@rurahrah000 - More info on BLUE. Not as bad as I thought on 2024 patients treated guidance. However, I'm not sure what their GTN will be to calculate net sales. Still interesting:
Bluebird’s financial picture was hurt when the approval did not include a priority review voucher, which it had planned to sell for to Novartis AG for $103m. (Also see "Bluebird’s Lost Voucher: Lyfgenia Contains Same ‘Active Ingredient’ As Zynteglo, US FDA Says" - Pink Sheet, 14 Dec, 2023.)
However, president and CEO Andrew Obenshain noted in his J.P. Morgan presentation that bluebird’s three approved products and its $275m in cash as of 31 December plus additional non-dilutive financing options that the company is pursuing should build a bridge to profitability.
Bluebird anticipates starting the treatment process for 85-105 patients in 2024 with its three gene therapies – Lyfgenia, Zynteglo and Skysona (elivaldogene autotemcel) for the ultra-rare disease cerebral adrenoleukodystrophy (CALD) – which Obenshain said represents about $300m in gross revenue. The three products have a combined addressable population in the US of 22,000 patients, including 20,000 with sickle cell disease who have recurrent vaso-occlusive events (VOEs) and 1,500 with transfusion-dependent beta-thalassemia.
“Our market research has consistently shown that about 70% of sickle cell patients would consider a gene therapy if it was offered by their physicians,” Obenshain said. “And, importantly, 80% of providers want Lyfgenia and its competitor to be available at their institutions, so this is likely to be widely available across the US.”