Monitoring International Trends posted January 2015

Regulatory

The NBA monitors overseas regulatory decisions on products, processes or procedures which are or may be of relevance to its responsibilities.

Plasma and recombinant products

1. 
   In mid-December Bayer filed with the FDA a biologics licence application for BAY 81-8973, a compound for the treatment of haemophilia A in adults and children. BAY 81-8973 is a full-length recombinant factor VIII (rFVIII) which has demonstrated efficacy when used in standard dosage for prophylaxis two times or three times per week. This application follows Bayer’s recent submission to the European Medicines Agency (EMA) for approval of the same indication in the European Union.
   
2. 
   CSL Behring submitted a biologics license application to the FDA for its long-acting fusion protein linking recombinant coagulation factor IX with recombinant albumin (rIX-FP). If approved by the FDA, rIX-FP will provide patients with haemophilia B a long-acting treatment option with dosing intervals up to 14 days.
   
3. 
   Baxter and development partner Nektar Therapeutics announced early in January that a biologics license application for an investigational, extended half-life recombinant factor VIII treatment for haemophilia A had been submitted to the FDA. This submission for BAX 855 was based on positive results of a Phase III study. In the trial, 137 previously treated patients aged 12 years or more either experienced a twice weekly prophylaxis or an on demand treatment. In the twice weekly prophylaxis group, patients experienced a 95 per cent reduction in median annualized bleed rate compared with the on demand treatment group, 1.9 vs. 41.5, respectively. BAX 855 also treated bleeding episodes, with 96 per cent controlled with 1 or 2 infusions. The researchers reported no inhibitor development in patients after treatment and no serious adverse events such as hypersensitivity. The most common adverse reaction was headache. The trial is ongoing. The drug will also be trialled in paediatric patients with severe haemophilia.
   
4. 
   The FDA approved Octapharma’s manufacturing facility in Vienna for the production of Octagam 10% [Immune Globulin Intravenous (Human) 10% (100 mg/mL) Liquid Preparation], which became available in the US during October 2014. Octagam 10% for the US market can now be manufactured at FDA-licensed facilities in Stockholm, Sweden and Vienna.
   
5. 
   Baxter has filed a biologics license application with the FDA for BAX 111, an investigational drug for the treatment of patients with von Willebrand disease (VWD), the most common type of inherited bleeding disorder¹¹. If approved, BAX 111 will be the first recombinant treatment in clinical development for VWD. The filing was based on results a Phase III, multi-center clinical trial assessing the safety, efficacy and pharmacokinetics of BAX111. Trial sites were located in the US, Europe, Australia, Japan, Russia and India. BAX 111 was granted an orphan-drug designation by the FDA and the European Commission in November 2010.
   

Blood donation, processing, storage and use; blood substitutes

1. 
   In a letter dated 16 December 2014, the FDA approved Cerus’ Intercept Blood System for plasma. This system is used by blood establishments in the preparation of plasma in order to reduce the risk of transfusion-transmitted infections (TTI), including HIV, hepatitis B and C and West Nile Virus. The photochemical process involves a controlled exposure to ultraviolet light and amotosalen, a chemical that facilitates the inactivation process. The plasma is then purified to remove the chemical and its byproducts. Some viruses (e.g. human parvovirus B19) and spores formed by certain bacteria are resistant to the Intercept process.
   
2. 
   In a letter dated 18 December 2014, the FDA approved Cerus’ Intercept Blood System for platelets. It noted that “this device is intended to be used for ex vivo preparation of apheresis platelet components in order to reduce the risk of transfusion-transmitted infection (TTI) including sepsis, and to potentially reduce the risk of transfusion-associated graft versus host disease (TA-GVHD).”
   
3. 
   The FDA approved Roche’s cobas TaqScreen MPX Test, v2.0, to detect and identify HIV1, HIV2, and hepatitis B and C in the plasma of donated blood, organs, and tissues.
   
4. 
   At the end of December, the FDA issued draft guidance for industry on Bacterial Detection Testing by Blood Collection Establishments and Transfusion Services to Enhance the Safety and Availability of Platelets for Transfusion. Comment was invited.
   

Other

1. 
   The FDA has approved a test to screen for Severe Combined Immunodeficiency (SCID) in newborns. It has recommended all States screen newborns for SCID along with other disorders.
   
2. 
   In early January, Cohera Medical of Pittsburgh received a letter from the FDA saying its premarket approval application for TissuGlu Surgical Adhesive is approvable (the company then works with the FDA to achieve final approval). TissuGlu is a strong, biocompatible, and easy-to-use surgical adhesive. It is indicated for the approximation of tissue layers where subcutaneous dead space exists between tissue planes in abdominoplasty. It is an alternative to the use of closed suction drains, reducing the number of post-operative invasive treatments and improving the patient recovery process. TissuGlu has been CE approved and for sale in Germany through a Cohera direct sales force since 2012 and has been used in over 2,000 surgical procedures since that time.
   
3. 
   Hospira submitted a biologics license application to the FDA for its anaemia drug Retacrit, a biosimilar. Retacrit received approval in Europe in 2008.
   
4. 
   The FDA approved Daiichi-Sankyo's Savaysa (edoxaban tablets) to reduce the risk of stroke and dangerous blood clots (systemic embolism) in patients with atrial fibrillation that is not caused by a heart valve problem. The anticoagulant edoxaban inhibits factor Xa. It was approved in Japan in 2011 for the prevention of venous thromboembolism in patients undergoing total knee replacement surgery, total hip replacement surgery and hip fracture surgery.
   
5. 
   The UK’s National Institute for Health and Care Excellence (NICE) recommended Boehringer Ingelheim's dabigatran, or Pradaxa, for National Health Service patients in England and Wales, for the treatment and prevention of deep vein thrombosis and pulmonary embolism.
   
6. 
   GSK announced the submission of a supplemental new drug application to the FDA for eltrombopag (Promacta¹²), seeking an additional indication in paediatric patients six years and older with chronic immune (idiopathic) thrombocytopenia (ITP)¹³ who have had an insufficient response to corticosteroids, immunoglobulins or splenectomy.
   

3.