The 510(k) marketing clearance was granted to Shire, the developer, following its submission of a “premarket notification,” which is necessary if a new device is intended for commercial distribution, or if an already existing device will be significantly modified so that its safety or effectiveness could be affected.
The software, called myPKFiT, will assess prophylactic (preventive) dosing regimens for hemophilia A patients treated with ADVATE, a recombinant factor VIII (FVIII) product. Shire plans availability of myPKFIT for this spring.
myPKFIT is the first and only FDA-cleared software helping healthcare professionals estimate key pharmacological data of ADVATE-treated hemophilia A patients age 16 and older and weighing at least 45 kilograms.
The new tool enables estimation of a patient’s individual pharmacokinetic (PK) profile (drug response in the body) with as few as two measurable blood samples; that is compared with the current nine to 11 as recommended by the International Society on Thrombosis and Haemostasis (ISTH).
Using PK data and additional patient information (age, body weight, and FVIII clotting activity measurements), healthcare professionals will be able to evaluate various prophylaxis regimens to create a personalized ADVATE treatment dose and schedule to maintain appropriate FVIII activity levels.
“The FDA clearance of myPKFiT for ADVATE marks an important milestone in the personalization of hemophilia care, building on Shire’s strong commitment to continued innovation in hematology,” Howard B. Mayer, MD, ad-interim global head of research and development at Shire, said in a press release.
“We know patients have complex needs and treatment goals that cannot be met with a one-size-fits-all approach,” said Michael Denne, head of U.S. Hematology Medical Affairs at Shire. “myPKFiT for ADVATE offers a personalized approach to hemophilia care that allows healthcare professionals to consider their patients’ individual needs and to educate them on their personal PK profiles.”
Hemophilia primarily affects males, with an incidence of one in 5,000 male births in the U.S. Hemophilia A is the most common type of the condition. Lower amounts of VIII in the blood are associated with aggravated disease severity. Of note, more than half of hemophilia A patients have the severe form of the disease.
ADVATE is currently used for the control and prevention of bleeding episodes, and for preoperative management of children and adults with hemophilia A. The treatment is currently approved in 69 countries worldwide, including the U.S., Canada, and 28 countries in the European Union.