Rare diseases

News

FDA panel backs Zevra's arimoclomol, after earlier rejection

An FDA advisory committee has recommended approval of Zevra Therapeutics' arimoclomol for the ultra-rare disorder Niemann-Pick disease, three years after the regulator rejected the drug.

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Otsuka buys Jnana and PKU drug in $1.1bn deal

Japan's Otsuka Pharma has agreed to take control of US biotech Jnana Therapeutics in a deal that will give it rights to a clinical-stage candidate for the rare metabolic disorder phenylketo

News

Ipsen's Alagille drug backed by CHMP again, with new name

In unusual circumstances, Ipsen has been given the green light for its odevixibat drug for severe itching (pruritus) in patients with rare liver disease Alagille syndrome (ALGS) in the EU –

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Ionis plans phase 3 for Biogen-rejected Angelman drug

Ionis has committed to a phase 3 programme for its antisense therapy for rare genetic disorder Angelman syndrome, just weeks after Biogen decided against exercising an option to license the

News

Lexeo therapy gives hope to Friedreich's ataxia patients

An experimental gene therapy developed by Lexeo Therapeutics has shown early promise in treating one of the most serious complications of the rare neurodegenerative disorder, Friedreich's a

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