Sanofi and Sobi’s once-weekly haemophilia A treatment has demonstrated highly effective bleed protection in children with severe forms of the disease, according to late-stage data presented at the International Society on Thrombosis and Haemostasis (ISTH) annual meeting. The phase 3 XTEND-Kids study evaluated a once-weekly 50IU/kg dose of the factor VIII replacement therapy, Altuviiio, for 52 weeks in previously treated patients aged under 12 years. The oral presentation at ISTH detailed results from the study and confirmed that the therapy met the primary endpoint, with no inhibitor development to factor VIII detected. Key secondary endpoints were also met, including annualised bleeding rate and maintenance of factor VIII activity above pre-specified levels. Sanofi added that the therapy was well-tolerated and demonstrated a safety profile similar to the phase 3 XTEND-1 trial, confirming safety and efficacy in both adults and children. Haemophilia A is a rare, lifelong bleeding disorder in which a ...
Shire’s Adynovi has been approved for use in Europe for on-demand and prophylactic use in patients 12 years and older with the rare bleeding disorder haemophilia A.
Irish biotechnology firm Shire has received 510(k) marketing clearance for its myPKFiT for ADVATE [Antihemophilic Factor (Recombinant)] software from the US Food and Drug Administration (FDA).
The National Institute for Health and Care Excellence (NICE) has issued a draft Appraisal Consultation Document that does not recommend CSL Behring’s Hemgenix (etranacogene dezaparvovec) gene therapy for haemophilia B. The company has been seeking approval for use of the therapy in adults with severe or moderately severe haemophilia B without a history of factor IX inhibitors. In its guidance, NICE’s evaluation committee said that while there is clear evidence that the therapy reduces the number of bleeding episodes haemophilia B patients have each year, there is not enough evidence on how well it works in the long term, meaning cost-effectiveness estimates are “uncertain”. It also cited problems with the indirect comparison between the gene therapy and factor IX replacement therapies. Haemophilia B is a genetic bleeding disorder resulting from missing or insufficient levels of blood clotting factor IX, a protein needed to produce blood clots to stop bleeding. Patients ...
Hemophilia B is defined as a bleeding disorder, caused by a defect in genes coded for a protein named clotting factor IX (FIX). More than 400, 000 people are affected with hemophilia B worldwide.
The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has put forward seven new medicines for approval in the region, including new treatment options for haemophilia A, diabetes, and a very rare genetic disorder.
US-based Generation Bio is cutting its workforce by 40%, becoming the latest company to announce lay-offs to save money this year. The restructuring includes the departure of key personnel, with medical chief Douglass Kerr and development leader Tracy Zimmerman among those leaving. The 29 November announcement adds to a series of setbacks in the field, with the cell therapy startup NexImmune reducing its workforce by over half this year, halting the development of its three adoptive T cell therapies. Generation anticipates that the downsizing and streamlining of its research and development focus will result in a cost-saving of $120m over the next three years, as outlined in a 27 November SEC filing. In April, Takeda announced that it was moving away from the adeno-associated virus (AAV) gene therapies sector amidst difficulties associated with gene therapy research development at the preclinical stage, highlighting that it is unlikely that many current AAV ...
Swedish Orphan Biovitrum (Sobi) has said it will acquire CTI BioPharma for approximately $1.7bn, marking a notable expansion to the Swedish drugmaker’s rare haematology pipeline. The deal will grant Sobi access to the US biopharma’s recently-approved Vonjo (pacritinib), a novel oral kinase inhibitor for patients with myelofibrosis and severe thrombocytopenia. The drug was granted accelerated approval by the US Food and Drug Administration (FDA) in February last year to treat adults with intermediate or high-risk primary or secondary myelofibrosis with low platelet counts. This was based on positive results from a phase 3 trial of Vonjo in patients with myelofibrosis. Results showed that 29% of patients receiving Vonjo had a reduction in spleen volume of at least 35% compared to 3% of patients receiving best available therapy. Sobi said Vonjo will be ‘highly complementary’ to Doptelet (avatrombopag), its oral thrombopoietin receptor agonist which also addresses rare haematological platelet disorders. Guido ...
March 23 (Reuters) – Sanofi’s (SASY.PA) asthma drug Dupixent met all targets in a trial to treat “smoker’s lung”, potentially adding billions to the French drugmaker’s growth prospects, but also underscoring a heavy reliance on its bestseller. In a late stage trial Dupixent, jointly developed with Regeneron (REGN.O), led to a 30% reduction in moderate or severe acute exacerbations of chronic obstructive pulmonary disease (COPD), a potentially deadly disease marked by progressive lung function decline. Shares in Sanofi and Regeneron surged after they said in a joint statement on Thursday a Phase III trial involving 939 current or former smokers also showed improvements in lung function, quality of life and respiratory symptoms. The French group’s stock was up 5.2% at 1205 GMT, reaching a seven-month high and far outperforming the STOXX Europe 600 Health Care index (.SXDP), which was little changed. Regeneron jumped 8.6% in U.S. ...
Dutch biotech UniQure has won orphan drug designation (ODD) for its investigational treatment for Huntington’s Disease, a rare, inherited neurodegenerative disorder that leads to loss of muscle coordination, behavioural abnormalities and cognitive decline.
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