First and only FDA-cleared PK dosing software to support healthcare professionals in creating personalized dosing regimens for patients 16 and older with hemophilia A1
myPKFiT for ADVATE software availability planned for end of Q1 2018
Cambridge, Ma. – December 19, 2017 – Shire plc (LSE: SHP, NASDAQ: SHPG), the global biotechnology leader in rare diseases, today announced the U.S. Food and Drug Administration (FDA) has granted 510(k) marketing clearance to myPKFiT for ADVATE [Antihemophilic Factor (Recombinant)], a free web-based software that is the first and only FDA-cleared pharmacokinetic (PK) dosing software for hemophilia A patients 16 and older weighing at least 45 kilograms treated with ADVATE. Using as few as two measurable blood samples, myPKFiT for ADVATE generates a patient’s estimated individual PK profile to aid healthcare professionals in personalizing a patient’s prophylaxis ADVATE dose and schedule.
The myPKFiT for ADVATE software represents an innovative approach to estimating a patient’s PK curve, a key measure for assessing drug exposure over time. With myPKFiT for ADVATE, healthcare professionals can estimate a full PK curve with as few as two measurable blood samples, compared to 9 to 11 as recommended by guidelines from the International Society on Thrombosis and Haemostasis (ISTH). Using the patient’s PK curve and additional patient information, healthcare professionals can develop a personalized, PK-guided prophylactic ADVATE treatment regimen tailored to the individual patient’s needs.
“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,” said Howard B. Mayer, M.D., ad-interim Global Head of Research and Development, Shire.
Hemophilia A, the most common type of hemophilia, is a rare bleeding disorder that causes longer-than-normal bleeding due to lack of clotting factor VIII in the blood. The severity of hemophilia A is determined by the amount of factor in the blood, with more severity associated with lower amounts of factor. More than half of patients with hemophilia A have the severe form of the condition. Hemophilia primarily affects males, with an incidence of one in 5,000 male births in the United States.
“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, 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.”
A version of the myPKFiT for ADVATE software has been CE marked in Europe since 2014.
myPKFiT for ADVATE software is Rx Only. For safe and proper use of the myPKFiT for ADVATE software, please refer to the complete instructions for use in the User Manual when the software is available in the United States, projected to be by the end of Q1 2018.
myPKFiT for ADVATE Indications for Use
The myPKFiT for ADVATE software is intended for use by licensed healthcare professionals (HCPs) who are familiar with hemophilia care. myPKFiT for ADVATE can be used to generate ADVATE dosage and frequency recommendations for routine prophylaxis for an individual patient 16 years of age or older and body weight of 45kg or greater, using that patient’s age and body weight information and local laboratory FVIII one-stage clotting activity measurements of sparse samples collected from that patient. A minimum of two sparse sampling points are required at the recommended 3-4 hours (± 30 minutes) and at 24-32 hours (± 1 hour) post-infusion.
HCPs will also be able to evaluate various prophylaxis dose regimens tailored to an individual patient’s needs and treatment plan. The software output may be used to guide decisions on appropriate ADVATE dose and infusion intervals to maintain FVIII activity levels at or above a user-specified minimum FVIII activity level between 1% and 3% above natural baseline for an individual patient in accordance with FDA approved dosing recommendations provided in the ADVATE Prescribing Information (PI).
myPKFiT for ADVATE should only be used to evaluate prophylactic dosing regiments for hemophilia A patients treated with ADVATE, as per the ADVATE PI. myPKFiT for ADVATE is not indicated for the treatment of von Willebrand disease. myPKFiT for ADVATE should not be used for patients who have developed neutralizing antibody (inhibitor) to FVIII products.
ADVATE is a full-length (derived from the complete FVIII gene) recombinant FVIII product that is processed without any blood-based additives.
ADVATE is currently approved in 69 countries worldwide, including the United States, Canada, 28 countries in the European Union, Algeria, Argentina, Australia, Brazil, Brunei, Chile, China, Colombia, Ecuador, Hong Kong, Iceland, India, Iraq, Israel, Japan, Kuwait, Macau, Malaysia, Mexico, Morocco, New Zealand, Norway, Panama, Puerto Rico, Qatar, Russia, Saudi Arabia, Serbia, Singapore, South Korea, Suriname, Switzerland, Taiwan, Tunisia, Turkey, Ukraine, Uruguay, and Venezuela.
ADVATE [Antihemophilic Factor (Recombinant)] Important Information
ADVATE is a recombinant antihemophilic factor indicated for use in children and adults with hemophilia A (congenital factor VIII deficiency) for:
ADVATE is not indicated for the treatment of von Willebrand disease.
Patients who have life-threatening hypersensitivity reactions, including anaphylaxis, to mouse or hamster protein or other constituents of the product.
Allergic-type hypersensitivity reactions, including anaphylaxis, have been reported with ADVATE. Symptoms include dizziness, paresthesia, rash, flushing, facial swelling, urticaria, dyspnea, pruritus, and vomiting. Discontinue ADVATE if hypersensitivity symptoms occur and administer appropriate emergency treatment.
Neutralizing antibodies (inhibitors) have been reported following administration of ADVATE predominantly in previously untreated patients (PUPs) and previously minimally treated patients (MTPs). Monitor all patients for the development of factor VIII inhibitors by appropriate clinical observation and laboratory testing. If expected plasma factor VIII activity levels are not attained, or if bleeding is not controlled with an expected dose, perform an assay that measures factor VIII inhibitor concentration.
Please see accompanying ADVATE full Prescribing Information available at http://www.shirecontent.com/PI/PDFs/ADVATE_USA_ENG.pdf
For more information on ADVATE, please visit www.ADVATE.com.
|Ian Karpfirstname.lastname@example.org||+1 781 482 9018|
|Robert Coatesemail@example.com||+44 203 549 0874|
|Gwen Fisherfirstname.lastname@example.org||+1 781 482 9649|
|Jaren Madden||Jaren.email@example.com||+1 617 584 6893|
Shire is the global leader in serving patients with rare diseases. We strive to develop best-in-class therapies across a core of rare disease areas including hematology, immunology, genetic diseases, neuroscience, and internal medicine with growing therapeutic areas in ophthalmics and oncology. Our diversified capabilities enable us to reach patients in more than 100 countries who are struggling to live their lives to the fullest.
We feel a strong sense of urgency to address unmet medical needs and work tirelessly to improve people’s lives with medicines that have a meaningful impact on patients and all who support them on their journey.
Statements included herein that are not historical facts, including without limitation statements concerning future strategy, plans, objectives, expectations and intentions, the anticipated timing of clinical trials and approvals for, and the commercial potential of, inline or pipeline products, are forward-looking statements. Such forward-looking statements involve a number of risks and uncertainties and are subject to change at any time. In the event such risks or uncertainties materialize, Shire’s results could be materially adversely affected. The risks and uncertainties include, but are not limited to, the following:
a further list and description of risks, uncertainties and other matters can be found in Shire’s most recent Annual Report on Form 10-K and in Shire’s subsequent Quarterly Reports on Form 10-Q, in each case including those risks outlined in “ITEM 1A: Risk Factors”, and in Shire’s subsequent reports on Form 8-K and other Securities and Exchange Commission filings, all of which are available on Shire’s website.
All forward-looking statements attributable to us or any person acting on our behalf are expressly qualified in their entirety by this cautionary statement. Readers are cautioned not to place undue reliance on these forward-looking statements that speak only as of the date hereof. Except to the extent otherwise required by applicable law, we do not undertake any obligation to update or revise forward-looking statements, whether as a result of new information, future events or otherwise.