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AstraZeneca’s Voydeya granted FDA approval for rare blood disease PNH

Up to 20% of patients treated with C5 inhibitors experience clinically significant extravascular haemolysis

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FDA grants Invivyd’s Pemgarda emergency use authorisation for COVID-19 prevention

The long-acting monoclonal antibody is authorised for use in adults and adolescents

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Merck’s Winrevair granted FDA approval to treat pulmonary arterial hypertension in adults

The rare blood vessel disorder affects approximately 500 to 1,000 people in the US every year

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AstraZeneca’s Ultomiris receives FDA approval to treat rare autoimmune disease NMOSD

Neuromyelitis optica spectrum disorder is estimated to affect approximately 6,000 adults in the US

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Johnson & Johnson’s Opsynvi granted FDA approval in pulmonary arterial hypertension

The rare form of pulmonary hypertension is responsible for up to 1,000 new cases in the US every year

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AbbVie’s Elahere granted full FDA approval for previously-treated ovarian cancer

The company recently gained access to the therapy through its $10.1bn acquisition of ImmunoGen

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FDA approves Italfarmaco’s Duvyzat as first nonsteroidal Duchenne muscular dystrophy drug

The neuromuscular genetic disease is estimated to affect one in every 3,500 to 6,000 male births worldwide

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Gates Medical Research Institute initiates phase 3 study of tuberculosis vaccine candidate

According to WHO, the infectious disease was estimated to affect 10.6 million people in 2022

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Takeda’s Iclusig combination granted FDA accelerated approval for rare form of leukaemia

The drug is now the first targeted therapy approved as a frontline treatment for Ph-positive ALL

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FDA approves Orchard’s Lenmeldy gene therapy for metachromatic leukodystrophy

The rare genetic disease is estimated to affect one in every 40,000 people in the US

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FDA approves Madrigal’s Rezdiffra as first drug for fatty liver disease NASH

Over six million people in the US are affected by NASH with moderate-to-advanced liver scarring

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GSK’s Jemperli regimens demonstrate potential for wider endometrial cancer use

Approximately 417,000 new cases of endometrial cancer are reported globally every year

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