I don't know what to make of...

I don't know what to make of this


https://endpts.com/inctyes-ruxolitinib-scores-its-second-approval-in-two-days-this-time-in-chronic-graft-versus-host-disease/

September 23, 2021 07:13 AM EDT
FDA+Incyte's ruxolitinib scores its second approval in two days, this time in chronic graft-versus-host disease


A day after approving a topical formulation of Incyte’s cash cow ruxolitinib for atopic dermatitis, regulators are back with more good news for the company’s favorite JAK inhibitor.

The FDA has given Jakafi, the oral version of the drug, the green light to treat chronic graft-versus-host disease after one or two lines of systemic therapy, Incyte said Wednesday, marking the drug’s second approval in just as many days.

GVHD is a condition that occurs after some allogeneic stem cell transplants, in which the donor stem cells view the host as foreign and attack. Jakafi won approval to treat the acute form of the disease back in 2019, which typically occurs within 100 days of the transplant and can cause rashes, discoloration and vomiting. But the chronic form usually comes later than 100 days and can be worse, triggering symptoms like shortness of breath, pain, muscle weakness and more. Both forms can be deadly, and can affect multiple organ systems.

It’s estimated that more than 14,000 people in the US are living with GVHD, and around half don’t respond to steroids, leaving few treatment options.

“This approval represents a significant advancement in the treatment of appropriate patients with chronic GVHD — for both the patients who face a poor prognosis and the healthcare providers who struggle to effectively treat them,” principal investigator Robert Zeiser said in a statement.

In the Phase III REACH3 study, Jakafi achieved an overall response rate of 49.7% at Week 24, compared to 25.6% in a group of patients given the best available therapy (p<0.0001). The ORR after six full treatment cycles was 70% for Jakafi compared to 57% for BAT, according to Incyte.

The most common side effects rated Grade 3 or higher were thrombocytopenia (a low blood platelet count), anemia, neutropenia (lower-than-normal levels of neutrophils, a type of white blood cell), and pneumonia, according to full results published back in July. However, deaths reported as primarily due to chronic GVHD complications and/or its treatment were higher in the Jakafi arm (13.3%, versus 7.9% in the control arm), Incyte said.

Ruxolitinib is Incyte’s greatest revenue stream — bringing in nearly $2 billion in 2020 — and has kept Incyte sailing over the years despite setbacks across their pipeline. It’s also approved for polycythemia vera (PV) in adults who have had an inadequate response to or are intolerant of hydroxyurea, intermediate or high-risk myelofibrosis (MF), and — as of yesterday — mild to moderate atopic dermatitis.

The drug will now go up against Kadmon’s Rezurock, which scored an OK in chronic GVHD back in July — six weeks before its PDUFA date. Upon releasing the first glimpse at pivotal data back in 2019, Kadmon framed their drug as one of a growing number of options patients might use, noting that many of the patients in the trial didn’t respond to Incyte’s Jakafi or AbbVie and J&J’s Imbruvica.