Rare diseases

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In U-turn, NICE backs first drug for AL amyloidosis

Almost four years after it was approved in Europe to treat the incurable rare disease systemic amyloid light-chain (AL) amyloidosis, Johnson & Johnson’s Darzalex has b

News

Clinigen educates on early access to rare disease therapies

On Rare Disease Day, Clinigen has launched a campaign to try to encourage doctors and patients to explore how they can get early access to investigational therapies.

News

Novo Nordisk joins molecular glue club with Neomorph deal

Big pharma’s interest in using protein degradation as a therapeutic strategy shows no sign of abating, as Novo Nordisk signs a wide-ranging deal with US biotech Neomorph t

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FDA starts review of Argenx drug in rare disease CIDP

The US FDA has said it plans to deliver a decision in June on the use of Argenx’ Vyvgart Hytrulo in chronic inflammatory demyelinating polyneuropathy (CIDP), as the compan

R&D

Synlogic shuts down after PKU drug fails phase 3 trial

Shares in Synlogic have halved in value after the company reported that a phase 3 trial of its main pipeline drug for rare metabolic disorder phenylketonuria (PKU) failed

News

Roche gets first world okay for PNH drug crovalimab

China has become the first country in the world to approve Roche’s anti-complement C5 antibody crovalimab, the only treatment for the rare disorder paroxysmal nocturnal ha

In U-turn, NICE backs first drug for AL amyloidosis

Clinigen educates on early access to rare disease therapies

Novo Nordisk joins molecular glue club with Neomorph deal

FDA starts review of Argenx drug in rare disease CIDP

Synlogic shuts down after PKU drug fails phase 3 trial

Roche gets first world okay for PNH drug crovalimab

Ambrosia's sweet $100m round, and other biofinancings

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Rare diseases

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