FDA Approves Siponimod – ICER weighs in and April PDUFA Dates

The FDA approved siponimod (Mayzent, Novartis) on 3/26/2019 to treat adults with relapsing multiple sclerosis. In a draft review ICER felt that compared to best supportive care siponimod reduced the risk of disability progression and decreased inflammation. ICER analysis found that siponimod lowered the risk of relapse by 46% and had an annualized relapse rate of 0.07/year compared to 0.16/year with placebo. Due to the lack of comparative trials with other disease modifying therapies and the difference in patient populations and outcome assessments with current trials, ICER analysts did not feel enough data existed to compare siponimod to those therapies. ICER estimated a quality-adjusted life year (QALY) of $826,000 for use of siponimod over best supportive care in the treatment of secondary progressive multiple sclerosis, assuming the same price as ocrelizumab. The cost per QALY dropped to $396,000 in patients with relapses within two years of the initiation of treatment. ICER reported an annual acquisition price for ocrelizumab of $64,308 after discounts. Novartis has announced an annual WAC of $88,000 for  siponimod. There have been no estimates or reports of potential discounts for siponimod, during the week the drug was approved. ICER will accept comments on the draft report until April 10 and then release a final report on May 2 with a discussion of the report set for May 23.

March was a busy review month for the FDA, but in April, there is only one PDUFA deadline. Risankizumab (Skyrizi, Boehringer Ingelheim and AbbVie) is an interleukin-23 inhibitor being developed for the treatment of Psoriasis and Crohn’s disease. The drug is an Orphan Drug and has a PDUFA date of 4/25/2019. Risankizumab will compete with other IL-23-targeting drugs (ustekinumab, tildrakizumab, guselkumab), IL-17 blockers (secukinumab) and TNF-blockers (adalimumab, etanercept). The drug is given as a subcutaneous injection every three months. The NDA is based on four Phase III trials. In two trials 75% of risankizumab patients achieved Psoriasis Area and Severity Index (PASI 90) compared to 42 to 48% with ustekinumab and 2 to 5% with placebo. In a third 72% of risankizumab patients achieved PASI 90 compared to 47% with adalimumab. The fourth trial was a withdrawal and re-treatment trial where 73% of risankizumab patients achieved PASI 90 compared to 2% with placebo.