Clinical Edge

Summaries of Must-Read Clinical Literature, Guidelines, and FDA Actions

FDA Approves Rebinyn to Treat Hemophilia B

Novo Nordisk news release; 2018 Feb 8

The FDA has approved Rebinyn for the treatment of hemophilia B.

Indications: Rebinyn, Coagulation Factor IX (Recombinant), GlycoPEGylated, is a recombinant DNA-derived coagulation Factor IX concentrate indicated for use in adults and children with hemophilia B for:

  • On-demand treatment and control of bleeding episodes.
  • Perioperative management of bleeding.

Dosage and administration: 40 IU/kg body weight for minor and moderate bleeds/minor surgery, and 80 IU/kg body weight for major bleeds/major surgery.

Efficacy and safety: In phase 3 studies, a single dose of 40 IU/kg in adults elevated factor activity above baseline levels by 94% and sustained average factor levels of 17% 7 days post-dose. Adults also achieved an 83-hour average half-life after receiving a single infusion.

Side effects/risks: The most common adverse reactions are itching and injection site reactions.

Citation:

Novo Nordisk launches Rebinyn in the United States for people with hemophilia B. [news release]. Plainsboro, NJ: Novo Nordisk Inc. February 8, 2018. http://press.novonordisk-us.com/2018-02-08-Novo-Nordisk-Launches-Rebinyn... Accessed February 12, 2018.

Rebinyn [package insert]. Plainsboro, NJ: Novo Nordisk Inc. 2018. https://www.fda.gov/downloads/BiologicsBloodVaccines/BloodBloodProducts/.... Accessed February 12, 2018.

This Week's Must Reads

Case studies shed light on treatment for rare form of MCL , Mori S et al. Clin Lymphoma Myeloma Leuk. 2019 Feb;19(2):e93-7.

Histopathological clues for differentiating follicular lymphomas, Servitje O et al. J Cutan Pathol. 2019;46:182-9.

Single agent daratumumab is not an option in B-cell lymphomas, Salles G et al. Clin Lymphoma Myeloma Leuk. 2019 Jan 2. doi: 10.1016/j.clml.2018.12.013.

Ibrutinib combo advances in CNS lymphoma, Grommes C et al. Blood. 2019;133(5):436-45.

Best frontline options for advanced follicular lymphoma, Shah NN et al. Clin Lymphoma Myeloma Leuk. 2019 Feb;19(2):95-102.

Must Reads in Bleeding Disorders

A clot lysis contradiction with mild bleeding, Vries MJA et al. Thromb Res. 2018 Dec 4. doi: 10.1016/j.thromres.2018.12.004

Factor VIII inhibitors seen in some patient subgroups, not others, Gangadharan B et al. ASH 2018, Poster 3774

Could thrombin generation be a better biomarker?, Nossair F et al. ASH 2018, Poster 3788

Novoeight looks safe in hemophilia A extension trial, Lentz SR et al. Haemophilia. 2018 Nov;24(6):e391-e394

Durable responses for hemophilia A gene therapy, High K et al. ASH 2018, Abstract 487