More than 50? Interest from drug developers in NP drugs is said to have waned in recent years because most of those approved to date, while having demonstrated reduced toxicity, haven’t been as successful in demonstrating improved efficacy. This remains one of the big challenges in the field.
There’s been only one LNP siRNA - patisiran (Onpattro), a lipid nanoparticle encased siRNA rare disease drug developed by Alnylam - approved by both FDA and the EC last August. Phase 3 trial results can be found here.
It would seem that the FDA was impressed with patisiran. It was granted breakthrough therapy designation and FDA Commissioner, Scott Gottlieb, said on the drug’s approval
“New technologies like RNA inhibitors, that alter the genetic drivers of a disease, have the potential to transform medicine, so we can better confront and even cure debilitating illnesses.”
It might be recalled that PYC referred to results achieved with the GaINac- cargo approach as a delivery benchmark in January’s update.
Alnylam licensed the LNP technology that was used in the development of patisiran from Canadian biotech, Arbatus, which has since licensed exclusive rights to the LNP technology to its new JV, Genevant. Genevant is aggressively aiming to advance between five and ten product candidates into the clinic by 2020 across RNAi, mRNA, and gene editing modalities. It currently has just one partner other than Arbutus - BioNTech.
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