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darunavir-STR

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PureTech’s deupirfenidone gets FDA and EC Orphan Drug Designations for idiopathic pulmonary fibrosis

Rare diseases are defined as conditions affecting fewer than 200,000 people in the US or fewer than five in 10,000 individuals in the European Union

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GSK’s Blenrep combinations granted EC approval for relapsed/refractory multiple myeloma

More than 50,000 cases of the blood cancer are diagnosed in Europe each year

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Pfizer’s Hympavzi receives EC approval to treat haemophilia A and B patients

The genetic blood disorder affects more than 800,000 people globally

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Sanofi/Regeneron’s Dupixent approved by EC for younger eosinophilic oesophagitis patients

Dupixent is now the first drug in the EU indicated for use in this patient population

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Ipsen’s Kayfanda granted EC approval to treat cholestatic pruritus in Alagille syndrome

The rare genetic disorder affects approximately three in every 100,000 births globally

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EC approves Bavarian Nordic’s mpox vaccine Imvanex for use in adolescents

More than 120 countries have confirmed over 103,000 cases of mpox since the global outbreak began in 2022

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Janssen/Pharmacyclics’ ibrutinib shows continued survival benefit in chronic lymphocytic leukaemia

The slow-growing blood cancer affects approximately 4.92 per 100,000 people every year in Europe

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Janssen’s Carvykti recommended by CHMP for earlier multiple myeloma treatment

The blood cancer affects approximately 160,000 people worldwide

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Janssen reveals positive results for nipocalimab in rheumatoid arthritis

About 13 million people worldwide are affected by the chronic inflammatory disease

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Janssen’s Tecvayli approved by EC for reduced dosing frequency in multiple myeloma

Tecvayli was the first BCMA-targeting bispecific antibody to be approved in Europe

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Janssen’s Talvey granted FDA accelerated approval for difficult-to-treat blood cancer

The therapy showed an overall response rate of more than 70% in a broad range of patients

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Janssen receives positive CHMP opinion for Talvey in multiple myeloma

More than 50,900 people in Europe were diagnosed with multiple myeloma in 2020

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