Roche is to present new data on its late-stage spinal muscular atrophy (SMA) drug risdiplam at the American Academy of Neurology (AAN) meeting early next month, and the candidate has potential to be the first oral treatment approved for the muscle degenerative disease.
If all goes well, risdiplam could enter a field currently dominated by Biogen’s first-to-market and only approved SMA therapy Spinraza.
That drug was approved in the US in 2016 and in the EU six months later, and is of the company’s key growth drivers, generating $518m in revenues in the quarter.
However, Spinraza has to be administered intrathecally every four months, while Roche’s drug, which could hit the market in 2021, has a much more more convenient oral formulation.
Roche is expected to present updated safety data for the survival motor neuron 2 (SMN2) splicing modifier at the AAN congress, along with one-year data from the dose-finding Part 1 of the FIREFISH study on key motor milestones, motor function and survival in infants with Type 1 SMA.
It will also present an exploratory efficacy analysis from part 1 of the SUNFISH study, which evaluated the drug in those aged 2-25 years with SMA types 2 or 3.
“The great need for therapeutic options in areas of neuroscience such as SMA, HD and NMOSD means that every development is a collective step forward,” said Sandra Horning, chief medical officer and head of global product development.
Risdiplam’s safety data could see Roche continue to build its case for the drug in SMA, and the data could even pose as a threat to Novartis’ SMA type 1 gene therapy Zolgensma. The one-time therapy has generated great excitement, and is just weeks away from an FDA decision.
However its approval and future use has been called into question after the death of two patient deaths in clinical trials.
One patient died from respiratory failure in a separate European trial of Zolgensma, but the incident was found to be unrelated to the gene therapy following an investigation.
Last month, another patient death occurred during the phase 3 STR1VE trial, however this time a Novartis spokesperson confirmed that it could be related to the treatment after an investigation.
Despite no conclusive results yet, these concerns cast a cloud over the drug’s stellar efficacy data, whereby 100% of patients enrolled into the STR1VE study of the infants with the SMA type 1 subset were still alive at 24 months.
The disease normally has a survival rate of just 50% by the age of 10 months in these infant patients born with SMA type 1.
Novartis is also scheduled to present more data from its STR1VE and START trials, the latter of which tested Zolgensma as an intrathecal formula, which is injected into the spinal canal.
Nevertheless, all eyes will be on pricing, which could shape the future of the SMA market.
Biogen’s Spinraza is priced at $750,000 for the first year, then $375,000 per year thereafter.
Roche has not made a comment regarding its pricing strategy for risdiplam. Zolgensma’s price is unconfirmed, but Novartis CEO Vas Narasimhan has previously indicated that the one time therapy could be priced anywhere between $1.5-$5m.