Genentech’s Risdiplam showed significant improvement in motor function in people aged 2-25 who have been diagnosed with Type 2 or 3 spinal muscular atrophy. The positive news provides the company with a boost of confidence as it anticipates potential approval of the medication later this year.
This morning, the company released anticipated one-year data from the pivotal Part 2 of the SUNFISH trial. This is the first placebo-controlled trial to include adults with SMA that demonstrated risdiplam improved or stabilized motor function in those patients. For people with SMA, motor function improvement translates to the preservation of independence. Data from the trial was presented at the 2nd International Scientific and Clinical Congress on Spinal Muscular Atrophy in Evry, France.
Spinal muscular atrophy (SMA) is a severe, inherited, progressive neuromuscular disease that causes devastating muscle atrophy and disease-related complications. It is one of the most common rare diseases, affecting approximately one in 11,000 babies. SMA leads to the progressive loss of nerve cells in the spinal cord that controls muscle movement. Depending on the type of SMA, an individual’s physical strength and their ability to walk, eat or breathe can be significantly diminished or lost.
The findings from this phase of the SUNFISH trial provide further validation for risdiplam, an investigational oral liquid survival motor neuron-2 (SMN-2) splicing modifier for people living with SMA that can be administered at home, Genentech said in its announcement. The trial hit “medically-meaningful and statistically significant results in primary and key secondary endpoints,” the company added, and also represented “broad, real-world spectrum of people living with SMA.”
Part 2 of the SUNFISH study showed the change from baseline in the primary endpoint of the Motor Function Measure scale, which showed the change from baseline was significantly greater in people treated with risdiplam, compared to placebo. Also, the trial hit the key secondary endpoint in Revised Upper Limb Module test. Patients showed an improvement in this area, a 1.59 point difference, Genentech said in its announcement.
The company noted that, as expected, exploratory subgroup analyses showed that the strongest responses in MFM-32 versus placebo were observed in the youngest age group – a 78.1% versus 52.9% difference, Disease stabilization was observed in the 18-25 years age group at 57.1% versus 37.5%, Genentech said. Safety findings remained consistent with other risdiplam studies.
Levi Garraway, chief medical officer and head of global product development for Roche, the parent company of Genentech, said they were encouraged by the results from the broad group of SMA patients.
“This study has helped us understand which measurement scales are the most relevant for patients, as well as the importance of stabilization in people with more established disease,” Garraway said in a statement.
The data from Part 2 of the SUNFISH trial follows hard on the heels of the announcement of positive results from Part 2 of the FIREFISH study. That study focused on the use of risdiplam in patients aged 1-7 months with Type 1 SMA. The primary endpoint of Part 2 of the FIREFISH trial was the proportion of infants sitting without support for at least five seconds at 12 months of treatment.
In November, the U.S Food and Drug Administration granted Priority Review for risdiplam with a decision for approval by May 24. The company is seeking approval for risdiplam in a broad population of people living with Types 1, 2 or 3 SMA. The risdiplam NDA submission incorporates 12-month data from the dose-finding Part 1 sections of the FIREFISH and SUNFISH pivotal studies, as well as data from the confirmatory Part 2 of SUNFISH.
If risdiplam is approved, it will become the third SMA treatment in the United States behind Biogen’s Spinraza and Novartis’ gene therapy treatment, Zolgensma. In order to make up for being third to market, Roche said last month it could undercut the other two drugs in its pricing model. Spinraza is priced at $750,000 for the first year and $375,000 each year afterward. Novartis’ Zolgensma is priced at $2.1 million. It is believed that the gene therapy could be a one-time treatment for the disease.
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