Spinal muscular atrophy (SMA) is a genetic neuromuscular condition, affecting approx. 1 in 6,000 babies born globally each year. Numerous healthcare companies throughout the world are putting their best foot forward to develop an effective treatment of this disease. 

Roche Holding AG is one such Swiss multinational healthcare company. Roche has reportedly announced that a phase 3 trial of risdiplam has met its primary endpoint. The clinical trial was carried out to evaluating risdiplam in patients that are suffering from type 2 or 3 SMA. 

Through the positive results of the phase 3 trial, Roche’s hopes of taping into a slice of the SMA market have been buoyed. 

The company’s phase 3 trial enrolled patients that are suffering from less serious types of the debilitating muscle wasting disease. The type 2 and type 3 forms of the disease are less serious forms of Spinal Muscular Atrophy. However, they could still result in respiratory infections, mobility problems, and death. 

Reportedly, to evaluate risdiplam, the company enrolled 180 patients that are suffering from type 2 or type 3 SMA in its phase 2/3 clinical trial’s second part and further randomized to obtain placebo or the splicing modifier SMN2 through oral administration. 

A year after the treatment, the company noticed a change that is statistically significant on the motor function scale of MFM-32, which allowed the trial to meet its primary endpoint. Roche is planning to share these results with regulators to bring risdiplam to market in several nations including the U.S. 

However, the company has not yet shared the data from the second portion of Phase 2/3 clinical trial, making it difficult to determine its probability of winning an approval. However, the promising results of the first part of the 2/3 clinical trial signal that risdiplam could give tough competition to existing drugs like Biogen’s Spinraza and Novartis’ Zolgensma.

Sources cite that the company is planning to share the data from a 2/3 clinical trial at an upcoming medical congress. Meanwhile, the Swiss Big Pharma is working to generate data through another trial that has enrolled patients who are suffering from type 1 SMA as well. 

If the data supports a 2020 approval for all three SAM types, the SMA community would get access to the company’s third drug within a span of approximately three years. Roche would then have to persuade patients that risdiplam is a better alternative than Novartis’ gene therapy based Zolgensma and Biogen’s antisense oligonucleotide Spinraza. 

Source credit - https://www.fiercebiotech.com/biotech/roche-s-challenger-for-sma-market-hits-goal-phase-3