A number of development projects on selected growth disorders and metabolic diseases are being pursued in the therapeutic area of Endocrinology. Merck Serono can build upon its long-standing experience in these therapeutic indications.
The furthest advanced project is the development of high-dose recombinant human growth hormone (r-hGH) for the treatment of HIV-associated fat redistribution syndrome, also called HIV-associated adipose redistribution syndrome (HARS). Merck Serono continues discussions with the U.S. FDA concerning the approval of r-hGH for this indication. Serostim® is approved for HIV-associated wasting. A long-acting growth hormone, ARX-201, is being investigated in cooperation with Ambrx, a U.S. biopharmaceutical company, in Phase I/II clinical trials for the treatment of growth hormone deficiency.
In November 2007, Merck Serono submitted an application to the European Medicines Agency (EMEA) for the marketing authorization of sapropterin (formerly known as Phenoptin™) for the oral treatment of hyperphenylalaninemia (HPA) due to phenylketonuria or tetrahydrobiopterin deficiency. Sapropterin received Orphan Medicinal Product designation to treat HPA from both the FDA and the EMEA.
