Ojjaara (momelotinib)
An approved treatment for Myelofibrosis.
The same compound appears under different names depending on the context. Here is how to identify Momelotinib wherever you encounter it, plus the key facts at a glance.
- Generic name
- Momelotinib
- Brand name
- Ojjaara
- Development codes
- CYT387, GS-0387
- Drug class
- JAK1/JAK2 and ACVR1 inhibitor
- Manufacturer
- GSK (acquired with Sierra Oncology in 2022)
- How it's taken
- The dose is 200 mg by mouth once a day, with or without food, with the tablet swallowed whole and not cut, crushed or chewed.
A once-daily tablet approved by the FDA on September 15, 2023 for adults with intermediate or high-risk myelofibrosis who have anemia, including myelofibrosis that follows polycythemia vera or essential thrombocythemia. In a trial of people already treated with a JAK inhibitor, symptoms were cut at least in half in 25% on Ojjaara vs 9% on danazol. In a head-to-head trial against ruxolitinib, the share of anemic patients whose spleen shrank by at least 35% was similar (31% vs 33%).
Where Momelotinib fits
One of 4 JAK inhibitors approved for myelofibrosis, and the only one whose approval is specifically for people with anemia. It was tested head to head against ruxolitinib in SIMPLIFY-1 and against danazol in people previously treated with a JAK inhibitor in MOMENTUM.
How Momelotinib works
Like other JAK inhibitors, momelotinib blocks JAK1 and JAK2, including the mutant JAK2 V617F, which calms the overactive signaling that drives inflammation, an enlarged spleen and symptoms in myelofibrosis. It also blocks ACVR1 (also called ALK2). In myelofibrosis, overactive ACVR1 raises hepcidin, a liver hormone that locks iron away. By blocking ACVR1, momelotinib lowers hepcidin and makes more iron available for making red blood cells, and patients in trials showed a sustained drop in hepcidin over 24 weeks[1].
Mechanism: Oral inhibitor of JAK1, JAK2 (including JAK2 V617F) and ACVR1 (ALK2); blocking ACVR1 lowers the iron-regulating hormone hepcidin, making more iron available for red blood cell production
Side effects and safety
- Infections and hepatitis B reactivation. Serious, including fatal, infections in 13% of patients. Do not start during an active infection. People with hepatitis B are tested before starting.
- Low platelets and neutrophils. New or worsening platelets below 50,000 in 20% of patients and severe neutropenia in 2%. Managed by lowering or pausing the dose.
- Liver toxicity. ALT and AST rose in about a quarter of patients, and 2 of 993 trial patients had reversible drug-induced liver injury. Liver tests at baseline, monthly for 6 months, then as needed.
- Severe skin reactions. Including toxic epidermal necrolysis. Pause Ojjaara and seek care for a severe rash or blistering.
- Heart attack, stroke, blood clots and cancer. Seen with another JAK inhibitor in rheumatoid arthritis. Extra caution in current or past smokers and people with heart risk factors.
- Symptom flare after stopping. Myelofibrosis symptoms can flare after stopping a JAK inhibitor. Do not stop without talking to your doctor; a gradual taper may be advised.
- Complete blood count with platelets before starting and periodically
- Liver tests at baseline, monthly for 6 months, then as needed
- Hepatitis B tests before starting in people with hepatitis B infection
- Watch for signs of infection
Ojjaara has no boxed warning and no contraindications. In MOMENTUM, the most common side effects on Ojjaara vs danazol were low platelets (28% vs 17%), diarrhea (22% vs 9%), bleeding (22% vs 18%), fatigue (21% vs 20%) and nausea (16% vs 9%). In the anemic patients in SIMPLIFY-1, the most common vs ruxolitinib were dizziness (24% vs 15%), fatigue (22% vs 25%), bacterial infection (21% vs 12%), bleeding (21% vs 18%), low platelets (21% vs 34%), diarrhea (20% vs 20%) and nausea (20% vs 3%). Serious infections, some fatal, occurred in 13% of patients, and hepatitis B can reactivate, so people with hepatitis B are tested and managed before starting. New or worsening rises in the liver enzymes ALT and AST occurred in 23% and 24% of patients, so liver tests are done at baseline, monthly for 6 months and then as needed. Other warnings cover low platelet and neutrophil counts, severe skin reactions including toxic epidermal necrolysis, and, based on another JAK inhibitor, heart attack and stroke, blood clots and cancers[1].
This is not a complete list of side effects. Talk to your doctor or pharmacist about what to expect and when to seek medical attention.
Taking Momelotinib
The dose is 200 mg by mouth once a day, with or without food, with the tablet swallowed whole and not cut, crushed or chewed. A missed dose is skipped and the next one taken the following day. People with severe liver impairment (Child-Pugh C) start at 150 mg a day. For low platelets, low neutrophils, liver problems or other serious side effects, the dose is paused or lowered in 50 mg steps, and it is stopped if 100 mg a day is not tolerated. With Ojjaara, rosuvastatin should start at 5 mg and not go above 10 mg a day[1].
Availability and cost
Only available as the brand-name product.
Help paying for Ojjaara
Pick your insurance to see which help fits. Drugmaker copay cards can't be used with Medicare, Medicaid or TRICARE; charity funds are the usual route there.
- Copay help
OJJAARA Copay Program: eligible commercially insured patients may pay as little as $0. Requires US residence and no eligibility for or enrollment in a government-funded program.
For: private insurance · source - Free medicine program
GSK Patient Assistance Program, run by the GSK Patient Access Programs Foundation, may provide Ojjaara free to uninsured patients, commercially insured patients whose plan still does not cover it after appeal, and Medicare drug plan members who cannot afford it and are not in Extra Help. Household income limits apply. Not for people with Medicaid, VA, DoD or TRICARE coverage.
For: no insurance, underinsured, Medicare · source - Bridge or quick-start supply
Quick Start helps with delays filling the first prescription, and the Bridge program helps commercially insured patients already on treatment through coverage interruptions.
For: private insurance · source - Insurance and case manager help
Together with OJJAARA support at 1-844-4GSK-ONC (1-844-447-5662), Monday to Friday, 8 AM to 8 PM ET.
The official page does not say who qualifies. Ask the program. · source
Good to know: The annual copay maximum could not be confirmed on a live GSK page (a figure appears only in search listings of a retired GSK page), so it was left out. The GSK Patient Access Programs Foundation describes itself as separate from GSK; its Medicare page is https://gskpaf.org/gsk/prescription-medicine-patient-assistance/ojjaara-medicare/.
- From a charity · Blood Cancer United (formerly The Leukemia & Lymphoma Society)Clinical Trials Co-Pay Fund (all blood cancers) fundOpen
Pays for: Insurance premiums and treatment-related copays, deductibles and coinsurance, up to $3,500 per year. Requires health insurance (any kind).
- From a charity · Blood Cancer United (formerly The Leukemia & Lymphoma Society)Patient Aid Program fundOpen
Pays for: One-time $100 stipend for non-medical expenses (transportation, food, housing, utilities); no income or insurance requirement, up to $100 per year.
The foundation says: “CURRENT FUND STATUS: Open. Fund is currently Open.” - From a charity · CancerCare Co-Payment Assistance FoundationMyeloproliferative Neoplasms fundOpen
Pays for: Copays, coinsurance and deductibles for treatment, up to $7,000 per year. Requires Medicare, Medicaid or TRICARE.
The foundation says: “Status: Open. Grant Amount: $7,000 (Initial Grant Amount $7,000; Program CAP Amount $10,000)” - From a charity · TotalAssist (formerly PAN Foundation)Myeloproliferative Neoplasms fundOpen
Pays for: Out-of-pocket costs for approved medications, up to $9,500 per year. Requires health insurance (any kind).
- From a charity · The Assistance FundMyeloproliferative Neoplasms (MPN) fundWaitlist
Pays for: Copays, coinsurance, deductibles and other health-related expenses.
The foundation says: “WAITLIST — Accepting Waitlist Patients. TAF is currently accepting requests to join the enrollment waitlist for this program. Waitlists a…”
Access and eligibility
Approved for adults with intermediate or high-risk myelofibrosis (primary, or after polycythemia vera or essential thrombocythemia) who have anemia. Safety and effectiveness in children have not been established. The main trials required platelets of at least 25,000 (MOMENTUM) or 50,000 (SIMPLIFY-1). Breastfeeding is not advised during treatment and for at least 1 week after the last dose, and women who could become pregnant should use highly effective contraception during treatment and for at least 1 week after.
Source: Together with OJJAARA
Access program details are provided for informational purposes and may vary based on insurance coverage, geographic location, and individual circumstances. Confirm current eligibility directly with the manufacturer or your specialty pharmacy.
Clinical trial results
MOMENTUM (NCT04173494) randomized 195 symptomatic, anemic adults with myelofibrosis who had already taken a JAK inhibitor to Ojjaara or danazol (2:1) for 24 weeks. Symptom scores fell by at least 50% in 25% on Ojjaara vs 9% on danazol. Transfusion independence (no transfusions and no hemoglobin below 8 g/dL in weeks 12 to 24) was reached by 30% vs 20%, meeting the goal of being no worse than danazol, and spleen volume fell by at least 35% in 22% vs 3%[1][8]. SIMPLIFY-1 (NCT01969838) compared Ojjaara with ruxolitinib in 432 people who had not taken a JAK inhibitor. Among the 181 with anemia, spleen volume fell by at least 35% in 31.4% vs 32.6%, and the label notes a numerically lower symptom response on Ojjaara (25% vs 36%)[1][7].
Development history
Momelotinib was discovered at Cytopia, an Australian biotechnology company, where it carried the code CYT387. YM BioSciences acquired Cytopia in 2011, Gilead acquired YM in 2013, and Sierra Oncology acquired momelotinib from Gilead in August 2018[5][6]. GSK completed its acquisition of Sierra Oncology on July 1, 2022[4]. The FDA approved Ojjaara on September 15, 2023, and GSK described it as the first and only treatment indicated for myelofibrosis patients with anemia[2][3].
Explore Myelofibrosis trials
Other Myelofibrosis treatments
The FDA has approved 4 JAK inhibitors for myelofibrosis: ruxolitinib (Jakafi, November 2011), fedratinib (Inrebic, August 2019), pacritinib (Vonjo, February 2022) and momelotinib (Ojjaara, September 2023). Vonjo's approval is for people with platelets below 50,000 per microliter, and Ojjaara's is for people with anemia.
Common questions about Momelotinib
▸What is Momelotinib (Ojjaara)?
A once-daily tablet approved by the FDA on September 15, 2023 for adults with intermediate or high-risk myelofibrosis who have anemia, including myelofibrosis that follows polycythemia vera or essential thrombocythemia. In a trial of people already treated with a JAK inhibitor, symptoms were cut at least in half in 25% on Ojjaara vs 9% on danazol. In a head-to-head trial against ruxolitinib, the share of anemic patients whose spleen shrank by at least 35% was similar (31% vs 33%).
▸How does Momelotinib work?
Like other JAK inhibitors, momelotinib blocks JAK1 and JAK2, including the mutant JAK2 V617F, which calms the overactive signaling that drives inflammation, an enlarged spleen and symptoms in myelofibrosis. It also blocks ACVR1 (also called ALK2). In myelofibrosis, overactive ACVR1 raises hepcidin, a liver hormone that locks iron away. By blocking ACVR1, momelotinib lowers hepcidin and makes more iron available for making red blood cells, and patients in trials showed a sustained drop in hepcidin over 24 weeks[1].
▸What are the side effects of Momelotinib?
Ojjaara has no boxed warning and no contraindications. In MOMENTUM, the most common side effects on Ojjaara vs danazol were low platelets (28% vs 17%), diarrhea (22% vs 9%), bleeding (22% vs 18%), fatigue (21% vs 20%) and nausea (16% vs 9%). In the anemic patients in SIMPLIFY-1, the most common vs ruxolitinib were dizziness (24% vs 15%), fatigue (22% vs 25%), bacterial infection (21% vs 12%), bleeding (21% vs 18%), low platelets (21% vs 34%), diarrhea (20% vs 20%) and nausea (20% vs 3%). Serious infections, some fatal, occurred in 13% of patients, and hepatitis B can reactivate, so people with hepatitis B are tested and managed before starting. New or worsening rises in the liver enzymes ALT and AST occurred in 23% and 24% of patients, so liver tests are done at baseline, monthly for 6 months and then as needed. Other warnings cover low platelet and neutrophil counts, severe skin reactions including toxic epidermal necrolysis, and, based on another JAK inhibitor, heart attack and stroke, blood clots and cancers[1].
▸How is Momelotinib taken?
The dose is 200 mg by mouth once a day, with or without food, with the tablet swallowed whole and not cut, crushed or chewed. A missed dose is skipped and the next one taken the following day. People with severe liver impairment (Child-Pugh C) start at 150 mg a day. For low platelets, low neutrophils, liver problems or other serious side effects, the dose is paused or lowered in 50 mg steps, and it is stopped if 100 mg a day is not tolerated. With Ojjaara, rosuvastatin should start at 5 mg and not go above 10 mg a day[1].
▸Is Momelotinib FDA approved?
Yes, Momelotinib (Ojjaara) is FDA approved (2023) for the treatment of Myelofibrosis.
▸How is Ojjaara different from Jakafi?
Both block JAK1 and JAK2, but momelotinib (Ojjaara) also blocks ACVR1, which lowers hepcidin and frees iron for red blood cell production. Ojjaara is taken once a day and is approved specifically for myelofibrosis with anemia. In SIMPLIFY-1, a head-to-head trial, anemic patients had similar spleen responses on Ojjaara and ruxolitinib (Jakafi) (31% vs 33%), with a numerically lower symptom response on Ojjaara (25% vs 36%).
▸Does Ojjaara help anemia in myelofibrosis?
In MOMENTUM, among anemic patients previously treated with a JAK inhibitor, 30% on Ojjaara were transfusion independent in weeks 12 to 24 vs 20% on danazol, and 35% vs 17% needed no transfusions during the 24 weeks. Results vary from person to person, and your hematologist will track hemoglobin and transfusion needs.
▸What blood tests are needed on Ojjaara?
The label calls for a complete blood count with platelets before starting and periodically, liver tests at baseline, monthly for the first 6 months and then as needed, and hepatitis B tests before starting in people with hepatitis B infection.
Sources and references
Every factual claim on this page is drawn from the public sources listed below. Click any reference to open the original document.
- U.S. National Library of Medicine, DailyMed. OJJAARA (momelotinib) tablets, for oral use: prescribing information. https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=4a672fd1-eb7b-4aa9-9b23-845e4b5dc400
- U.S. Food and Drug Administration · 2023-09-15. NDA 216873 approval letter. https://www.accessdata.fda.gov/drugsatfda_docs/appletter/2023/216873Orig1s000ltr.pdf
- GSK · 2023-09-15. Ojjaara (momelotinib) approved in the US as the first and only treatment indicated for myelofibrosis patients with anaemia. https://www.gsk.com/en-gb/media/press-releases/ojjaara-momelotinib-approved-in-the-us-as-the-first-and-only-treatment-indicated-for-myelofibrosis-patients-with-anaemia/
- GSK · 2022-07-01. GSK completes acquisition of Sierra Oncology. https://www.gsk.com/en-gb/media/press-releases/gsk-completes-acquisition-of-sierra-oncology/
- Sierra Oncology (SEC filing) · 2018-08-22. Sierra Oncology acquires momelotinib from Gilead (Form 8-K exhibit). https://www.sec.gov/Archives/edgar/data/1290149/000119312518254453/d612613dex991.htm
- ClinicalTrials.gov. A Study of Momelotinib Versus Danazol in Symptomatic and Anemic Myelofibrosis Participants (MOMENTUM). https://clinicaltrials.gov/study/NCT04173494
- ClinicalTrials.gov. Momelotinib Versus Ruxolitinib in Subjects With Myelofibrosis (SIMPLIFY-1). https://clinicaltrials.gov/study/NCT01969838
- The Lancet · 2023. Momelotinib versus danazol in symptomatic patients with anaemia and myelofibrosis (MOMENTUM): results from an international, double-blind, randomised, controlled, phase 3 study. https://doi.org/10.1016/S0140-6736(22)02036-0
- U.S. Food and Drug Administration · 2011-11-16. NDA 202192 approval letter (Jakafi for myelofibrosis). https://www.accessdata.fda.gov/drugsatfda_docs/appletter/2011/202192s000ltr.pdf
- U.S. Food and Drug Administration · 2019-08-16. NDA 212327 approval letter (Inrebic). https://www.accessdata.fda.gov/drugsatfda_docs/appletter/2019/212327Orig1s000ltr.pdf
- GSK. Together with OJJAARA patient support. https://www.togetherwithgsk.com/ojjaara/patient/