Rare diseases

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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

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Amgen preps Uplizna filing for a second indication

Amgen has moved closer to a second indication for its B cell-depleting therapy Uplizna, acquired as part of its $27.8 billion takeover of Horizon Therapeutics last year, a

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FDA clears Amgen’s Bkemv as first biosimilar of Soliris

The US FDA has approved the first biosimilar version of AstraZeneca/Alexion’s Soliris therapy for paroxysmal nocturnal haemoglobinuria (PNH) and atypical haemolytic uraemi

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Maze gets new Pompe partner after FTC blocks Sanofi deal

Five months after Sanofi backed away from a proposed deal with Maze Therapeutics on a Pompe disease therapy after an antitrust challenge, Shionogi has snapped up the drug.

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Prime editing comes of age as FDA clears human trials

Prime Medicine has been given the go-ahead by the FDA for the first human trial of its prime editing technology, which promises to deliver one-shot therapies for a range o

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X4 Pharma gets first FDA okay for WHIM syndrome drug

Patients in the US with the ultra-rare disease WHIM syndrome now have their first approved treatment, after X4 Pharma’s Xolremdi was given a green light by the FDA.

KalVista files first oral on-demand HAE drug with FDA

Amgen preps Uplizna filing for a second indication

FDA clears Amgen’s Bkemv as first biosimilar of Soliris

Maze gets new Pompe partner after FTC blocks Sanofi deal

Prime editing comes of age as FDA clears human trials

X4 Pharma gets first FDA okay for WHIM syndrome drug

Threat of Trump's pharma tariffs looms again

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