Baxalta Announces Submission of Supplemental BLAs to Expand Use of ADYNOVATE to Pediatric Patients and Surgical Settings

Baxalta Incorporated (NYSE: BXLT), a global biopharmaceutical leaderdedicated to delivering transformative therapies to patients with orphandiseases and underserved conditions, announced today that it has submittedsupplemental Biologics License Applications (sBLAs) to the U.S. Food andDrug Administration (FDA) seeking approval for the use of ADYNOVATE[Antihemophilic Factor (Recombinant), PEGylated] to treat children under theage of 12 with hemophilia A and for use in surgical settings. ADYNOVATE is the only extended circulating half-life recombinant Factor VIII(rFVIII) treatment for hemophilia A based on the demonstrated efficacy ofADVATE [Antihemophilic Factor (Recombinant)]. ADYNOVATE was approved by theFDA in November 2015 for use in adolescent and adult hemophilia A patients(12 years and older) for prophylaxis to reduce the frequency of bleedingepisodes and on-demand treatment and control of bleeding. "We know there is a great need for innovative new treatments among pediatricpatients and during surgery and we look forward to working with the FDA asthey review ADYNOVATE for these patients," said Brian Goff, executive vicepresident and president, Hematology. "We are committed to continuallyadvancing direct factor replacement treatments and specifically to expandinguse of ADYNOVATE, providing as many patients as possible access to itsproven prophylaxis and simple, twice-weekly dosing schedule." The submission of ADYNOVATE to treat children under the age of 12 was basedon results of a Phase 3 trial designed to assess the efficacy and safetyincluding immunogenicity of ADYNOVATE, which was initially reported inDecember 2015. Results from the study showed ADYNOVATE met its primaryendpoint and no patients developed inhibitory antibodies to ADYNOVATE. Inaddition, no treatment-related serious adverse events were reported. Morethan 70 percent (72.7 percent) of patients had no joint bleeds while ontreatment with ADYNOVATE (n=66) and nearly 40 percent (37.9 percent)experienced zero bleeds. The median overall annualized bleeding rate (ABR)among patient participants treated with ADYNOVATE was 2.0 (range 0-49.8;mean ABR 3.0), which was comparable to the rates seen in the adult study. The filing was also supported by the positive results of a Phase 3 studyevaluating the efficacy and safety of ADYNOVATE for the perioperativecontrol of hemostasis among 15 patients with severe hemophilia A undergoingsurgical procedures, which was reported in December 2015. The study datademonstrated that ADYNOVATE achieved hemostasis control in the perioperativeperiod (from start of the procedure until discharge or day 14) for patientswith severe hemophilia A. Baxalta continues to invest in ADYNOVATE to expand the product's value formore patients worldwide. Baxalta plans to file for marketing authorizationin Europe in the first quarter of 2016 and expects regulatory approval ofthe treatment in Japan in the first half of the year. ADYNOVATE is alsounder regulatory review in Canada and Switzerland. ADYNOVATE is built on the full-length ADVATE molecule, a leading treatmentfor hemophilia A that been used by patients worldwide for more than 12years. Through a collaboration with Nektar Therapeutics (NASDAQ: NKTR),ADYNOVATE leverages proprietary PEGylation technology designed to extend theamount of FVIII available for use in the body. The technology was selectedbecause it maintains the integrity of the parent molecule (ADVATE) andreduces the time at which the body clears ADYNOVATE, resulting in anextended circulating half-life. This proprietary technology has been usedfor more than 15 years in a number of approved medicines that treat chronicor serious conditions.