PTC Eyes Transition From Capital-Raising To Money-Making Company
With $1.1bn in cash plus revenue from its existing commercial products and royalties from Roche’s sales of newly approved Evrysdi to invest in R&D, PTC Therapeutics may achieve profitability in 2023.
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The rollercoaster ride continues for Novartis’s gene therapy Zolgensma, with the company committing to providing further clinical data on an intrathecal formulation for older spinal muscular atrophy patients.
Risdiplam’s efficacy across two clinical trials and oral dosing made it a credible threat before its US FDA approval, but its weight-based pricing falls below Biogen’s Spinraza and Novartis’s Zolgensma.
Some reimbursement agencies in Europe have been misinterpreting Translarna’s label and stopping access to the drug for boys with Duchenne muscular dystrophy who have been taking it but whose disease has progressed, PTC says.