Rare diseases

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AZ, Ionis get CHMP backing for ATTR polyneuropathy drug

AstraZeneca and Ionis are set fair for EU approval of Wainzua, their treatment for polyneuropathy associated with transthyretin-mediated amyloidosis (ATTR), after the EMA's human medicines

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Lundbeck drops in on epilepsy drug with $2.6bn Longboard bid

Neuroscience specialist Lundbeck has agreed to buy Longboard Pharmaceuticals for $2.6 billion, expanding its pipeline with an epilepsy drug tipped as a future blockbuster.

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FDA knocks back Zealand's dasiglucagon once again

Zealand Pharma has suffered another setback in its bid to bring glucagon receptor agonist dasiglucagon for ultra-rare disease congenital hyperinsulinism (CHI) to the US market.

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Second Niemann-Pick approval brings more hope to patients

In the space of four days, the number of FDA-approved therapies for ultra-rare genetic disorder Niemann-Pick disease has gone from zero to two, transforming the prospects for patients.

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FDA clears first drug for Niemann-Pick disease

Zevra Therapeutics' Miplyffa has become the first medicine to be approved by the FDA for the ultra-rare genetic disorder Niemann-Pick disease.

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NICE clears NHS use of MSD's Welireg for genetic disorder

Patients in England and Wales with a rare genetic disorder will soon be able to access treatment with MSD's Welireg via the NHS, nearly a year after their counterparts in Scotland.

AZ, Ionis get CHMP backing for ATTR polyneuropathy drug

Lundbeck drops in on epilepsy drug with $2.6bn Longboard bid

FDA knocks back Zealand's dasiglucagon once again

Second Niemann-Pick approval brings more hope to patients

FDA clears first drug for Niemann-Pick disease

NICE clears NHS use of MSD's Welireg for genetic disorder

Lilly bags FDA okay for Wegovy pill rival orforglipron

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