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AstraZeneca: US FDA grants saracatinib Orphan Drug Designation for idiopathic pulmonary fibrosis

The US Food and Drug Administration (FDA) has granted Orphan Drug
Designation (ODD) for saracatinib, a potential new medicine for the
treatment of idiopathic pulmonary fibrosis (IPF), a type of lung
disease that results in scarring (fibrosis) of the lungs. Saracatinib
is an inhibitor of src kinase which regulates broad cell functions
including cell growth and cell differentiation.1 Saracatinib has
completed Phase I development.

IPF is a chronic, progressive, irreversible and usually fatal
interstitial lung disease1 which affects approximately 100,000 people
in the US.2 On average, patients who are diagnosed with IPF live
between two and five years from diagnosis, given the limited
medicines available to treat the disease.1 The FDA grants ODD status
to medicines intended for the treatment, diagnosis or prevention of
rare diseases or disorders that affect fewer than 200,000 people in
the US.

Mene Pangalos, Executive Vice President, R&D BioPharmaceuticals, said:
"Idiopathic pulmonary fibrosis has a significant impact on patients'
lives and new therapies are urgently needed. IPF is a recent addition
to our respiratory research strategy and we are interested to see
whether saracatinib could be a useful approach for the treatment of
this intractable disease."

IPF is characterised by thickening and scarring of the connective
(interstitial) tissue in the lungs. The cause is thought to be due to
an abnormal wound-healing process that results in excessive tissue
build-up in the lung.1 Pre-clinical trials of saracatinib showed that
it inhibits fibroblast activity and collagen deposition, which are
key features of lung fibrosis.3

About IPF

IPF causes shortness of breath and progressive damage of the lung,
resulting in life-threatening complications such as respiratory
failure. IPF progression varies greatly between patients but over
time, most experience increasing respiratory symptoms, increased
scarring of the lungs and a gradual decline in lung function.
`Idiopathic' refers to the unknown cause of disease, however there is
proof of genetic predisposition in some patients.1

About saracatinib

Saracatinib is a small molecule, highly-potent and selective inhibitor
of src tyrosine kinase.3 The potential new medicine was discovered by
AstraZeneca and has previously been in clinical development in
oncology. Phase II trials for saracatinib in IPF have not yet

About AstraZeneca in Respiratory Disease

Respiratory is one of AstraZeneca's main therapy areas, and our
medicines reached more than 18 million patients as maintenance
therapy in 2018. AstraZeneca's aim is to transform asthma and COPD
treatment through inhaled combinations at the core of care, biologics
for the unmet needs of specific patient populations, and scientific
advancements in disease modification.

The Company is building on a 40-year heritage in respiratory disease
and AstraZeneca's capability in inhalation technology spans
pressurised metered-dose inhalers and dry powder inhalers, as well as
the Aerosphere Delivery Technology. The Company also has a growing
portfolio of respiratory biologics, including Fasenra
(anti-eosinophil, anti-IL-5r?), now approved for severe, eosinophilic
asthma and in development for severe nasal polyposis, and tezepelumab
(anti-TSLP), which has been granted Breakthrough Therapy designation
by the US Food and Drug Administration in patients with severe
asthma, and is in Phase III trials. AstraZeneca's research is focused
on addressing underlying disease drivers focusing on the lung
epithelium, lung immunity and lung regeneration.

About AstraZeneca

AstraZeneca is a global, science-led biopharmaceutical company that
focuses on the discovery, development and commercialisation of
prescription medicines, primarily for the treatment of diseases in
three therapy areas - Oncology, Cardiovascular, Renal & Metabolism
and Respiratory. AstraZeneca operates in over 100 countries and its
innovative medicines are used by millions of patients worldwide. For
more information, please visit
( and follow us on Twitter @AstraZeneca

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1. Lederer, D J and Martinez F J. Idiopathic Pulmonary Fibrosis. N Engl J Med. 2018;378:1811-23.

2. Genetic Home Reference of the National Institutes of Health National Library of Medicine, accessed on 16 October 2018.

3. Hu, M et al. Therapeutic targeting of src kinase in myofibroblast differentiation and pulmonary fibrosis. J Pharmacol Exp Ther 2014; 351:87-95.


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