Background: Available therapies
for myelofibrosis can exacerbate cytopenias and are not indicated for patients with severe thrombocytopenia.
«Ruxolitinib
for myelofibrosis: Indication of considerable added benefit.»
Currently, the only treatment
for myelofibrosis is Incyte's Jakafi, which generated sales and royalty revenue at an annualized run rate of $ 1.01 billion based on third - quarter results.
Not exact matches
The underlying reason is that Geron and partner Johnson & Johnson (NYSE: JNJ) are developing a first - in - class telomerase inhibitor called imetelstat
for the rare blood disorders myelodysplastic syndromes (MDS) and
myelofibrosis (MF).
Incyte's (NASDAQ: INCY) Jakafi racked up $ 1.1 billion in sales last year as the only targeted therapy
for patients with
myelofibrosis, but a majority of patients who take it eventually relapse.
In preliminary drug - mutation matching studies, they found that JAK1 - mutated SS cells were sensitive to JAK inhibitors, drugs that are currently approved
for treatment of other hematologic cancers such as polycythemia vera and
myelofibrosis.
Ruxolitinib (trade name: Jakavi) has been approved since August 2012
for the treatment of adults with
myelofibrosis.
Ruxolitinib is an option
for patients with so - called primary or secondary
myelofibrosis whose spleen is already enlarged (splenomegaly) or who have other disease - related symptoms.
But especially in the case of advanced
myelofibrosis and systemic mastocytosis, patients may survive only
for several years.
A recent phase III study revealed a benefit with pacritinib
for patients with
myelofibrosis and thrombocytopenia who have experienced treatment failure with previous therapies.
Patients with higher - risk
myelofibrosis (with no exclusions
for baseline anaemia or thrombocytopenia) were randomly assigned (2:1) to receive oral pacritinib 400 mg once daily or best available therapy (BAT) excluding JAK2 inhibitors until disease progression or unacceptable toxicity.
Pacritinib could be a treatment option
for patients with
myelofibrosis, including those with baseline cytopenias
for whom options are particularly limited.