Prevalence of Duchenne in the general population is said to be approximately 1 in 40,000.
Exondys 51, Sarepta’s first approved drug for the condition, targeted those who were amenable to exon 51 skipping, which is estimated to be about 13-14% of the DMD population. That gives a prevalence for this subgroup of approximately 1 in 300,000, suggesting a US population of ~1,100. Sarepta’s most recently approved DMD drug, Vyondys 53, targets another DMD (8-9%) subgroup. That gives a prevalence for this subgroup of approximately 1 in 450,000.
Retinitis Pigmentosa is said to impact 1 in 3,500-4,000 of the general population. If the subgroup to be targeted by PYC’s lead drug represents approximately 2% of the RP population, this would equate with a prevalence of approximately 1 in 200,000, suggesting a US population of ~ 1,650. These rough, back of envelope calculations suggest that the market for PYC’s lead drug, if successfully developed, should be 50% larger than that for Exondys 51.
Exondys 51 sales in the last quarter reached US$99 m. Annual cost of the drug per patient is dependent on weight but is estimated to average almost US$900,000.
A personalised tool to help users track selected stocks. Delivering real-time notifications on price updates, announcements, and performance stats on each to help make informed investment decisions.
(20min delay)