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Pin to quick picksAstrazeneca Share News (AZN)

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AstraZeneca, Merck NF1 treatment granted orphan status by EMA

Fri, 03rd Aug 2018 07:52

(Sharecast News) - AstraZeneca and Merck said the European Medicines Agency (EMA) has granted orphan designation to selumetinib, a MEK 1/2 inhibitor, for the treatment of neurofibromatosis type 1 (NF1) an incurable genetic condition that affects one in 3,000 newborns worldwide. NF1 is caused by a spontaneous or inherited mutation in the NF1 gene and affects approximately one in 3,000 births, the two companies said.The disease is associated with many symptoms, including soft lumps on and under the skin, skin pigmentation, and, in 20-50% of patients, benign tumours on the nerve sheaths.People with NF1 may experience a number of other complications such as learning difficulties, visual impairment, twisting and curvature of the spine, high blood pressure, and epilepsy, Astra and Merck said.NF1 also increases a person's risk of developing other cancers, including malignant brain and peripheral nerve sheath tumours, and leukaemia. Symptoms begin during early childhood, with varying degrees of severity, and can reduce life expectancy by up to 15 years, they added."There is no cure for NF1, a life-long and devastating condition, and current treatment choices for these patients are very limited. The granting of an orphan designation is a positive step forward for children with NF1 and their families," said Sean Bohen, AstraZeneca executive vice-president, global medicines development and chief medical officer.In July 2017, AstraZeneca and Merck announced a global strategic oncology collaboration to co-develop and co-commercialise Lynparza, the world's first PARP inhibitor and potential new medicine selumetinib, a MEK inhibitor, for multiple cancer types.According to the two companies, orphan designation is a status assigned to a medicine intended for use in rare diseases. To be granted orphan status by the EMA, a medicine must be intended for the treatment, prevention or diagnosis of a disease that is seriously debilitating/life threatening and has a prevalence of up to five in 10,000 in the European Union.Additionally, the intended medicine must aim to provide significant benefit to those affected by the condition.
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8 Feb 2024 14:42

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8 Feb 2024 08:44

TOP NEWS: AstraZeneca confident after 2023 profit more than doubles

(Alliance News) - AstraZeneca PLC on Thursday said it expects another year of strong growth after reporting pretax profit in 2023 more than doubled.

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8 Feb 2024 07:58

AstraZeneca shares slip as Q4 profits miss forecasts

(Sharecast News) - Biopharma titan AstraZeneca delivered a 6% increase in sales in 2023 despite a $3.7bn decline in Covid-19 medicines revenues, as it guided to a strong pick-up in growth this year - though profits came in slightly under analysts' forecasts.

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8 Feb 2024 07:50

LONDON BRIEFING: Unilever sets new buyback; Astra profit surges

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2 Feb 2024 08:38

AstraZeneca celebrates latest successful trial of acoramidis in Japan

(Alliance News) - AstraZeneca PLC on Friday said positive results from a Phase 3 trial of acoramidis are consistent with global findings, bring the drug closer to regulatory submission in Japan.

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2 Feb 2024 06:22

TOP NEWS: US sends drugmakers initial offers in Medicare price talks

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1 Feb 2024 15:52

UK earnings, trading statements calendar - next 7 days

Friday 2 February 
YouGov PLCTrading Statement
Monday 5 February 
Home REIT PLCTrading Statement
Porvair PLCFull Year Results
Trident Royalties PLCTrading Statement
Vodafone Group PLCTrading Statement
Tuesday 6 February 
Alumasc Group PLCHalf Year Results
BP PLCFull Year Results
Filtronic PLCHalf Year Results
Mattioli Woods PLCHalf Year Results
Renishaw PLCHalf Year Results
Virgin Money UK PLCTrading Statement
Wednesday 7 February 
Ashmore Group PLCHalf Year Results
Barratt Developments PLCHalf Year Results
HydrogenOne Capital Growth PLCTrading Statement
Phoenix Spree Deutschland LtdTrading Statement
PZ Cussons PLCHalf Year Results
Smurfit Kappa Group PLCFull Year Results
UK Commercial Property REIT LtdTrading Statement
Thursday 8 February 
Anglo American PLCTrading Statement
AstraZeneca PLCFull Year Results
British American Tobacco PLCFull Year Results
Compass Group PLCTrading Statement
Redrow PLCHalf Year Results
SSE PLCTrading Statement
Unilever PLCFull Year Results
  
Comments and questions to newsroom@alliancenews.com
  
A full 21-day events calendar is provided each day with a subscription to Alliance News UK Professional.
  
Copyright 2024 Alliance News Ltd. All Rights Reserved.

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TOP NEWS: AstraZeneca, Daiichi's Enhertu granted priority review in US

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22 Jan 2024 11:45

BioNTech challenges AstraZeneca with breast cancer precision drug trial

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TOP NEWS: AstraZeneca's Voydeya receives approval in Japan for PNH

(Alliance News) - AstraZeneca PLC on Friday said its inhibitor Voydeya, also known as danicopan, received approval in Japan for adults with paroxysmal nocturnal haemoglobinuria, or PNH.

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16 Jan 2024 17:17

FTSE 100 hits one-month low, precious miners biggest drag

FTSE 100 down 0.5%, FTSE 250 flat

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16 Jan 2024 16:17

London close: Stocks finish lower as wage growth eases

(Sharecast News) - London's stock markets finished in the red on Tuesday, as investors assessed the latest UK jobs data.

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