With Roche's SMA candidate risdiplam stalled at the FDA for three more months, it's a reprieve for the two therapies it would have threatened—Biogen's Spinraza and Novartis' gene therapy Zolgensma. But the relief could be short-lived, and might even work to Roche's favor in the end.
That's because the agency prolonged its review to weigh new data in patients with Type 2 or 3 of the disease, a move that might yield a broader approval for risdiplam if and when it comes, SVB Leerink analysts wrote Tuesday.
Plus, had Roche won a green light now, it might have waited to launch for a few months anyway, given the COVID-19 lockdowns around the U.S.
For Novartis and Biogen, the delay certainly offers an “incremental positive,” SVB Leerink's Mani Foroohar noted in a Tuesday missive to investors.
In absolute dollar terms, though, the effect is relatively minor. Foroohar is only projecting $36 million in 2020 risdiplam sales, versus $2.2 billion and $660 million for Spinraza and Zolgensma, respectively. But the news “could ease near-term competitive dynamic,” he said.
Risdiplam is considered a threat to those existing meds because, if approved, it would be the first oral treatment for people with SMA, while both Spinraza and Zolgensma are injectables.
Foroohar argues Spinraza should see most of the benefit from the regulatory delay because the Biogen drug is allowed in both pediatric and adult patients with different subtypes of SMA. In contrast, Zolgensma as an intravenous infusion is currently only for infants younger than 2.
But because Novartis is looking to tap into older patients with an intrathecal formulation, Foroohar also sees “potentially meaningful benefit” for the gene therapy dependent on the timing of that expansion, “where competition with risdiplam is likely to be most relevant.”
The FDA is now slated to make a decision by August 24. And to hear Roche tell it, the extension might eventually help risdiplam gain an approval in a broader range of patients.
The delay is caused by additional data Roche unveiled in February from part 2 of the pivotal Sunfish trial in older patients with Type 2 or 3 SMA, which are less severe forms compared with Type 1. The results showed that patients treated with risdiplam delivered more significant improvements on a commonly used scale that measures motor function in people with neurological disorders.
Roche submitted those data to the FDA based on their discussions, even though the agency had already granted priority review for the drug back in November. Apparently, the FDA suggests it needs more time to review the full body of data, which could give Roche an opportunity for a broader label at launch.
“We think getting a broader label is worth a three-month trade-off when thinking about competition that exits on the market already,” Cantor Fitzgerald analyst Alethia Young wrote in a Tuesday note to clients.
Indeed, rolling out a new drug amid the ongoing COVID-19 pandemic could be challenging, especially without the help of all the marketing tools available and potentially a lack of attention from physicians and patients. In a recent report, RBC Capital analyst Brian Abrahams argued that new launches would be even more difficult for products aiming to compete with existing drugs. In risdiplam’s case, Spinraza has established itself as the SMA market leader and Zolgensma is also gaining Street-beating interest.
Roche could use the additional three months to wait out the COVID-19 crisis. And who doesn’t like a broader label?