10. Risdiplam

Roche
Roche's risdiplam will follow Novartis' Zolgensma and Biogen's Spinraza into the spinal muscular atrophy market. (Roche)

Drug: risdiplam
Company: Roche
Used for: spinal muscular atrophy
Est. 2024 sales: $803 million

In the extremely pricey spinal muscular atrophy (SMA) market, Novartis and Biogen have taken the lead with their drugs passing FDA muster in 2019. But Roche and partner PTC Therapeutics are aiming to take their own late-stage SMA candidate to market, and a priority review could help get it in physicians' hands faster.

Roche's risdiplam could hit $803 million in global sales by 2024, according to EvaluatePharma, if the FDA gives it the green light by its May decision deadline. 

The FDA agreed to review the drug in November 2019 based on 12-month data from a study called Firefish in type 1 SMA patients, plus data in type 2 and 3 patients from a study called Sunfish. The drugmaker is testing the candidate in a broad patient population, ranging from newborns to age 60, including those who have taken other SMA medications. 

In mid-January, risdiplam hit its primary endpoint in another phase 3 trial in infant patients with type 1 SMA, which assessed whether infants were able to sit without support for at least five seconds after receiving daily doses of risdiplam for one year. 

Risdiplam, a survival motor neuron 2 splicing modifier, is an orally administered liquid that patients would take at home. Biogen's Spinraza is an injection with several loading doses over the first two-and-a-half months and maintenance doses every four months thereafter. Novartis’ gene therapy Zolgensma is a one-time treatment for patients under 2. 

If the oral drug reaches approval, Cantor analyst Alethia Young said in a recent note that it could be a "game changer" in SMA. 

RELATED: Roche scores FDA priority review for risdiplam, threatening Biogen and Novartis SMA meds

Analysts believe the new Roche drug could bring in $2.5 billion to $3 billion at peak. As of November 2019, Biogen’s Spinraza had pulled in $1.55 billion. Zolgensma had generated $160 million since launch. 

Risdiplam scored the FDA’s orphan drug designation in January 2017 and fast-track designation in April 2017. While the FDA’s official decision date is May 24, Cantor's Young wrote that an approval could come earlier than that. 

Regardless of when the drug scores a nod, analysts expect risdiplam to bring big changes to the market upon launch. Young wrote that it’ll “meaningfully change the SMA landscape in 2020."

Biogen launched its Spinraza first in the field and the rollout has surpassed all expectations. Novartis has hit some hiccups with Zolgensma, including a data manipulation controversy that didn't dent demand. Analysts still believe the drug will generate blockbuster sales. 

10. Risdiplam

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