Rare diseases

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New data in C3G sets up filings for Novartis’ Fabhalta

Just days after getting FDA approval for Fabhalta as the first oral therapy for paroxysmal nocturnal haemoglobinuria (PNH), Novartis has reported new data showing it is al

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Novartis’ Fabhalta is first FDA-cleared oral therapy for PNH

Novartis’ targeted factor B inhibitor iptacopan has become the first oral monotherapy to be approved by the FDA for rare blood disorder paroxysmal nocturnal haemoglobinuri

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MSD reaches $610m deal to buy lysosome biotech Caraway

MSD has agreed to pay up to $610 million to acquire Caraway Therapeutics, a Cambridge, Massachusetts biotech focusing on neurodegenerative disorders and rare diseases.

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Takeda gets okay in US for rare blood disorder drug

Patients in the US with ultra-rare blood clotting disorder congenital thrombotic thrombocytopenic purpura (cTTP) have an FDA-approved therapy for the first time, after Tak

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X4 cues up FDA verdict in April for WHIM syndrome drug

The FDA has started a priority review of X4 Pharma’s mavorixafor, angling to become the first therapy for the rare disease WHIM syndrome, with a decision due by 30th April

News

Ipsen to refile Alagille drug after EU blocks orphan status

Ipsen has said that it plans to file a new marketing application for its treatment for itch in Alagille syndrome (ALGS), Bylvay, after failing to persuade an EMA committee

New data in C3G sets up filings for Novartis’ Fabhalta

Novartis’ Fabhalta is first FDA-cleared oral therapy for PNH

MSD reaches $610m deal to buy lysosome biotech Caraway

Takeda gets okay in US for rare blood disorder drug

X4 cues up FDA verdict in April for WHIM syndrome drug

Ipsen to refile Alagille drug after EU blocks orphan status

MSD, Moderna take mRNA jab into phase 3 for lung cancer

Revolutionising research: Inside the NCVR evolution in the U...

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