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
Baxalta announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency expressed a positive opinion for the marketing authorization of Obizur [Antihaemophilic Factor (Recombinant), Porcine Sequence]. Baxalta requested authorization for the treatment of bleeding episodes in adult patients with acquired haemophilia6 caused by antibodies to Factor VIII (FVIII).The European Commission has yet to announce its decision on the recommendation7. John Orloff, head of Research & Development and chief scientific officer, Baxalta, said: “The marketing authorization anticipated later this year for Obizur will be an important milestone, offering patients with acquired haemophilia A in Europe a treatment option that transforms their care by allowing physicians to monitor treatment response”. The CHMP positive opinion follows a global, prospective, controlled, multi-centre Phase II/III open-label clinical trial testing the efficacy of Obizur in treating serious bleeding episodes in adults with acquired haemophilia A. All 28 patients treated with Obizur had their bleeding stopped or decreased. Adverse reactions were development of inhibitors to porcine FVIII. Obizur is already approved in the US and is being reviewed by regulators in Australia, Canada, Switzerland, and Colombia.
The FDA accepted for review CSL Behring's Biologics License Application (BLA) for its recombinant factor VIII single-chain (rVIII SingleChain or CSL 627), designed for molecular stability in treating haemophilia A. The product met all primary endpoints in the pivotal trial. The BLA relies on the AFFINITY clinical development program, including a phase I/III open-label, multi-centre trial examining safety and efficacy. Pharmacokinetics were compared with a current standard of care, recombinant human anti-haemophilic factor VIII (octocog alfa). CSL627 has a strong affinity for von Willebrand factor, leading to greater stability and integrity of FVIII in circulation.
Swissmedic accepted for review a Marketing Authorization Application for CSL Behring’s long-acting fusion protein linking recombinant coagulation factor IX with recombinant albumin (rIX-FP) for the prophylaxis and treatment of bleeding episodes in haemophilia B. This rIX-FP allows dosing intervals up to 14 days.
Baxalta announced in the US that the Centers for Medicare and Medicaid Services (CMS) have expanded coverage to include in-home use of Hyqvia [Immune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase], a treatment for adults with primary immunodeficiency (PI). After the FDA approved Hyqvia in 2014, CMS covered both provider facility and in-office treatment with Hyqvia. This new decision expands its provisions to include durable medical equipment coverage of the infusion pump required to administer Hyqvia, facilitating the self-administration (at home) option for patients. In the clinical trial for Hyqvia, the majority of PI patients expressed a desire for the ability to administer infusions at home.
ADMA Biologics has submitted its Biologics License Application to the FDA seeking marketing authorization for RI-002. This is a plasma-derived, polyclonal, intravenous immune globulin derived from human plasma containing naturally occurring polyclonal antibodies (Streptococcus pneumoniae, H.influenza type B, cytomegalovirus (CMV), measles, tetanus, etc.) as well as standardized, high levels of antibodies to respiratory syncytial virus (RSV)8. In a Phase III study in patients with primary immunodeficiency, RI-002 met its primary endpoint of no serious bacterial infections (SBI). The requirement specified by FDA guidance is ≤ 1 SBI per patient-year.
Other
FDA officials have met with Google to “begin a discussion on how we might collaborate with Google on identifying adverse event data, using Google’s technologies and data.”9
The FDA granted Fresenius Kabi approval for a labelling change that permits blood centres to use the Fenwal Amicus system for the storage of Amicus-derived platelets in plasma for up to seven days. Where blood centres do store apheresis-derived platelets for seven days they must label each product with a statement that the product must be tested with a bacterial detection device cleared by FDA and labelled as a “safety measure”. Fresenius Kabi is the sole distributor of the Verax Platelet PGD test, a rapid test approved by the FDA as a “safety measure” for leuko reduced apheresis platelets within 24 hours prior to transfusion.
Hospira received approval from the FDA to launch bivalirudin for injection, a generic version of The Medicines Company's Angiomax. Bivalirudin is a direct thrombin inhibitor indicated amongst other things for use as an anticoagulant in patients with unstable angina undergoing percutaneous transluminal coronary angioplasty.
The FDA, the US Department of Homeland Security’s Industrial Control Systems Cyber Emergency Response Team, and Hospira announced that Hospira’s Symbiq Infusion System could be accessed remotely through a hospital’s network. An unauthorized user could control the device and change the dosage the pump delivers. While neither the FDA nor Hospira are aware of any unauthorized access of a Symbiq Infusion System in a US health care setting, the FDA urged health care facilities to discontinue use of these pumps.
Remedium Technologies has FDA 510(k) clearance to market its Hemogrip patch. The patch controls bleeding occurring from access to veins or arteries during surgery. The product is based on chitosan, from the exoskeleton of crustaceans. Based at the University of Maryland, Remedium10 has a number of other bleeding control products in development, including a clear transparent film, haemostatic gauze and a foam spray.
