Alnylam Pharmaceuticals’ (ALNY) target indications include genetic medicines, cardio-metabolic diseases, and hepatic infectious diseases. These are indications with high unmet need, early biomarkers for assessing clinical activity in phase one studies, and a definable path for drug development, approval, patient access, and commercialization.
Alnylam Pharmaceuticals aims to have three products on the market and ten RNAi therapeutic clinical programs by the end of 2020. Of these ten programs, Alnylam aims to have four in the late stages of development across its three target indications.
In line with this vision, Alnylam filed its first new drug application (or NDA) in December 2017 for patisiran. In January 2018, the European Medicines Agency accepted Alnylam’s marketing authorization application (or MAA) for patisiran. Additionally, the FDA accepted its NDA in February 2018 and has set an action date of August 11 for the drug.
Alnylam Pharmaceuticals’ givosiran is an investigational RNAi therapeutic under development for treating acute hepatic porphyrias (or AHPs). In April 2018, Alnylam presented new phase one and two open-label extension (or OLE) study results. Additionally, Alnylam completed enrolling the first 30 patients in its ENVISION phase three study, which consists of the interim analysis cohort for potential accelerated approval by the FDA.
Further, Alnylam expects to report interim analysis results in September 2018, and assuming positive results, it expects to submit a new drug application (or NDA) with the FDA by the end of 2018 and an MAA with the EMA in 2019.
In the next part of the series, we’ll take a look at the other programs in Alnylam’s research pipeline.