Takeda and AMAG receive a positive CHMP opinion in Europe for Rienso® (ferumoxytol)

Takeda Pharmaceutical Company Limited (Takeda) and AMAG Pharmaceuticals, Inc. today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion for ferumoxytol, a new intravenous (IV) iron therapy with a proposed indication for the treatment of iron deficiency anaemia (IDA) in adult patients with chronic kidney disease (CKD).

“This positive CHMP opinion marks an important step forwards for ferumoxytol. Takeda are looking forward to making this valuable new therapeutic option available to clinicians” said Trevor Smith, Head of Commercial Operations, Europe & Canada, Takeda Pharmaceuticals.

The CHMP opinion was based on data from three pivotal phase III clinical trials where ferumoxytol, administered as a rapid injection.^1,2,3 Each of the three pivotal safety and efficacy studies achieved statistical significance in its primary endpoint: the mean change in haemoglobin (Hb) from baseline at Day 35 after the first dose. Ferumoxytol significantly increased Hb levels as compared to oral iron across the spectrum of CKD. ^1,2,3 Overall, 1,726 subjects were exposed to ferumoxytol in the development program, including 1,562 patients with all stages of CKD. ² These studies also showed ferumoxytol was well tolerated by CKD patients with IDA and had a similar treatment related adverse event rate to oral iron. ¹ These outcomes were also supported in additional retrospective observational data from three large haemodialysis clinics in the United States involving more than 8,600 patients and more than 33,300 administered doses of ferumoxytol (nearly 50% of patients received repeat dosing with 4 or more doses). In this data set, mean Hb increased 0.5-0.9 g/dL post-treatement and mean Hb stabilised in the range of 11–11.7 g/dL over the 10 month post-dose period with no new safety signals identified with repeat dosing. ^4,5

Iron deficiency is a common cause of anaemia often seen in the later stages of CKD, as renal function deteriorates and erythropoiesis (red blood cell production) declines. IDA can have a profound impact on patients’ lives, causing fatigue, shortness of breath and an increase in the risk of cardiovascular (CV) complications including congestive heart failure. ^6,7

“Iron deficiency anaemia can be a debilitating condition for chronic kidney disease patients and appropriate management of this condition carries positive clinical implications for patients. Therefore, treatment of anaemia at all stages of CKD is essential and the potential availability of ferumoxytol offers an additional therapeutic option to help effectively and conveniently manage this condition.” Iain Macdougall, Consultant Nephrologist and Professor of Clinical Nephrology at King’s College Hospital, London.

About ferumoxytol

Ferumoxytol is an IV iron therapy with a proposed indication for the treatment of IDA in adult patients with CKD. Its structure and formulation allows for the administration of 510 mg in a shorter time frame than existing IV iron preparations . Ferumoxytol significantly increases Hb levels in CKD patients both on dialysis and in patients not on dialysis compared with oral iron. Clinical trials have also highlighted that ferumoxytol is well tolerated.

Ferumoxytol was developed by AMAG Pharmaceuticals, Inc. and is marketed outside the US by Takeda Pharmaceuticals following announcement of a comarketing agreement in March 2010. Ferumoxytol is currently approved for use in Canada and the US as Feraheme®.

References

1. Spinowitz BS, Kausz AT, Baptista J, et al. Ferumoxytol for treating iron deficiency anemia in CKD. J Am Soc Nephrol 2008; 19: 1599–1605
2. AMAG Pharmaceuticals. Data on file.
3. Provenzano R, Schiller B, Rao M, et al. Clin J Am Soc Nephrol 2009;4:386–3935
4. Schiller B, Bhat P, Sharma A, Li Z, Fortin G, McLaughlin J, Strauss W. Safety of Feraheme® (Ferumoxytol) in hemodialysis patients at 3 dialysis chains over a 1-year period. J Am Soc Nephrol 2011;22:477A–478A. Abstr FR-PO1573.
5. Sharma A, Bhat P, Schiller B, Fortin G, McLaughlin J, Li Z, Strauss W. Efficacy of Feraheme® (Ferumoxytol) administration on target hemoglobin levels and other iron parameters across 3 dialysis chains. J Am Soc Nephrol 2011;22:485A. Abstr FR-PO1603
6. O’Mara NB. Anemia in patients with chronic kidney disease. Diabetes Spectrum 2008;21:12–19.
7. National Kidney Foundation. KDOQI clinical practice guidelines and clinical practice recommendations for anemia in chronic kidney disease. Am J Kidney Dis 2006;47(suppl 3):11–1458