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Beta thalassaemia

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MHRA extends licence of Vertex’s cystic fibrosis drugs for young children

Kaftrio and Kalydeco are now approved for use in certain patients aged two to five years

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AstraZeneca’s Truqap plus Faslodex receives FDA approval for advanced breast cancer

About 290,000 people in the US will be diagnosed with breast cancer in 2023

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Bristol Myers Squibb’s lung cancer drug Augtyro receives FDA approval

The drug was added to BMS’ portfolio last year through its $4.1bn acquisition of Turning Point

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MHRA authorises world-first gene therapy for two inherited blood disorders

Casgevy is now the first licensed treatment that uses the gene-editing tool CRISPR

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Takeda’s enzyme replacement therapy approved by FDA for rare blood clotting disorder

The ultra-rare inherited disease is estimated to affect fewer than 1,000 people in the US

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Takeda’s Fruzaqla receives FDA approval for metastatic colorectal cancer

More than 150,000 new cases of colorectal cancer will be diagnosed in the US in 2023

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Valneva’s chikungunya vaccine approved by FDA for adults at increased exposure risk

The decision makes Ixchiq, which is administered as a single injection, the world's first licensed vaccine against the mosquito-borne disease

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Eli Lilly’s tirzepatide injection approved by MHRA and FDA for weight loss in adults

The drug has been authorised for use alongside a reduced-calorie diet and physical activity

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US FTC challenges more than 100 patents as ‘improperly listed’ with FDA

The agency has sent letters to ten companies including AbbVie, AstraZeneca and GSK

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FDA grants fast track designation to AviadoBio’s dementia gene therapy

The regulator has also given clearance for the candidate to be studied in frontotemporal dementia patients

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bluebird bio presents positive results for inherited blood disorder gene therapies

The company’s sickle cell disease therapy was accepted for FDA priority review earlier this year

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Merck’s Keytruda combination granted FDA approval for biliary tract cancer

Approximately 20,000 people are diagnosed with the disease each year in the US

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