Rare diseases

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Rare disease drug from LEO's Timber felled by failed study

Just a few months after buying Timber Pharma, LEO Pharma has revealed that the main drug it acquired as part of the deal has failed a phase 3 trial.

News

PBC drug choices rise in US with OK for Gilead's seladelpar

At the start of the year, there had been no new therapies for rare cholestatic liver disease primary biliary cholangitis (PBC) in the US for almost a decade.

News

FDA okays Ascendis hypoparathyroidism drug at second attempt

After turning it down a year ago, the FDA has cleared once-daily injectable Yorvipath for hypoparathyroidism from Ascendis Pharma.

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.

News

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 –

Rare disease drug from LEO's Timber felled by failed study

PBC drug choices rise in US with OK for Gilead's seladelpar

FDA okays Ascendis hypoparathyroidism drug at second attempt

FDA panel backs Zevra's arimoclomol, after earlier rejection

Otsuka buys Jnana and PKU drug in $1.1bn deal

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

Neurogene drops high-dose arm in Rett study after death

Coming soon: The Life Sciences Industry Report 2025 – insigh...

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