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
In the final rule concerning Hospital Inpatient PPS and CMS Fiscal Year (FY) 2017, (scheduled for publication in the Federal Register on
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
Effective for the Fiscal Year 2017 (
Introduced in 2001, NTAP was created by
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
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
▼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
"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
References:
1 Defitelio (defibrotide sodium) [package insert].
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.
4 Tsirigotis 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.
7 Mohty M, Malard F, Abecassis M, et al. Sinusoidal obstruction syndrome/veno‐occlusive disease: current situation and perspectives—a position statement from the
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SOURCE
Investors: Kathee Littrell, Vice President, Investor Relations, Ireland, +353 1 634 7887, U.S., +1 650 496 2717; Media: Laurie Hurley, Vice President, Corporate Affairs, Ireland, +353 1 634 7894, U.S., +1 650 496 2796