recombinant factor IX
(Idelvion®)Approved, 2016-03-04 USFDA
A recombinant albumin-human coagulation factor IX (FIX) fusion protein indicated for the treatment and prevention of bleeding in patients with hemophilia B.
Research Code CSL-654
|Type||Recombinant coagulation factor|
- Albutrepenonacog alfa
Sequence Length: 1018modified (modifications unspecified)
- Originator CSL Behring
- Class Albumins; Antihaemorrhagics; Blood coagulation factors; Recombinant fusion proteins
- Mechanism of Action Blood coagulation factor replacements; Factor X stimulants
- Orphan Drug Status Yes – Haemophilia B
- Marketed Haemophilia B
Most Recent Events
- 21 Mar 2016 Launched for Haemophilia B (In adolescents, In children, In adults) in USA (IV) – First global launch
- 07 Mar 2016 Preregistration for Haemophilia B in Australia (IV) before March 2016
- 04 Mar 2016 Registered for Haemophilia B (In children, In adolescents, In adults) in USA (IV)
|Description||Fusion protein linking recombinant coagulation Factor IX with recombinant albumin|
|Molecular Target||Factor IX|
|Mechanism of Action|
|Therapeutic Modality||Biologic: Fusion protein|
|Latest Stage of Development||Approved|
|Indication Details||Treat and prevent bleeding episodes in hemophilia B patients; Treat hemophilia B|
|Regulatory Designation||U.S. – Orphan Drug (Treat and prevent bleeding episodes in hemophilia B patients);
EU – Orphan Drug (Treat and prevent bleeding episodes in hemophilia B patients);
Switzerland – Orphan Drug (Treat and prevent bleeding episodes in hemophilia B patients)
BNF Category:Antifibrinolytic drugs and haemostatics (02.11)
Pharmacology: Albutrepenonacog alfa is a recombinant factor IX (rIX-FP) albumin fusion protein, designed to exhibit an extended half-life. Factor IX has a short half-life which necessitates multiple injections. Epidemiology: Haemophilia B is a genetic disorder caused by missing or defective factor IX, a clotting protein. It has a prevalence of around 1 in 50,000 live births in the UK and is more common in males. In 2012-13, there were 476 hospital admissions in England due to haemophilia B, accounting for 508 finished consultant episodes and 125 bed days. Indication: Haemophilia B
Albutrepenonacog alfa was approved by the U.S. Food and Drug Administration (FDA) on March 4, 2016. It was developed and marketed as Idelvion® by CSL Behring.
Albutrepenonacog alfa is a recombinant albumin-human coagulation factor IX (FIX) fusion protein, which replaces the missing FIX needed for effective hemostasis. It is indicated for the treatment and prevention of bleeding in children and adults with hemophilia B.
Idelvion® is available as injection (lyophilized powder) for intravenous use, containing 250 IU, 500 IU, 1000 IU or 2000 IU of albutrepenonacog alfa in single-use vials. In control and prevention of bleeding episodes and perioperative management, the required dosage is determined using the following formulas: Required Dose (IU) = Body Weight (kg) x Desired Factor IX rise (% of normal or IU/dL) x (reciprocal of recovery (IU/kg per IU/dL)). In routine prophylaxis, the recommended dose is 25-40 IU/kg (for patients ≥12 years of age) or 40-55 IU/kg (for patients <12 years of age) every 7 days.
On 25 February 2016, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation for the medicinal product IDELVION, intended for treatment and prophylaxis of bleeding in patients with Haemophilia B. IDELVION was designated as an orphan medicinal producton 04 February 2010. The applicant for this medicinal product is CSL Behring GmbH.
IDELVION will be available as 250 IU, 500 IU, 1000 IU and 2000 IU Powder and solvent for solution for injection. The active substance of IDELVION is albutrepenonacog alfa, an antihaemorrhagic, blood coagulation factor IX, (ATC code: B02BD04). It works as replacement therapy and temporarily increases plasma levels of factor IX, helping to prevent and control bleeding.
The benefits with IDELVION are its ability to stop the bleeding when given on demand and prevent bleeding when used as routine prophylaxis or for surgical procedures. The most common side effects are injection site reaction and headache.
The full indication is: “the treatment and prophylaxis of bleeding in patients with Haemophilia B (congenital factor IX deficiency)”. Idelvion can be used in all age groups. It is proposed that IDELVION be prescribed by physicians experienced in the treatment of haemophilia B.
Detailed recommendations for the use of this product will be described in the summary of product characteristics (SmPC), which will be published in the European public assessment report (EPAR) and made available in all official European Union languages after the marketing authorisation has been granted by the European Commission.
|INN or common name||albutrepenonacog alfa|
|Therapeutic area||Hemophilia B|
|Active substance||albutrepenonacog alfa|
|Date opinion adopted||25/02/2016|
|Company name||CSL Behring GmbH|
|Application type||Initial authorisation|
//////Albutrepenonacog alfa, CSL-654, Idelvion; Recombinant factor IX – CSL Behring, Recombinant factor IX fusion protein linked with human albumin, rFIX-FP – CSL Behring; rIX-FP, Orphan Drug Status, Haemophilia B, recombinant factor IX , FDA 2016
|Human medicines European public assessment report (EPAR): Idelvion, albutrepenonacog alfa, Hemophilia B, 11/05/2016, , 9, Authorised|