Rare diseases

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 –

News

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

Sales & Marketing

How data is critical to commercialising drugs for rare disea...

Orphan drugs are designed to treat rare diseases, but “rare” is a relative term.

News

KalVista files first oral on-demand HAE drug with FDA

KalVista Pharma has filed its first marketing application to the US FDA, seeking approval for its oral plasma kallikrein inhibitor sebetralstat as an oral treatment for hereditary angioedem

Otsuka buys Jnana and PKU drug in $1.1bn deal

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

Ionis plans phase 3 for Biogen-rejected Angelman drug

Lexeo therapy gives hope to Friedreich's ataxia patients

How data is critical to commercialising drugs for rare disea...

KalVista files first oral on-demand HAE drug with FDA

The long-delayed promise of the microbiome, with Sam Possemi...

How to meet your physicians where they are online

Rare diseases

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