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Momelotinib Targeted Therapies Treatments

FDA Approves Momelotinib

FDA Approves Momelotinib (Ojjaara) for Myelofibrosis Patients with Anemia

Benefits and Considerations: Navigating Momelotinib’s Usage with Dr. Mascarenhas

In a significant development, the US Food and Drug Administration (FDA) has granted approval to OJJAARA (momelotinib) for the treatment of intermediate or high-risk myelofibrosis (This includes cases of primary myelofibrosis and secondary myelofibrosis occurring after polycythemia vera and essential thrombocythemia) in adults with anemia, according to a media release by GSK.

Myelofibrosis, a rare blood cancer affecting approximately 25,000 patients in the US, often leads to severe complications such as anemia, constitutional symptoms, and splenomegaly. About 40% of patients already have moderate to severe anemia at the time of diagnosis, and nearly all patients are estimated to develop anemia during the course of their disease, as reported by the American Cancer Society.

Dr. John Mascarenhas is a myelofibrosis specialist at Mount Sinai.

With the FDA approval of momelotinib, myelofibrosis patients likely have questions about its usage and possible benefits of the drug.

Dr. John Mascarenhas (Mount Sinai), an expert in myelofibrosis treatment, highlights the unique features of momelotinib: “Momelotinib is a JAK inhibitor so it’s much like ruxolitinib (Jakafi) except there are some nuances that make these drugs a little bit different,” he says. “It also inhibits ACVR1, which is another pathway that regulates iron availability for red blood cell production.”

The journey of momelotinib to FDA approval involved the MOMENTUM study. While initially expected to be approved in late June, it faced a delay for re-review by the FDA, as reported by GSK.

Regarding potential benefits and considerations in momelotinib’s use, Dr. Mascarenhas explains, “Momelotinib is a JAK inhibitor so it’s much like ruxolitinib (Jakafi) except as I mentioned before, there are some nuances that make these drugs a little bit different. It also inhibits ACVR1, which is another pathway that regulates iron availability for red blood cell production. In its long development history, it’s differentiated itself from other drugs in large part by its ability to improve hemoglobins in a subset of patients.”

In terms of treatment, Dr. Mascarenhas emphasizes the importance of personalized care: “I encourage patients to discuss with their physicians if that drug might make sense for them or any of the other drugs that we’ve talked about, whether it’s fedratinib, ruxolitinib, or pacritinib. As we’ve said, it really has to be tailored to the patient.”

In conclusion, the FDA’s approval of momelotinib marks a significant step forward in addressing the challenges faced by myelofibrosis patients, especially those with anemia. Dr. Mascarenhas’s insights provide a deeper understanding of the drug’s unique attributes and its potential to benefit those affected by this blood cancer.

The Latest Myelofibrosis Clinical Trials

If you’re interested in learning more about the latest myelofibrosis clinical trials, explore our conversation with Myelofibrosis experts Dr. Mascarenhas and Dr. Tania Jain (Johns Hopkins Medicine), and Clinical Trial Nurse Ashley Giacobbi (The Leukemia & Lymphoma Society) as they explain cutting-edge therapies.

But there are an unprecedented number of clinical trials that, again, if a patient is out there and has myelofibrosis and things are not doing well, or their spleen is too large, or they’re not feeling well, or they’re having side effects from medicines, one or more of these clinical trials may be an option for them.

Dr. Ruben Mesa |MF Clinical Trials

Myelofibrosis Patient Stories

Stacy S.

Stacy S., Myelofibrosis



Symptoms: Fatique, cold hands and feet
Treatments: Agrylin (for thrombocythemia), Ruxolitinib (Jakafi), Fedratinib (INREBIC), stem cell transplant
Ruth R., Myeloproliferative Neoplasm (MPN)Symptoms: Anemia, bleeding Treatments: Chemotherapy, clinical trial
Natalia's Myelofibrosis Story
Natalia A., Myelofibrosis Symptoms: Anemia, fatigue, weakness, shortness of breath Treatments: Phlebotomies, iron pills, blood transfusion

Mary L., Myelofibrosis



Symptoms: Fatigue, extreme dizziness (later diagnosed as vertigo)
Treatments: Pegasys, hydroxyurea (current)
Kristin D.

Kristin D., Myelofibrosis



Symptoms: None; caught at routine blood work
Treatment: Stem cell transplant
Joseph C. feature profile

Joseph C., Myelofibrosis



Symptoms: None; caught at routine blood work
Treatment: Clinical trial: VONJO (pacritinib)

Jeremy S., Myeloproliferative Neoplasm



Concurrent Diagnoses: Polycythemia vera (PV) & Chronic Lymphocytic Leukemia (CLL)

Holly S., Myelofibrosis



Symptoms: Severe fatigue, throbbing pain in left calf, significant weight loss, itching and rashes, bruising, and shortness of breath

Treatments: Oral chemotherapy (hydroxyurea), immunotherapy injections (peginterferon)
Doug A. feature profile

Doug A., Myelofibrosis



Symptom: Fatigue
Treatments: ruxolitinib, selinexor (clinical trial)
Cathy T. feature profile

Cathy T., Myelofibrosis



Symptoms: None; caught at a routine blood test
Treatment: Stem cell transplant
Ben H.

Ben H., Myelofibrosis



Symptoms: None; caught at a routine blood test
Treatments: Hydroxyurea & aspirin, ruxolitinib
Andrew SchorrDiagnosis: Myelofibrosis, Chronic Lymphocytic Leukemia (CLL)Treatment: Clinical trial, Gazyva, Jakafi, Increbic, Reblozyl and steroids
Andrea S. feature profile

Andrea S., Myelofibrosis



Symptoms: Fatigue, anemia
Treatments: Targeted therapy (JAK inhibitor), blood transfusions, allogeneic stem cell transplant

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