French biotech Pharnext has seen the value of its shares collapse after it revealed that its lead drug, a treatment for Charcot-Marie-Tooth (CMT) disease, failed to make t
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
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
MSD has agreed to pay up to $610 million to acquire Caraway Therapeutics, a Cambridge, Massachusetts biotech focusing on neurodegenerative disorders and rare diseases.
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
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