rhNGFRecombinant human Nerve Growth Factor
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NEW YORK, July 23, 2014 /PRNewswire/ -- Dompe, a leader in R&D for new therapeutic solutions for the treatment of rare diseases, announces that rhNGF (Recombinant Human Nerve Growth Factor) has been designated as an orphan drug by the American Food and Drug Administration (FDA).
This is the second orphan drug designation by the FDA for rhNGF, after the recent designation for the treatment of retinitis pigmentosa, a rare genetic disease that affects more than 1 million people worldwide[sup.
The more than 170 patients enrolled in the study are divided into three different groups, treated respectively with two different doses of rhNGF and with the placebo.
rhNGF is now in Phase II development and patient enrolment is underway.
Preliminary data from Phase I of the study, involving patients affected by moderate or severe neurotrophic keratitis, were presented at the ARVO Annual Meeting (Orlando, USA, 4-8 May 2014) and demonstrated that rhNGF is well tolerated.
Although the data are still "masked" and, therefore, it is not yet known which patients received rhNGF and which received the placebo, complete resolution of corneal lesions was recorded in the majority of patients, with similar percentages in the two groups treated with rhNGF at different doses, as well as an increase in corneal sensitivity in approximately one in every three patients.
We are, therefore, very interested in potential treatments such as that offered by rhNGF, which could lead to new perspectives in the treatment of retinitis pigmentosa.
For more information about rhNGF and Retinitis Pigmentosa: http://www.
Francesco Sinigaglia, CEO of Anabasis Pharma, commented: "It is excellent that we have been able to find a cost efficient and patent protected microbial process for the manufacture of rhNGF.