The European Medicines Agency (EMA) has granted PRIME designation for Roche’s investigational oral medicine risdiplam for treating people with spinal muscular atrophy (SMA).
Risdiplam, an orally administered, survival motor neuron-2 (SMN2) gene splicing modifier, has shown improvements in motor function in people with SMA Types 1, 2 and 3.
An increasing body of clinical evidence suggests that SMA is a multisystem disorder, and the loss of SMN protein may affect many tissues and cells beyond the central nervous system. Risdiplam is systemically distributed and designed to durably increase SMN protein levels in the central nervous system and throughout the body.
Roche leads the clinical development of risdiplam as part of a collaboration with the SMA Foundation and PTC Therapeutics.
“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,” said Sandra Horning, Roche’s Chief Medical Officer and Head of Global Product Development.
“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 designation is based on data from Part 1 of the pivotal studies FIREFISH (evaluating safety and determining dosage in infants with Type 1 SMA) and SUNFISH (in children and adults with Type 2 and 3 SMA) as well as a continuing medical need for alternative treatments and administration options for patients with SMA.
Interim data from Part 1 of the FIREFISH study showed that infants with Type 1 SMA treated with risdiplam met developmental milestones, including sitting without support.
Nineteen of 21 risdiplam-treated patients (90%) remained alive with two having discontinued due to the fatal progression of their disease. No infant required a tracheostomy or permanent ventilation since study initiation, and no infant has lost the ability to swallow.
Interim data from Part 1 of the SUNFISH study in Type 2 and 3 SMA, demonstrated a median >2-fold increase in SMN protein levels in the blood following 12 months of treatment.