's (LON:REDX) Lisa Anson recaps on its lead product RXC004 which is in a Phase I clinical trial and is part of a potentially breakthrough group of cancer drugs called Porcupine inhibitors. Anson also details the recent investment by its majority shareholder Redmile which has...

1 hour, 24 minutes ago

2 min read