Oral therapy for SMA listed on the PBS
Australians with spinal muscular atrophy (SMA) types 1, 2 or 3a who are aged 18 years or under when treatment begins now have PBS subsidised access to risdiplam (Evrysdi).
The oral daily therapy for SMA is a less invasive treatment option for patients compared to spinal injections with nusinersen which may also reduce the frequency of visits to specialist hospitals.
Federal Health Minister Greg Hunt said that without PBS subsidy, about 100 Australians a year would pay more than $123,000 for treatment.
As previously reported in the limbic, the PBAC recommended PBS listing of risdiplam based on cost effectiveness comparable with nusinersen.
Ocrelizumab advice on neutropenia
Ocrelizumab (Ocrevus) patients may experience late onset neutropenia, the TGA has advised.
The Product Information’s ‘Adverse Effects’ and ‘Special Warnings and Precautions for Use’ sections have been updated to include reported times to onset, severity and recommendations for identifying the side effect.
Late onset neutropenia has been reported from four weeks to several months after the latest ocrelizumab infusion, the Product Information said.
Although some clinical trial cases were Grade 3 or 4, most were Grade 1 or 2.
“In patients with signs and symptoms of infection, measurement of blood neutrophils is recommended,” the Product Information read.
As of 16 June 2021, the TGA was aware of four ocrelizumab-associated neutropenia cases, with ocrelizumab being the sole-suspected drug in three of those cases.