Treatments for SMA and MS approved for PBS listing

Multiple sclerosis

By Michael Woodhead

29 Apr 2021

Positive recommendations have been made for PBS listings of risdiplam for Spinal Muscular Atrophy (SMA) and ofatumumab for multiple sclerosis, by the Pharmaceutical Benefits Advisory Committee (PBAC).

At its March 2021 meeting the PBAC recommended PBS listing of risdiplam (Evrysdi) for patients with SMA Types 1, 2 or 3a who are aged 18 years or under, based on cost effectiveness comparable with nusinersen.

However the committee did not recommend risdiplam for patients with Type 3b SMA or for adults (over 18) patients.

Treatments for MS also received recommendations for PBS listing, including ofatumumab (Kesimpta) for the treatment or relapsing-remitting multiple sclerosis (RRMS).

The PBAC said the cost-effectiveness of ofatumumab would be acceptable if it were cost minimised to the least costly of fingolimod, natalizumab, alemtuzumab, ocrelizumab, cladribine and ozanimod.

A new brand of glatiramer, Glatira 40 mg/mL injection, and the listing of Glatira 20 mg/mL injection, was also recommended for listing on the PBS for the treatment of MS.

There were several neurology treatments that failed to win recommendations for PBS listing at the meeting.

Deutetrabenazine (Austedo) was not recommended as a second-line treatment of chorea associated with Huntingdon’s Disease in patients who have failed prior tetrabenazine treatment. The PBAC said it considered the most appropriate clinical place for deutetrabenazine was first-line treatment as an alternative to tetrabenazine (irrespective of prior or co-therapy with an antipsychotic).

It acknowledged there was a high clinical need for new therapies to assist patients with the wide range of debilitating physical and mental impacts of HD and improve quality of life.

“The Committee considered that whilst deutetrabenazine likely represents an effective treatment option for the treatment of chorea symptoms in people with HD the most appropriate approach for considering its comparative efficacy, safety and cost-effectiveness was to compare it to tetrabenazine, its non-deuterated analogue.”

The PBAC also did not recommend the listing of eslicarbazepine (Zebinix)) for the treatment of intractable partial epileptic seizures, with or without secondary generalised seizures.

The committee said the clinical data and indirect treatment comparison did not support the efficacy of eslicarbazepine compared to placebo, and the claim of non-inferior effectiveness and safety of the treatment compared with lacosamide.

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