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Opdivo

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FDA gives Rare Paediatric Disease designation to Satellite Bio for urea cycle disorders

The mortality rates of UCDs exceeds 25%, with no curative treatment available for newborns

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EMA’s PRIME scheme launches three major new features

The scheme supports the development of treatments targeting unmet medical needs in the EU

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Bristol Myers Squibb evolves and expands multiple myeloma programme

Created in 2016, the Standing in the Gaap programme aims to advance more equitable care

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BMS’ Opdivo gets expanded FDA and EU approval for Hodgkin lymphoma

This type of cancer is the most common one diagnosed in teenagers

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Bristol Myers Squibb’s Sotyktu approved by US FDA for psoriatic arthritis

Sotyktu is the first TYK2 inhibitor approved for active psoriatic arthritis

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US FDA launches ultra-rare diseases framework

The draft guidance specifically discusses genome editing and RNA-based therapies

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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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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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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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BMS announces affordable drug price agreement with US government

The company will provide Eliquis, its oral blood thinner, free of charge to Medicaid

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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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