Pharmiweb ChannelsAll | PharmaCo | Clinical Research | R&D/BioTech | Sales/Mktg | Healthcare | Recruitment | Pharmacy | Medical Comms RSS Feed RSS Feeds


Press Release


Shire plc
Posted on: 19 Dec 17


First and only FDA-cleared PK dosing software to support healthcare professionals in creating personalized dosing regimens for patients 16 and older with hemophilia A

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:
· Control and prevention of bleeding episodes.
· Perioperative management.
· Routine prophylaxis to prevent or reduce the frequency of bleeding episodes.

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.


Hypersensitivity Reactions
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
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.


  • Serious adverse reactions seen with ADVATE are hypersensitivity reactions, including anaphylaxis, and the development of high-titer inhibitors necessitating alternative treatments to factor VIII.
  • The most common adverse reactions observed in clinical trials (>5% of subjects) were pyrexia, headache, cough, nasopharyngitis, arthralgia, vomiting, upper respiratory tract infection, limb injury, nasal congestion, and diarrhea .

Please see accompanying ADVATE full Prescribing Information available at  

  For more information on ADVATE, please visit .  

For further information please contact:

Investor Relations   
Ian Karp +1 781 482 9018
Robert Coates +44 203 549 0874

Gwen Fisher +1 781 482 9649
Jaren Madden +1 617 584 6893


About Shire

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.

Forward-Looking Statements

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:

  • Shire's products may not be a commercial success;
  • increased pricing pressures and limits on patient access as a result of governmental regulations and market developments may affect Shire's future revenues, financial condition and results of operations;
  • Shire conducts its own manufacturing operations for certain of its products and is reliant on third party contract manufacturers to manufacture other products and to provide goods and services. Some of Shire's products or ingredients are only available from a single approved source for manufacture. Any disruption to the supply chain for any of Shire's products may result in Shire being unable to continue marketing or developing a product or may result in Shire being unable to do so on a commercially viable basis for some period of time;
  • the manufacture of Shire's products is subject to extensive oversight by various regulatory agencies. Regulatory approvals or interventions associated with changes to manufacturing sites, ingredients or manufacturing processes could lead to, among other things, significant delays, an increase in operating costs, lost product sales, an interruption of research activities or the delay of new product launches;
  • certain of Shire's therapies involve lengthy and complex processes, which may prevent Shire from timely responding to market forces and effectively managing its production capacity;
  • Shire has a portfolio of products in various stages of research and development. The successful development of these products is highly uncertain and requires significant expenditures and time, and there is no guarantee that these products will receive regulatory approval;
  • the actions of certain customers could affect Shire's ability to sell or market products profitably. Fluctuations in buying or distribution patterns by such customers can adversely affect Shire's revenues, financial conditions or results of operations;
  • Shire's products and product candidates face substantial competition in the product markets in which it operates, including competition from generics;
  • adverse outcomes in legal matters, tax audits and other disputes, including Shire's ability to enforce and defend patents and other intellectual property rights required for its business, could have a material adverse effect on the Company's revenues, financial condition or results of operations;
  • inability to successfully compete for highly qualified personnel from other companies and organizations;
  • failure to achieve the strategic objectives, including expected operating efficiencies, cost savings, revenue enhancements, synergies or other benefits at the time anticipated or at all with respect to Shire's acquisitions, including NPS Pharmaceuticals Inc., Dyax Corp. or Baxalta Incorporated may adversely affect Shire's financial condition and results of operations;
  • Shire's growth strategy depends in part upon its ability to expand its product portfolio through external collaborations, which, if unsuccessful, may adversely affect the development and sale of its products;
  • a slowdown of global economic growth, or economic instability of countries in which Shire does business, as well as changes in foreign currency exchange rates and interest rates, that adversely impact the availability and cost of credit and customer purchasing and payment patterns, including the collectability of customer accounts receivable;
  • failure of a marketed product to work effectively or if such a product is the cause of adverse side effects could result in damage to Shire's reputation, the withdrawal of the product and legal action against Shire;
  • investigations or enforcement action by regulatory authorities or law enforcement agencies relating to Shire's activities in the highly regulated markets in which it operates may result in significant legal costs and the payment of substantial compensation or fines;
  • Shire is dependent on information technology and its systems and infrastructure face certain risks, including from service disruptions, the loss of sensitive or confidential information, cyber-attacks and other security breaches or data leakages that could have a material adverse effect on Shire's revenues, financial condition or results of operations;
  • Shire incurred substantial additional indebtedness to finance the Baxalta acquisition, which has increased its borrowing costs may decrease its business flexibility; and

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.


This announcement is distributed by Nasdaq Corporate Solutions on behalf of Nasdaq Corporate Solutions clients.

The issuer of this announcement warrants that they are solely responsible for the content, accuracy and originality of the information contained therein.

Source: Shire plc via GlobeNewswire

Last updated on: 19/12/2017

Site Map | Privacy & Security | Cookies | Terms and Conditions is Europe's leading industry-sponsored portal for the Pharmaceutical sector, providing the latest jobs, news, features and events listings.
The information provided on is designed to support, not replace, the relationship that exists between a patient/site visitor and his/her physician.