New FDA-approved indications for ADYNOVATE provides more hemophilia A patients access to proven prophylaxis with a simple, twice-weekly dosing schedule
Lexington, Mass. – December 27, 2016 – Shire plc (LSE: SHP, NASDAQ: SHPG) announced today that the U.S. Food and Drug Administration (FDA) has approved ADYNOVATE [Antihemophilic Factor (Recombinant), PEGylated], an extended circulating half-life recombinant Factor VIII (rFVIII) treatment for hemophilia A, in pediatric patients under 12 years of age. The FDA also approved ADYNOVATE for use in surgical settings for both adult and pediatric patients. ADYNOVATE is built on the full-length ADVATE® [Antihemophilic Factor (Recombinant)] molecule, a market leading treatment for hemophilia A with more than 13 years of real-world patient experience.
“Physicians need additional treatment options for pediatric patients and those undergoing surgery with hemophilia A,” said lead investigator Eric Mullins, M.D., Research Director, Hemophilia Treatment Center, Cincinnati Children’s Hospital Medical Center. “As someone who has routinely seen the benefits of factor replacement in adult patients with hemophilia A, I am pleased to be able to provide ADYNOVATE to more patients.”
The approval of ADYNOVATE to treat children under the age of 12 was based on data from a prospective, uncontrolled, open-label, multi-center Phase 3 trial designed to assess the immunogenicity along with the safety and efficacy of ADYNOVATE. Results from the study showed that ADYNOVATE met its primary endpoint with no previously treated children having developed inhibitory antibodies to ADYNOVATE. In addition, no treatment-related serious adverse events were reported. More than 70 percent (73 percent) of children had zero joint bleeds (n=48/66) while on prophylactic treatment with ADYNOVATE and nearly 40 percent (38 percent) experienced zero bleeds (n= 25/66). The median overall annualized bleeding rate (ABR) among pediatric patients treated with ADYNOVATE was 2.0 (mean ABR 3.04; range 2.21–4.19), which was similar to the rates seen in the adult study.
“At Shire, we are dedicated to developing innovative therapies for patients living with hemophilia A,” said Philip J. Vickers, Ph.D., Global Head of Research and Development at Shire. “Today’s announcement of the new FDA-approved indications for ADYNOVATE in pediatric patients less than 12 years of age and those undergoing surgery underscores our commitment to provide treatment options with proven efficacy profiles to more patients living with hemophilia A.”
Approval to use ADYNOVATE in surgical settings for both adult and pediatric patients was based on interim results of an ongoing Phase 3 study of perioperative control of hemostasis among 15 patients with severe hemophilia A undergoing surgical procedures.
ADYNOVATE was first approved by the FDA in November 2015. ADYNOVATE provides proven prophylaxis with simple, twice-weekly dosing for children and adult patients with hemophilia A, helping to further individualize options for this patient population. The most common adverse reactions reported in >= 1% of subjects in the clinical studies were headache and nausea. Through a collaboration with Nektar Therapeutics (NASDAQ: NKTR), ADYNOVATE leverages proprietary PEGylation technology designed to extend the amount of FVIII available for use in the body. The technology was selected because it maintains the integrity of the parent molecule (ADVATE) while reducing the time at which the body clears ADYNOVATE, resulting in an extended circulating half-life.
ADYNOVATE is built on the full-length ADVATE molecule, a market-leading treatment for hemophilia A that has been used by patients worldwide for more than 13 years. ADYNOVATE was first approved by the FDA in November 2015. ADYNOVATE provides proven prophylaxis with simple, twice-weekly dosing for children and adult patients with hemophilia A, helping to further individualize options for this patient population. The PEGylation technology was selected because it maintains the integrity of the parent molecule (ADVATE) and reduces the time at which the body clears ADYNOVATE, resulting in an extended circulating half-life.
ADYNOVATE [Antihemophilic Factor (Recombinant), PEGylated] Important Information
ADYNOVATE, Antihemophilic Factor (Recombinant), PEGylated, is a human antihemophilic factor indicated in children and adults with hemophilia A (congenital factor VIII deficiency) for:
Limitation of Use
ADYNOVATE is not indicated for the treatment of von Willebrand disease.
ADYNOVATE [Antihemophilic Factor (Recombinant), PEGylated] DETAILED IMPORTANT RISK INFORMATION
ADYNOVATE is contraindicated in patients who have had prior anaphylactic reaction to ADYNOVATE, to the parent molecule (ADVATE [Antihemophilic Factor (Recombinant)]), mouse or hamster protein, or excipients of ADYNOVATE (e.g. Tris, mannitol, trehalose, glutathione, and/or polysorbate 80).
WARNINGS & PRECAUTIONS
Hypersensitivity reactions are possible with ADYNOVATE. Allergic-type hypersensitivity reactions, including anaphylaxis, have been reported with other recombinant antihemophilic factor VIII products, including the parent molecule, ADVATE. Early signs of hypersensitivity reactions that can progress to anaphylaxis may include angioedema, chest tightness, dyspnea, wheezing, urticaria, and pruritus. Immediately discontinue administration and initiate appropriate treatment if hypersensitivity reactions occur.
Formation of neutralizing antibodies (inhibitors) to factor VIII can occur following administration of ADYNOVATE. Monitor patients regularly for the development of factor VIII inhibitors by appropriate clinical observations and laboratory tests. Perform an assay that measures factor VIII inhibitor concentration if the plasma factor VIII level fails to increase as expected, or if bleeding is not controlled with expected dose.
The most common adverse reactions (≥1% of subjects) reported in the clinical studies were headache and nausea.
For ADYNOVATE Full Prescribing Information, visit http://www.shirecontent.com/PI/PDFs/ADYNOVATE_USA_ENG.pdf
ADVATE [Antihemophilic Factor (Recombinant)] Important Information
ADVATE [Antihemophilic Factor (Recombinant)] 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.
ADVATE [Antihemophilic Factor (Recombinant)] DETAILED IMPORTANT RISK INFORMATION
ADVATE is contraindicated in patients who have life-threatening hypersensitivity reactions, including anaphylaxis, to mouse or hamster protein or other constituents of the product.
WARNINGS & PRECAUTIONS
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.
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 (frequency ≥5% of subjects) were pyrexia, headache, cough, nasopharyngitis, arthralgia, vomiting, upper respiratory tract infection, limb injury, nasal congestion, and diarrhea.
For ADVATE Full Prescribing Information, visit http://www.shirecontent.com/PI/PDFs/ADVATE_USA_ENG.pdf
About Hemophilia A
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. Approximately 25-30% of individuals with severe hemophilia A develop inhibitors. Inhibitors are a rare, but a serious medical problem that can occur when a person with hemophilia has an immune response to treatment with clotting factor concentrates. Hemophilia primarily affects males, with an incidence of one in 5,000 male births.
SHIRE and the Shire Logo are registered trademarks of Shire Pharmaceutical Holdings Ireland Limited or its affiliates. ADYNOVATE and ADVATE are registered trademarks of Baxalta Incorporated, a wholly owned, indirect subsidiary of Shire plc.
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