Evrysdi (risdiplam) — Point32Health

Initial criteria

• Documented diagnosis of spinal muscular atrophy type 1, 2, or 3 confirmed by molecular genetic testing of any of the following: SMN1 homozygous gene deletion, homozygous conversion mutation (SMN1 gene conversion to SMN2 gene), or compound heterozygote mutation
• Prescribed by or in consultation with a board-certified neurologist with special qualification in child neurology and treatment of spinal muscular atrophy
• Documentation of baseline (pre-treatment) motor function skills as measured by one of the following: Hammersmith Infant Neurological Exam Part 2 (HINE-2) OR Hammersmith Functional Motor Scale Expanded (HFMSE) OR Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND)
• Documentation the patient is not using Evrysdi in combination with Spinraza
• Documentation of one of the following: (a) The patient has not previously received gene replacement therapy for the treatment of spinal muscular atrophy OR (b) The patient has previously received gene replacement therapy and has experienced a decline in clinical status since receipt of gene replacement therapy
• Documentation the patient is not ventilation dependent (defined as using a ventilator 16 hours or more a day)

Reauthorization criteria

• Documented diagnosis of spinal muscular atrophy type 1, 2, or 3 confirmed by molecular genetic testing
• Prescribed by or in consultation with a board-certified neurologist with special qualification in child neurology and treatment of spinal muscular atrophy
• Documentation the patient is not using Evrysdi in combination with Spinraza
• Documentation of one of the following: (a) The patient has not previously received gene replacement therapy for the treatment of spinal muscular atrophy OR (b) The patient has previously received gene replacement therapy and has experienced a decline in clinical status since receipt of gene replacement therapy
• Documentation the patient is not ventilation dependent (defined as using a ventilator 16 hours or more a day)
• Documentation of disease stabilization or clinical improvement of spinal muscular atrophy symptoms as demonstrated by at least one of the following assessments: (a) HINE-2: improvement or maintenance of at least a 2-point increase in ability to kick OR at least a 1-point increase in any other HINE-2 milestone excluding voluntary grasp, and net positive improvement in motor milestones or achieving new unexpected motor milestones; OR (b) HFMSE: improvement or maintenance of at least a 3-point increase in score or achieving new unexpected motor milestones; OR (c) CHOP-INTEND: improvement or maintenance of at least a 4-point increase in score or achieving new unexpected motor milestones

Approval duration

12 months