The FDA is investigating reports of patients developing blood cancer after receiving bluebird bio’s gene therapy Skysona (elivaldogene autotemcel) and is evaluating the need for “further regulatory… read more.
Avapritinib is recommended, within its marketing authorisation, as an option for treating advanced systemic mastocytosis (including aggressive systemic mastocytosis, systemic mastocytosis with an associated haematological neoplasm and mast… read more.
NICE (UK): Danicopan (Voydeya) from Alexion/AstraZeneca, is recommended, as an add-on to ravulizumab or eculizumab as an option for treating paroxysmal nocturnal haemoglobinuria (PNH) in adults who have… read more.
Pfizer Inc. announced that the FDA has approved Hympavzi (marstacimab-hncq) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and pediatric patients 12… read more.
Pfizer Inc. announced that it is voluntarily withdrawing all lots of Oxbryta voxelotor) for the treatment of sickle cell disease (SCD) at this time, in all markets where… read more.
Burosumab is NICE recommended, within its marketing authorisation, for treating X- linked hypophosphatemia (XLH) with radiographic evidence of bone disease in babies, children and young people 1 to… read more.
Iptacopan is recommended, within its marketing authorisation, as an option for treating paroxysmal nocturnal haemoglobinuria (PNH) in adults with haemolytic anaemia. Iptacopan is only recommended if the company… read more.
Octapharma USA, Inc. has announced the expanded approval of fibryga, Fibrinogen (Human) Lyophilized Powder for Reconstitution, for fibrinogen replacement in bleeding patients with acquired fibrinogen deficiency (AFD) by… read more.
Pfizer Inc. announced that the European Commission (EC) has granted conditional marketing authorization for Durveqtix (fidanacogene elaparvovec), a gene therapy for the treatment of severe and moderately severe… read more.
Sobi announced the publication of full results from the Phase III XTEND-Kids study in The New England Journal of Medicine (NEJM) confirming the safety and efficacy profile of… read more.
GSK plc announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) has approved Omjjara (momelotinib) for the treatment of myelofibrosis. Omjjara is a once-a-day, oral JAK1/JAK2 and… read more.
The Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending a change to the terms of the marketing authorisation for the medicinal product Eliquis… read more.
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