CMS Grants New Technology Add-On Payment to Defitelio for the Treatment of Hepatic Veno-Occlusive Disease with Renal or Pulmonary Dysfunction Following Hematopoietic Stem-Cell Transplantation

Add-On Payment to Provide Additional Medicare Reimbursement for Defitelio and to Support Patient Access in the Inpatient PPS Hospital Setting

News provided by

Jazz Pharmaceuticals plc

Aug 02, 2016, 07:15 ET

DUBLIN, Aug. 2, 2016 /PRNewswire/ -- Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced that the United States (U.S.) Centers for Medicare and Medicaid Services (CMS) has granted a New Technology Add-on Payment (NTAP) for Defitelio® (defibrotide sodium), which is indicated for the treatment of adult and pediatric patients with hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), with renal or pulmonary dysfunction following hematopoietic stem-cell transplantation (HSCT). NTAP will provide incremental reimbursement to the standard diagnosis-related group (DRG)-based reimbursement for Defitelio to hospitals paid under the Medicare Hospital Inpatient Prospective Payment System (PPS) and will support Medicare beneficiaries' access to Defitelio when they are treated in the PPS inpatient hospital setting.

In the final rule concerning Hospital Inpatient PPS and CMS Fiscal Year (FY) 2017, (scheduled for publication in the Federal Register on August 22, 2016), CMS states that: "Because Defitelio is the only FDA-approved treatment for VOD with multi-organ failure, it represents a substantial clinical improvement for patients afflicted with this disease, whose alternatives include supportive care agents that have not demonstrated improved survival or complete response rates. After consideration of the public comments we received, we have determined that Defitelio meets all of the criteria for approval of new technology add-on payments. Therefore, we are approving new technology add-on payments for Defitelio for FY 2017."

Additional information on the CMS final rule and its discussion of NTAP and Defitelio can be found on pages 584 – 603 of the pre-publication Federal Register (https://www.federalregister.gov/articles/2016/08/22/2016-18476/medicare-programs-hospital-inpatient-prospective-payment-systems-for-acute-care-hospitals-etc).

"CMS's decision to grant NTAP designation for Defitelio recognizes the significant therapeutic advance of this first-of-its kind treatment for children and adults with VOD with renal or pulmonary dysfunction following stem-cell transplantation. The NTAP designation also underscores the clinical value of Defitelio, which offers a potentially curative intervention for patients with VOD following HSCT, which may save lives with a single course of therapy; before Defitelio, patients had no options for this rare and often deadly complication of stem-cell transplantation," said Mike Miller, senior vice president and U.S. commercial lead at Jazz Pharmaceuticals. "Importantly, the NTAP payment will ultimately help improve Medicare beneficiaries' access to Defitelio in the inpatient PPS hospital setting."

Effective for the Fiscal Year 2017 (October 1, 2016), the NTAP payment for Defitelio has been established at $75,900 and represents the maximum allowable payment under NTAP, or 50% of the wholesale acquisition cost for a 21-day course of Defitelio for an average 70kg patient. NTAP payment is expected to remain in effect for a period of two to three years until the cost of Defitelio is included in the recalibration of the DRG payment rates.

Introduced in 2001, NTAP was created by Congress to help facilitate access to new, innovative technologies used to treat Medicare beneficiaries in the hospital inpatient setting. For a new technology to qualify for NTAP, it must meet the NTAP definition of newness and demonstrate a substantial clinical improvement relative to existing therapies already included in the DRG payment rates and meet specific cost thresholds.

About Defitelio

Defitelio® (defibrotide sodium) injection 80mg/mL is indicated for the treatment of adult and pediatric patients with hepatic VOD, also known as SOS, with renal or pulmonary dysfunction following HSCT.¹ Defitelio received U.S. Food and Drug Administration (FDA) marketing approval on March 30, 2016 for the treatment of adult and pediatric patients with hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), with renal or pulmonary dysfunction following hematopoietic stem-cell transplantation (HSCT) and is the first and only FDA-approved therapy for patients with this rare, potentially fatal complication.

Important Safety Information¹

Defitelio should not be given to patients who are:

Currently taking anticoagulants or fibrinolytics
Allergic to Defitelio or any of its ingredients

Defitelio may increase the risk of bleeding in patients with VOD and should not be given to patients with active bleeding. During treatment with Defitelio, patients should be monitored for signs of bleeding. In the event that bleeding occurs during treatment with Defitelio, treatment should be temporarily or permanently stopped. Patients should tell the doctor right away about any signs or symptoms of hemorrhage such as unusual bleeding, easy bruising, blood in urine or stool, headache, confusion, slurred speech, or altered vision.

Defitelio may cause allergic reactions including anaphylaxis. Patients who develop signs and symptoms of anaphylaxis such as trouble breathing, severe itching, skin rash or hives, or swelling of the face, lips, mouth or tongue should seek medical attention immediately.

The most common side effects of Defitelio are decreased blood pressure, diarrhea, vomiting, nausea and nose bleeds. Please see full Prescribing Information for Defitelio before prescribing.

In Europe, defibrotide is marketed under the name Defitelio®▼( (defibrotide). In October 2013, the European Commission granted marketing authorization to Defitelio under exceptional circumstances for the treatment of severe VOD in patients undergoing HSCT therapy. It is the first and only approved treatment in Europe for severe VOD. In Europe, Defitelio is indicated in patients over one month of age. It is not indicated in patients with hypersensitivity to defibrotide or any of its excipients or with concomitant use of thrombolytic therapy.

▼This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions via the national reporting system found under section 4.8 of the SmPC. (http://www.ema.europa.eu/ema/index.jsp?curl=/pages/medicines/human/medicines/002393/human_med_001646.jsp)

For more information about Defitelio in the U.S., please visit www.Defitelio.com.

About VOD
HSCT is an aggressive, potentially curative procedure to treat patients with malignant and non-cancerous hematologic disorders such as leukemia, lymphoma and aplastic anemia, and congenital immunodeficiency and autoimmune disorders.² VOD is a rare complication of HSCT, which occurs in approximately 9-14% of HSCT patients.^3,4 Hepatic VOD, also known as SOS, is an early and life-threatening complication affecting the sinusoidal endothelial cells of the liver, which can typically occur within the first 21 days following HSCT.^4,5 Hepatic VOD progresses to multi-organ dysfunction in approximately 30-50% of cases.⁵ VOD with multi-organ dysfunction (MOD) is associated with an overall mortality (death) rate of 84%.³ MOD is characterized by the presence of renal or pulmonary dysfunction.^6,7 VOD is often characterized by sudden weight gain, hepatomegaly (abnormally enlarged liver), and elevated bilirubin.^6,7

About Jazz Pharmaceuticals
Jazz Pharmaceuticals plc (Nasdaq: JAZZ) is an international biopharmaceutical company focused on improving patients' lives by identifying, developing and commercializing meaningful products that address unmet medical needs. The company has a diverse portfolio of products and product candidates, with a focus in the areas of sleep and hematology/oncology. In these areas, Jazz Pharmaceuticals markets Xyrem® (sodium oxybate) oral solution, Erwinaze® (asparaginase Erwinia chrysanthemi) and Defitelio® (defibrotide sodium) in the U.S. and markets Erwinase® and Defitelio® (defibrotide) in countries outside the U.S. For more information, please visit www.jazzpharmaceuticals.com.

"Safe Harbor" Statement under the Private Securities Litigation Reform Act of 1995
This press release contains forward-looking statements, including, but not limited to, statements related to the expected impact of NTAP designation for Defitelio and other statements that are not historical facts. These forward-looking statements are based on the company's current plans, objectives, estimates, expectations and intentions, and inherently involve significant risks and uncertainties. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, which include, without limitation, the risks and uncertainties relating to the extent to which hospitals will take advantage of the NTAP program with respect to Defitelio, and other risks and uncertainties affecting the company, including those described from time to time under the caption "Risk Factors" and elsewhere in Jazz Pharmaceuticals plc's Securities and Exchange Commission filings and reports (Commission File No. 001-33500), including the company's Quarterly Report on Form 10-Q for the quarter ended March 31, 2016 and future filings and reports by the company. Other risks and uncertainties of which the company is not currently aware may also affect the company's forward-looking statements and may cause actual results and timing of events to differ materially from those anticipated. The forward-looking statements herein are made only as of the date hereof or as of the dates indicated in the forward-looking statements, even if they are subsequently made available by the company on its website or otherwise. The company undertakes no obligation to update or supplement any forward-looking statements to reflect actual results, new information, future events, changes in its expectations or other circumstances that exist after the date as of which the forward-looking statements were made.

References:
¹Defitelio (defibrotide sodium) [package insert]. Palo Alto, CA: Jazz Pharmaceuticals; March 30, 2016.
² Ikehara S. New strategies for BMT and organ transplantation. Int J Hematol. 2002;76(Suppl 1):161-4.
³ Coppell JA, Richardson PG, Soiffer R, et al. Hepatic veno-occlusive disease following stem cell transplantation: incidence, clinical course, and outcome. Biol Blood Marrow Transplant. 2010;16(2):157-168.
⁴Tsirigotis PD, Resnick IB, Avni B, et al. Incidence and risk factors for moderate-to-severe veno-occlusive disease of the liver after allogeneic stem cell transplantation using a reduced intensity conditioning regimen. Bone Marrow Transplant. 2014;49(11):1389-1392.
⁵ Carreras E, Díaz-Beyá M, Rosiñol L, et al. The incidence of veno-occlusive disease following allogeneic hematopoietic stem cell transplantation has diminished and the outcome improved over the last decade. Biol Blood MarrowTransplant. 2011;17(11):1713-1720.
⁶ Carreras E. How I manage sinusoidal obstruction syndrome after haematopoietic cell transplantation. Brit J Haematol. 2015 Feb.; 168 (4); 481-91.
⁷Mohty M, Malard F, Abecassis M, et al. Sinusoidal obstruction syndrome/veno‐occlusive disease: current situation and perspectives—a position statement from the European Society for Blood and Marrow Transplantation (EBMT). Bone Marrow Transplant. 2015;50(6):781‐789.