Vonicog alfa

24556511000001108

New Medicines

Veyvondi (EU), Vonvendi (US) Treatment of haemorrhage and surgical bleeding, and prevention of surgical bleeding in adults with von Willebrand disease

Information

Veyvondi (EU), Vonvendi (US)
New molecular entity
Takeda
Takeda

Development and Regulatory status

Launched
Launched
Launched
October 2019
Yes
Yes
Nov 19Veyvondi is currently available only via a Named Patient Access Program in the UK (patientprograms@shire.com). It has been reviewed by the NHSE Clinical Priorities Advisory Group, and Shire expects a decision on reimbursement in Jan 20; if successful, Veyvondi will be reimbursed from Apr 20 [14].
Oct 19Available in the UK. Price: 650iu, 1=£643.50. 1300iu, 1=£1287.00 [13].
Sep 18Approved in EU [12].
Jun 18Recommended for EU approval by CHMP - the full indication is "in adults (age 18 and older) with von Willebrand disease (VWD), when desmopressin (DDAVP) treatment alone is ineffective or not indicated for the: - Treatment of haemorrhage and surgical bleeding; - Prevention of surgical bleeding. Veyvondi should not be used in the treatment of Haemophilia A." It is proposed that vonicog alfa be prescribed by physicians experienced in the treatment of haemostatic disorders [11].
Jun 17Shire announces that an EU Marketing Authorisation application has been filed with and validated by the EMA, based on the results of PIII trial programme. VEYVONDI was studied in patients (18 to 64 years), in a multi-center, open label, non-randomized study assessing safety, efficacy and pharmacokinetics, with and without rFVIII (NCT01410227) and in patients ≥18 years, in a prospective, uncontrolled, international, multi-center, open label, non-randomized study assessing control of hemostasis before, during or after surgical procedures, with or without rFVIII (NCT02283268) [6].
Dec 16Shire reports that a PIII trial (NCT02283268) in adult patients with severe, hereditary VWD who were undergoing minor and major elective surgical procedures met its primary endpoint of effectively controlling bleeding and blood Loss. The complete results will be used to support the filing for regulatory approval in EU in 2017 [10].
Aug 16Launched in the US [9].
Dec 15Approved in US [8].
Jul 15Filed for on-demand use in US; PIII for use during surgery [7].
Dec 14Baxter submits application for FDA approval of BAX111 for the treatment of Von Willebrand Disease [5].
Apr 14Baxter plans to file for approval before end of 2014 and intends to pursue a study of BAX 111 in a prophylaxis treatment setting before the end of the year [4].
Apr 14Designated orphan drug status in US in Nov 10 [4].
Oct 11Designated orphan drug status in EU in Nov 10 [2].
Sep 11PIII study started [1].

Category

Blood-coagulation-factor
Affects <2 in 10,000 people in the EU - fewer than 101,000 people in total [2].
Treatment of haemorrhage and surgical bleeding, and prevention of surgical bleeding in adults with von Willebrand disease
Intravenous

Trial or other data

Aug 15PIII data published in Blood shows BAX 111 successfully managed bleeding episodes for patients with von Willebrand disease. The treatment met its goals by helping all of the trial´s 37 patients stem bleeding when used on demand, Baxalta said, and one infusion was enough to control about 82% of bleeds [6].
Apr 14Results reported from the PIII study. The study of BAX 111 met its primary efficacy endpoint, as all patients achieved pre-specified success in the on-demand treatment of bleeding events (100%, 22 of 22 patients who experienced bleeds in the trial). There were no reports of inhibitor development or thrombotic events. The most common AEs were headache, vomiting/nausea and iron deficiency anaemia, which were not considered to be related to treatment. There was one serious adverse event related to treatment, characterized by chest discomfort and increased heart rate during infusion, which rapidly resolved without further complication [4].
Mar 13Estimated NCT01410227 completion Date March 2014 [3].
Oct 11NCT01410227 is a non-randomised, crossover PIII study assessing efficacy and safety of recombinant von Willebrand factor (rVWF) for treating and preventing bleeding episodes in 32 subjects with von Willebrand Disease (VWD). The primary outcome is the number of subjects with treatment success (defined as a mean efficacy rating score of <2.5 for a subject´s bleeding within the 12 month treatment period. Scores used to assess the extent of control of the bleeding episodes: Excellent =1, Good =2, Moderate =3, None =4.) Subjects will be treated with 50 or 80IU/kg rVWF IV – with or without rFVIII (ADVATE). Subjects must have been diagnosed with type 3 VWD (VWD:Ag ≤3 IU/dL) or severe non-type 3 VWD (VWF:RCo <20 IU/dL) or type 2N VWD (FVIII:C <10% and documented genetics) and have had a minimum of 6 documented bleeds requiring VWF coagulation factor replacement therapy during the previous 2 years. The study started in Sep 11 and is due to complete Feb 13 [1].

Evidence based evaluations

Veyvondi (EU), Vonvendi (US) Severe von Willebrand disease in children aged up to 17 years - on-demand treatment of non-surgical and surgical (elective and emergency) bleeding episodes

Information

Veyvondi (EU), Vonvendi (US)
Licence extension / variation
Takeda
Takeda

Development and Regulatory status

Phase III Clinical Trials
Phase III Clinical Trials
Phase III Clinical Trials
Yes
Yes

Category

Blood-coagulation-factor
Affects <2 in 10,000 people in the EU - fewer than 101,000 people in total [2].
Severe von Willebrand disease in children aged up to 17 years - on-demand treatment of non-surgical and surgical (elective and emergency) bleeding episodes
Intravenous

Veyvondi (EU), Vonvendi (US) Prophylactic treatment in adults with von Willebrand disease

Information

Veyvondi (EU), Vonvendi (US)
Licence extension / variation
Takeda
Takeda

Development and Regulatory status

None
Phase III Clinical Trials
Phase III Clinical Trials
Yes
Yes

Category

Blood-coagulation-factor
Affects <2 in 10,000 people in the EU - fewer than 101,000 people in total [2].
Prophylactic treatment in adults with von Willebrand disease
Intravenous