Rare diseases

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Alfasigma claims early FDA nod for ex-GSK liver drug

Alfasigma has claimed the first approval, in the US, for linerixibat, a drug for liver disease PBC licensed from GSK for $690m a few days ago.

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BioMarin halts Voxzogo trials due to safety signal

BioMarin has suspended dosing in phase 2 trials of its achondroplasia therapy Voxzogo aimed at broadening the drug's label.

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Multicoloured letters with AUTISM spelled in the middle

FDA approves leucovorin – but not for autism

The FDA stops short of approving leucovorin for autism, but will its clearance for ultra-rare condition CFD-FOLR1 lead to more off-label prescribing?

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Alfasigma licenses liver drug from GSK in $690m deal

With an FDA verdict looming, GSK has signed an eleventh-hour deal to give Alfasigma rights to its primary biliary cholangitis therapy linerixibat.

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PepGen slumps as FDA places hold on lead drug

Shares in PepGen fell sharply after the FDA placed a partial clinical hold on its lead drug for rare neuromuscular disease DM1.

News

Ascendis scores FDA approval for weekly dwarfism drug

Children with achondroplasia will soon have the option of a once-weekly - rather than daily - injection, courtesy of Ascendis' newly approved Yuviwel.

Alfasigma claims early FDA nod for ex-GSK liver drug

BioMarin halts Voxzogo trials due to safety signal

FDA approves leucovorin – but not for autism

Alfasigma licenses liver drug from GSK in $690m deal

PepGen slumps as FDA places hold on lead drug

Ascendis scores FDA approval for weekly dwarfism drug

Non-profit DNDi preps phase 3 trials for dengue antibody

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