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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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Diabeloop’s DBLG2 receives FDA and CE clearance for automated insulin delivery without meal input

While a lack of meal announcements can lead to lower glycaemic control, it reduces the mental burden for those with type 1 diabetes

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Sanofi announces positive results for venglustat in type 3 Gaucher disease

There are currently no approved treatments for the neurological symptoms of this disease

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Sanofi’s Rezurock receives positive CHMP opinion for chronic graft-versus-host disease

Up to 50% of allogeneic haematopoietic stem cell transplant patients develop chronic GVHD

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Johnson & Johnson’s Darzalex Faspro regimen receives FDA approval for multiple myeloma

Multiple myeloma has an average five-year survival rate of 59.8% and is currently incurable

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Sanofi and Regeneron’s Dupixent receives positive NICE final draft guidance for COPD

Around 1.4 million people in the UK are diagnosed with COPD, with two million thought to be undiagnosed

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Sanofi’s amlitelimab shows positive results in atopic dermatitis

Newly released study data shows consistent reductions in disease severity

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Novartis’ ianalumab receives FDA Breakthrough Therapy designation for Sjögren’s disease

Approximately half of those with Sjögren’s disease are thought to be undiagnosed

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FDA approves first gene therapy for Wiskott-Aldrich syndrome

Symptoms of the rare disease include bleeding, eczema and frequent infections

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FDA launches TEMPO digital health pilot

Digital technologies can improve remote patient care for those with chronic diseases

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Richard Pazdur, FDA’s drug evaluation chief, to leave FDA just weeks into new role

Pazdur was previously head of the FDA’s Oncology Center of Excellence

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Merck KGaA given FDA Fast Track designation for generalised myasthenia gravis drug

Cladribine capsules could become the first ever oral treatment for gMG

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