Roche has secured PRIME designation from the European Medicines Agency (EMA) for its risdiplam (RG7916) medicine to treat spinal muscular atrophy (SMA).
Risdiplam is an oral, investigational drug developed to help the SMN2 gene create more functional SMN protein in order to improve support to motor neurons and muscle function.
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SMA is an inherited, progressive neuromuscular disease. Previous studies show that the condition is a multisystem disorder and the SMN protein loss could impact tissues and cells beyond the central nervous system.
Risdiplam is designed for systematic distribution and durable increase of SMN protein levels in the central nervous system as well as throughout the body.
The medicine is being developed by Roche in partnership with the SMA Foundation and PTC Therapeutics.
Roche chief medical officer and Global Product Development head Sandra Horning said: “SMA is the leading genetic cause of death in young children, and families and clinicians continue to seek alternative treatment options for this progressively debilitating and life-threatening disease.
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By GlobalData“The EMA’s decision to grant PRIME designation recognises the potential of the oral systemic agent risdiplam to deliver clinically meaningful results for patients and address a continuing medical need in SMA.”
The EMA reviewed findings from Part 1 of the FIREFISH and SUNFISH clinical trials.
FIREFISH is investigating the safety and dosage of risdiplam in infants with Type 1 SMA, while SUNFISH involves children and adults with Type 2 and 3 SMA.
According to interim data from Part 1 of FIREFISH, infants administered with risdiplam met development milestones and were able to sit without support.
Data revealed that 90% of the patients remained alive, but two had to discontinue due to the fatal progression of their SMA.
SUNFISH Part 1 results showed a 3.1-point improvement in the median Motor Function Measure (MFM) change from baseline, which is the primary endpoint of the trial’s confirmatory part.
Till date, the investigators did not report any drug-related safety events that led to withdrawal.
