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haemophilia

NHS England to fund Hemlibra for patients with severe haemophilia

NHS England is to fund Roche and Chugai Pharma UK’s haemophilia drug Hemlibra, for people with sever congenital haemophilia A without factor VIII inhibitors.

Roche unveils wave of strong Hemlibra data in haemophilia A with or without factor VIII inhibitors

Roche revealed a swathe of new data on Hemlibra (emicizumab) at the International Society on Thrombosis and Haemostasis (ISTH) 2019 Congress, reinforcing the bispecific factor IXa- and factor X-directed antibody’s efficacy and safety in the treatment of haemophilia A in patients with or without factor VIII inhibitors.

The presented data was pooled from four separate HAVEN studies, drawn from participants of all ages with and without factor VIII inhibitors over a median of 83 weeks.

Pfizer and Sangamo's haemophilia A gene therapy shows promise at Phase 1/2

Pfizer and Sangamo Therapeutics have shared new Phase 1/2 data for their investigational SB-525 gene therapy in the treatment of haemophilia A, showing that it was “generally well-tolerated and demonstrated a dose-dependent increase in Factor VIII (FVIII) activity levels.”

The shared data was drawn from research conducted in 10 participants and was presented at the 27th Congress of the International Society on Thrombosis and Haemostasis (ISTH), in Melbourne, Australia.

European approval for Novo's Esperoct in haemophilia A

The European Commission has awarded approval to Novo Nordisk’s extended half-life factor VIII therapy Esperoct (turoctocog alfa pegol, N8-GP) for the treatment of haemophilia A in adolescent and adult patients, the drugmaker has revealed.

Specifically, the approval covers “the prophylaxis and on-demand treatment of bleeding as well as for surgical procedures”.

Sangamo and Pfizer's gene therapy increases FVIII activity in severe haemophilia A in early study

Sangamo Therapeutics and Pfizer have unveiled new Phase 1/2 data for their jointly developed investigational gene therapy SB-525, demonstrating that the treatment “was generally well-tolerated and demonstrated a dose-dependent increase in Factor VIII (FVIII) levels” across four dosages in severe haemophilia A.

The study evaluated the efficacy of four different dosages (9e11 vg/kg, 2e12 vg/kg, 1e13 vg/kg and 3e13 vg/kg) in eight participants, with two in each group.

Roche scores two EU approvals in haemophilia and rare autoimmune disease

The European Commission (EC) has approved Roche’s haemophilia treatment Hemlibra (emicizumab).

The approval comes as the EC approved Roche's MabThera (rituximab) for the rare autoimmune disease pemphigus vulgaris (PV) marking the first major advancement in treatment fo the disease in more than 60 years.

Hemlibra, a bispecific antibody targeting factor IXa- and factor X was approved as a treatment in routine prophylaxis of bleeding episodes in severe haemophilia A patients without factor VIII inhibitors.

Roche and Chugai's Hemlibra scoops up EU approval in the prevention of severe haemophilia A without factor VIII inhibitors

Roche has announced along with its partner Chugai that Hemlibra (emicizumab) has secured approval from the European Commission as a preventative treatment for bleeding episodes in patients with severe haemophilia A without factor VIII inhibitors.

The drug is suitable for use in any age of patient and can be self-administered via subcutaneous injection every one, two or four weeks. It already held authorisation for the treatment of haemophilia A with factor VIII inhibitors

Pfizer's biosimilar proves non-inferior to rituximab in lymphoma at ASH 2018

Joining the chorus of data presentations at the American Society of Hematology (ASH) Annual Meeting 2018, Pfizer has announced new data for its biosimilar version of Genentech’s Rituxan/MabThera (rituximab), revealing that the drug met its primary endpoint.

The 26-week data show that PF-05280586, as the biosimilar is known, proved itself to be clinically non-inferior in terms of overall response rate compared to rituximab in the first-line treatment of CD20-positive, low tumour burden, follicular lymphoma (LTB-FL); the drug recorded an ORR of 75.7% versus 70.7% for rituximab.

Hemlibra shows "clinically meaningful" bleed control in paediatric haemophilia A patients

In another reveal at the American Society of Hematology (ASH) Annual Meeting 2018, Roche unveiled new Phase 3 findings for Hemlibra (emicizumab) as a prophylactic in haemophilia A patients with factor VIII inhibitors younger than 12 years of age.

The data, drawn from the largest pivotal study of this patient population, showed that 76.9% of participants treated once weekly experienced zero treated bleeds over the course of the study period.

Bayer's Jivi approved in Europe for previously treated haemophilia A

Bayer has revealed that its recombinant Factor VIII (rFVIII) replacement therapy Jivi, also known as BAY94-9027, has received approval from the European Commission for the treatment and prophylaxis of bleeding in previously treated haemophilia A patients aged 12 years or older.

As a prophylactic, Bayer recommends that Jivi be used every five days, but notes that it can also we used weekly or twice-weekly.

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