FDA approves Baxalta Haemophilia A treatment
pharmafile | November 16, 2015 | News story | Manufacturing and Production, Research and Development, Sales and Marketing |
The US FDA has approved Baxalta’s twice-weekly therapy Adynovate for use in adults and adolescents, aged 12 years plus, with Haemophilia A.
The Baxter spin-off’s Antihemophilic Factor and PEGylated drug is modified to last longer in the blood and potentially require less frequent injections than unmodified Antihemophilic Factor when used to reduce the frequency of bleeding.
Adynovate is approved for on-demand (as needed) treatment and control of bleeding episodes and to reduce the frequency of bleeding episodes (prophylaxis) in Haemophilia A patients.
The drug consists of the full-length Coagulation Factor VIII molecule (historically known as Antihemophilic Factor) linked to other molecules, known as polyethylene glycol (PEGylated). This link between the molecules is believed to enable to longer-lasting action in patients’ blood.
“The approval of Adynovate provides an important therapeutic option for use in the care of patients with Haemophilia A and reduces the frequency of Factor VIII infusions needed to avoid bleeding,” said Karen Midthun, director of the FDA’s Center for Biologics Evaluation and Research.
“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.”
Haemophilia A is an inherited, sex-linked, blood-clotting disorder that primarily affects males, which is caused by defects found in the Factor VIII gene. The Centers for Disease Control and Prevention says Haemophilia A affects one in every 5,000 male births in the US.
Patients with hemophilia A may experience repeated episodes of serious bleeding, primarily into the joints, which can be severely damaged as a result.
The safety and efficacy of Adynovate were evaluated in a clinical trial of 137 adults and adolescents aged 12 years and older, which compared the recommended routine prophylactic (preventative) treatment regimen to on-demand therapy.
The trial demonstrated that Adynovate was effective in reducing the number of bleeding episodes during routine care and also that it was effective in treating and controlling bleeding episodes. No safety concerns were identified during the trial, says the FDA.
Baxalta said the treatment would become available in the US “in the coming weeks.” The company has already submitted a request for regulatory approval of Adynovate in Japan and expects to file for marketing authorisation in Europe following completion of the paediatric study.
Baxalta developed Adynovate in collaboration with Nektar Therapeutics.
Joel Levy
