Baxalta Incorporated, a global biopharmaceutical leader, announced that the US Food and Drug Administration (FDA) has approved Adynovate [Antihemophilic Factor (Recombinant), PEGylated], an extended circulating half-life recombinant Factor VIII (rFVIII) treatment for haemophilia A. Adynovate is built on the full-length ADVATE [Antihemophilic Factor (Recombinant)] molecule, a leading treatment for haemophilia A with more than 12 years of real-world patient experience.

“People with hemophilia want to minimise their bleeds and not let their haemophilia dictate their lives; having options empowers patients to make the best decisions for their personal care,” said Val Bias, chief executive officer, National Hemophilia Foundation. “We applaud Baxalta for continuing to innovate for the care of haemophilia.”

“Adynovate represents Baxalta’s commitment to meeting the needs of haemophilia A patients who want bleed reduction with a dosing schedule that better fits their personal needs,” said Ludwig Hantson, chief executive officer and president, Baxalta. “As the first new product approved under the Baxalta name, Adynovate represents a major milestone in achieving our goal of 20 product launches by 2020 and continuing to expand our world-leading haemophilia treatment portfolio.”

In the pivotal phase 3 clinical trial, which served as the foundation for the approval, Adynovate demonstrated efficacy in treating haemophilia patients through routine prophylaxis as well as for on-demand bleeding episodes. Patients, 12 to 65 years of age, in the prospective, global, multi-center, open label, non-randomized study were assigned to either twice weekly prophylaxis (40-50 IU/kg, n=120) or on-demand treatment (10-60 IU/kg, n=17) with Adynovate. The study found that previously-treated patients in a twice-weekly prophylaxis arm had 95 per cent fewer annual bleeds compared to those treated on-demand [median annual bleed rate (ABR) 1.9 vs. 41.5, respectively]. During the study, 38 per cent (n=120) of prophylaxis-treated patients experienced zero bleeds. Moreover, 57 per cent of patients experienced zero joint bleeds based on six months of prophylaxis with Adynovate.

Nearly all (98 per cent) of patients on prophylaxis with Adynovate did not have a dose adjustment in the study. Nearly all (96 per cent) bleeding episodes (n=591) were controlled with one or two infusions of Adynovate. No patients developed inhibitors to the treatment; the most common adverse reactions (=1 per cent of subjects) were headache and nausea.

Adynovate will be available in the United States in the coming weeks. Baxalta continues to invest in Adynovate to expand the product’s value for more patients worldwide. Currently, studies are ongoing in previously treated patients (PTPs) with severe hemophilia A undergoing surgery and in paediatric PTPs under the age of 12 with severe haemophilia A. Additionally, Baxalta will initiate a study in previously-untreated patients (PUPs) with severe haemophilia A. Baxalta has filed for regulatory approval of the treatment in Japan and following completion of the paediatric study, expects to file for marketing authorisation in Europe.

Haemophilia A is a challenging chronic disease; treatment regimens require regular infusions to reduce the risk of bleeding. Working closely with their health professionals, many patients continue to seek treatment options that can be better personalised to fit their needs, providing both effective bleed protection and simplified dosing schedules. Today, the disease affects approximately 16,000 people in the United States and more than 400,000 people worldwide. With an estimated 75 per cent of people with haemophilia undiagnosed and untreated or undertreated globally, Baxalta continues to innovate in order to address some of the greatest challenges associated with hematologic disorders, including haemophilia.

Through a collaboration with Nektar Therapeutics, Adynovate leverages proprietary pegylation technology designed to prolong the amount of FVIII available for use in the body. The technology was selected because it maintains the integrity of the parent molecule (ADVATE) and reduces the speed at which the body clears Adynovate, resulting in increased circulating half-life. This proprietary technology has been used for more than 15 years in a number of approved medicines that treat chronic or serious conditions. In addition to the patents relating to pegylated FVIII, Baxalta has an exclusive license in the field of haemophilia to certain other patents relating to pegylated FVIII proteins. These patents protect Baxalta’s pipeline of extended circulating half-life FVIII products, including Adynovate.