Risankizumab Approved – ICER Weighs In

Regulatory Updates

The FDA approved risankizumab (Skyrizi, AbbVie) on 4/23/2019 for the treatment of plaque psoriasis. Risankizumab is dosed every 12 weeks after an initial 2 injections. The drug will compete with ixekizumab, tildrakizumab, guselkumab, and secukinumab. ICER has estimated an annual price of $28,800–$42,100 for risankizumab to achieve a long-term cost-effectiveness benchmark of $100,000– $150,000 per QALY. ICER suggested that step therapy approaches may not be appropriate for psoriasis. In an analysis of current data ICER found that risankizumab may be more effective than TNF-blockers, but this is based on grey literature, since risankizumab studies have not been published. AbbVie announced the annual WAC for maintenance dosing of risankizumab is $59,000.

The FDA accepted the BLA for eptinezumab. Alder forecasts approval in May. If approved in 2019, Alder expects to launch the drug in 1Q20 for migraine prophylaxis. 

Genentech’s Risdiplam was granted PRIME status by the EMA for the treatment of Spinal muscular atrophy.

Celgene and Acceleron submitted a BLA and a MAA for luspatercept in April 2019. The drug will be compared to ESA drugs in non-transfusion-dependent beta-thalassemia and in myelofibrosis.

Biogen has decided not to initiate a Phase III trial of aducanumab in the prevention of Alzheimer’s disease.

Fulcrum Therapeutics licensed losmapimod from GSK in April 2019 and plans to develop the drug as a treatment for facioscapulohumeral muscular dystrophy. Fulcrum has an ongoing trial evaluating losmapimod for the treatment of facioscapulohumeral muscular dystrophy.

Announced Research Updates

Gilead announced that in the 48-week, 803 patient, Phase III, STELLAR-3 trial, treatment with selonsertib 18 mg or 6 mg did not improve liver fibrosis by 1 stage or more without disease worsening compared to placebo in patients with stage 3 fibrosis from NASH.

Published Research Update

In a 24-week, 41 patient, Phase I/II, open label trial, treatment with X4P-001 (X4 Pharmaceuticals) 200 mg BID or 400 mg QD plus axitinib 5 mg BID resulted in an objective response rate of 31.8% in 22 evaluable patients with advanced clear cell renal cell carcinoma.

In the 8-week, 256 patient, Phase IIb, open-label, NEW-HOPE trial, treatment with firibastat (Quantum Genomics) resulted in a 9.5 mmHg decrease in systolic blood pressure and a 4.2 mmHg decrease in diastolic blood pressure in a high-risk diverse hypertensive population.