US-based Alnylam Pharmaceuticals has received positive opinion from the European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP) recommending orphan drug designation (ODD) for ALN-TTRsc, a subcutaneously delivered RNAi therapeutic for the treatment of transthyretin (TTR) mediated amyloidosis (ATTR).

Currently, ALN-TTRsc is in a pilot Phase II clinical trial for the treatment of ATTR patients with TTR cardiac amyloidosis.

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The Phase II trial is aimed at assessing the tolerability of ALN-TTRsc in about 15 patients and the results are expected to be released towards the end of this year.

In addition, the trial will evaluate preliminary clinical activity as measured by knockdown of serum TTR levels and additional exploratory tests such as cardiac imaging, circulating cardiac biomarkers, six-minute walk test, New York Heart Association (NYHA) classification, and measures of heart failure symptoms and quality of life.

"We believe RNAi therapeutics represent a promising new approach for the treatment of ATTR, with the potential to make a meaningful impact for patients with this progressive and debilitating disease."

Alnylam senior vice-president of regulatory affairs and quality assurance Saraswathy Nochur said: "We believe RNAi therapeutics represent a promising new approach for the treatment of ATTR, with the potential to make a meaningful impact for patients with this progressive and debilitating disease.

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"We look forward to sharing Phase II clinical data from our ALN-TTRsc programme later in the year, and, assuming continued positive results, we plan to advance to a Phase III pivotal trial in ATTR patients with TTR cardiac amyloidosis by the end of the year."

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The company said that patients completing the Phase II trial will be eligible to participate in an open-label extension (OLE) trial for further assessment of general tolerability and clinical activity with long-term dosing.

The ALN-TTRsc Phase II OLE trial is expected to be started in mid-2014.

Assuming positive results, the company expects to start a Phase III trial in TTR cardiac amyloidosis patients by the end of 2014.

In January 2014, Genzyme and Alnylam formed an alliance to accelerate and expand the development and commercialisation of RNAi therapeutics worldwide.

As part of the alliance, Alnylam retains product rights in North America and Western Europe, while Genzyme obtains the right to access Alnylam’s current ‘5×15’ and future genetic medicines pipeline in the rest of the world (ROW), including co-development/co-commercialization and/or global product rights for certain programs.

In the case of ALN-TTRsc, both the firms are co-developing and co-commercialising the product in North America and Western Europe, while Genzyme will advance the product in the ROW.