Jazz gets FDA okay for Chimerix brain cancer drug
Jazz Pharmaceuticals has become the first drugmaker to get FDA approval for a treatment for diffuse midline glioma with H3 K27M mutations, a rare brain cancer mainly affecting young people.
The Ireland-domiciled company has won accelerated approval from the US regulator for oral DRD2 antagonist Modeyso (dordaviprone), acquired as part of its $935 million takeover of Chimerix earlier this year. The drug can be used to treat patients aged one or older whose cancer has progressed after prior therapy.
Patients with this form of high-grade glioma face a very difficult prognosis, with few treatment options beyond radiotherapy and palliative care. Disease relapse is virtually inevitable, and until now no systemic therapy has been available. There are estimated to be around 2,000 people diagnosed with H3 K27M-mutated glioma in the US each year.
Jazz is running a confirmatory Phase 3 trial in newly diagnosed, non-recurrent patients, called ACTION, which is expected to generate results next year. If positive, the FDA could grant Modeyso a full license and wider label, including as a first-line therapy.
The company said it will launch Modeyso in the "coming weeks," but has not yet revealed its pricing plans for the drug, taken as a capsule once a week. Chimerix has previously suggested it could have blockbuster potential, despite the small number of eligible patients, based on what it has said would be an 'ultra-orphan' pricing strategy.
There's another potential payoff for Jazz, as the FDA approval comes with a rare paediatric disease priority review voucher (PRV), which could be used to accelerate one of its other programmes or sold to another company. PRVs typically change hands for $100 million or more.
Modeyso has been approved on the back of data from 50 patients in open-label studies that revealed an overall response rate (ORR) of 22% and a median duration of response of 10.3 months and nearly three-quarters of patients (73%) gaining at least an extra six months before disease progression.
Neuro-oncology specialist Patrick Wen of Dana-Farber Cancer Institute and Harvard Medical School called Modeyso's approval a "major turning point" for the field and "meaningful advancement" for patients.
"This approval not only equips clinicians with the first targeted option for this disease, but also signals a meaningful shift in what patients and families can expect after diagnosis," said Joshua Allen, Chimerix's chief scientific officer.
