Cytheris SA, a clinical-stage biopharmaceutical company focused on treating lymphopenia driven diseases, has announced that the US Food and Drug Administration (FDA) has granted an Orphan Drug Designation for Cytheris’ CYT017, glycosylated recombinant human interleukin-7 (glycosylated r-h-IL7), for the treatment of Progressive Multifocal Leukoencephalopathy (PML). This designation from the FDA follows orphan drug designation for the same indication granted in Europe in July 2012 from the European Commission.
PML is a severe demyelinating disease of the central nervous system caused by the JC virus. PML is a very rare disease, affecting over 4,000 people in the US and EU alone. There is currently no specific marketed drug for the treatment of this devastating condition. JC virus reactivation occurs in conditions that lead to severe lymphopenia (lack of T cell immunity). These conditions include HIV infection, some cancers and organ transplantation treated with immunosuppressive therapies. CYT107 is ideally placed to bring clear benefits through its targeted activity on immune T cell recovery and enhancement.
“This second orphan drug designation strongly adds support from the FDA to that of the European Commission for the development of CYT107 as the treatment for PML. The disease has a mortality rate of around 40 to 50 per cent at one year whilst survivors often present severe neurological complications and disabilities,” said Therese Croughs, chief medical officer at Cytheris. “We have already gathered clinical data supporting the efficacy and safety of CYT107 in PML through several compassionate use treatments. We are committed to conduct a pivotal clinical study rapidly to obtain regulatory approval and make CYT107 available as soon as possible to all patients suffering from this devastating disease.”
Cytheris has previously obtained scientific advice from the European Medicines Agency (EMA) for a pivotal phase IIb study protocol with CYT107 in HIV-related PML. Cytheris has reached an agreement with the EMA on the key study endpoints and will start this study in early 2013.
“PML affects around over 4,000 people in the US and EU alone and it exacts a very heavy toll on all those affected. Bringing a treatment to this population will both save lives and vastly improve the outlook of those who survive,” said Damian Marron, chief executive officer at Cytheris. “It will also respond to the need of health services globally as well as the pharmaceutical industry to develop innovative, breakthrough drugs for targeted indications.”
Alongside treatment for PML, CYT107 has also been shown to be able to treat lymphopenia in HIV Immune Non-Responders (HIV-INR) who are virologically controlled by anti-retroviral treatment. Fifteen to twenty five per cent of HIV patients are lymphopenic despite optimal treatment (HAART), leading to a significant increase in risk of death and serious non-AIDS complications. CYT107 also holds great promise in other lymphopenic conditions such as post-transplant (see release of 12 October 2012), in idiopathic lymphopenia or in cancer-associated lymphopenia.