Friday Five – The Pharma Week in Review (3 September 2021)

COVID-19 vaccine boosters

New restrictions for JAKs

Jardiance shines at ESC

Novartis details ground-breaking Leqvio access agreement

  • Having secured Leqvio through its $10-billion acquisition of The Medicines Company at the back end of 2019, Novartis has long touted the blockbuster credentials of this novel cholesterol-reducing therapy.
  • With no competition in this space for the foreseeable future, removing as many market access barriers as possible is viewed as critical to Novartis achieving its commercial goals for Leqvio, which cardiologists have acknowledged to be a highly effective and safe product.
  • To this end a new agreement with NHS England, which looks to position Leqvio as a preventative therapy, could act as a blueprint for its launch in other markets and other drugs that can be proactively used to reduce future burden on healthcare systems, says Novartis.
  • See – ViewPoints: Novartis extends innovation to market access playbook.

Astellas setback highlights FDA caution

  • The lead gene therapy from Astellas' big-money buyout of Audentes Therapeutics in 2019 is back in hot water and may prove to be the death knell for the programme, which comes as a timely example of the type of safety issues the FDA has convened an AdCom to discuss this week (Thursday/Friday).
  • On Wednesday, Astellas disclosed that it had paused dosing of AT132, an AAV8-based gene therapy candidate being evaluated in the Phase I/II ASPIRO trial for X-linked myotubular myopathy (XLMTM) after abnormal liver function tests) were reported in one patient who received the lower dose of AT132.
  • The company said it reported the case as a serious adverse event to the FDA, which has not yet placed the study on another hold but still may do so.
  • Coincidentally, the FDA is hosting a two-day meeting of its Cellular, Tissue, and Gene Therapies Advisory Committee to discuss the safety of AAV-based gene therapy products. The panel was scheduled earlier this year after high profile safety stumbles were reported by three separate companies, which included Astellas along with Prevail Therapeutics and Passage Bio

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