MyelofibrosisErythrocytosisAcutePhlebotomy2022BesremiMyeloproliferative neoplasmSecondaryTesticularThrombosisContraindicationsTherapeuticsLymphomasGVHDSymptomClinicalHydroxyureaRuxolitinibJakafiInterferonAdultsPeripheralKinaseDiseaseDosageInhibitorMechanismCyanocobalaminSubcutaneous injectionEssentialOccurPatients2020Anticancer drugsHydroxocobalaminEfficacyMetabolicDoseBoneMedicallyPrimaryBloodCommon
Myelofibrosis11
- Navtemadlin is under clinical development by Kartos Therapeutics and currently in Phase III for Post-Polycythemia Vera Myelofibrosis (PPV-MF). (pharmaceutical-technology.com)
- According to GlobalData, Phase III drugs for Post-Polycythemia Vera Myelofibrosis (PPV-MF) have an 80% phase transition success rate (PTSR) indication benchmark for progressing into Pre-Registration. (pharmaceutical-technology.com)
- Total patients grew across myelofibrosis (MF), polycythemia vera (PV) and graft-versus-host disease (GVHD). (businesswire.com)
- Intermediate-2 or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis. (unboundmedicine.com)
- Jakafi's (a first-in-class JAK1/JAK2 inhibitor approved for polycythemiavera, myelofibrosis and refractory acute graft-versus-host disease) revenues came in at almost $580 million, up 7% from the year-ago quarter's number. (yahoo.com)
- Earlier in March, the FDA issued a complete response letter for ruxolitinib extended-release (XR) tablets for once-daily use in the treatment of certain types of myelofibrosis, polycythemia vera and graft-versus-host disease. (yahoo.com)
- Statin- and bisphosphonate treatment in patients with the Philadelphia-negative chronic myeloproliferative neoplasms - essential thrombocytosis, polycythemia vera and hypercellular myelofibrosis. (clinicaltrialsregister.eu)
- This has been motivated by the fact that this pathway is a potential target of future agents for the treatment of myeloproliferative disorders such as: polycythemia vera, essential thrombocythemia, and myeloid metaplasia with myelofibrosis. (pharmacologycorner.com)
- I'm Dr Gary Grad, a hematologist-oncologist who specializes in the treatment of myeloproliferative neoplasms such as polycythemia vera, or PV, and myelofibrosis, or MF. (jakafi.com)
- Jakafi treats a pair of bone-marrow diseases, myelofibrosis and polycythemia vera, as well as steroid-refractory acute graft-versus-host disease (GVHD), a complication of stem cell transplants. (thickmarkets.com)
- Ruxolitinib was the first drug approved (2011) to specifically treat patients with the intermediate- or high-risk myelofibrosis, including primary myelofibrosis, postpolycythemia vera myelofibrosis, and postessential thrombocythemia myelofibrosis. (org.in)
Erythrocytosis1
- For the individual in which phlebotomy works well, we typically see that the need for a number of phlebotomies goes down relatively rapidly and that we are inducing an iatrogenic state of iron deficiency to try to control the erythrocytosis that individuals have with polycythemia vera. (targetedonc.com)
Acute2
- The new regulatory nod marked the fourth for Jakafi, and it is the only FDA-approved treatment for two of its indications (acute GVHD and polycythemia vera). (thickmarkets.com)
- According to GlobalData, Phase I drugs for Acute Myelocytic Leukemia (AML, Acute Myeloblastic Leukemia) have a 71% phase transition success rate (PTSR) indication benchmark for progressing into Phase II. (meefro.com)
Phlebotomy4
- Aetna considers therapeutic phlebotomy experimental and investigational for treatment of the following conditions because its effectiveness for these indications has not been established (not an all-inclusive list). (aetna.com)
- Therapeutic phlebotomy may be indicated for hemochromatosis, polycythemia vera, porphyria cutanea tarda, and polycythemia secondary to arterio-venous fistulae, cyanotic congenital heart disease or cor pulmonale. (aetna.com)
- A continued need for phlebotomy well into the management of polycythemia vera can be problematic on a variety of levels. (targetedonc.com)
- Appropriately, they begin on cytoreductive therapy, and that hydroxyurea is indicated for individuals who are tolerating phlebotomy as the sole management of polycythemia vera. (targetedonc.com)
20222
- But who will be the winner of the 2022 drug naming tournament? (fiercepharma.com)
- It was approved by the U.S. Food and Drug Administration ("FDA") in February 2022 and is the only approved treatment for the indication. (drihealthcare.com)
Besremi5
- Besremi (ropeginterferon alfa-2b-njft) (MW, 60 kDa) is an interferon alfa-2b indicated for the treatment of adults with polycythemia vera. (medscape.com)
- BURLINGTON, Mass.--(BUSINESS WIRE) November 12, 2021 --PharmaEssentia Corporation (TPEx: 6446), a global biopharmaceutical innovator based in Taiwan leveraging deep expertise and proven scientific principles to deliver new biologics in hematology and oncology, today announced that the U.S. Food and Drug Administration (FDA) has approved BESREMi® (ropeginterferon alfa-2b-njft) for the treatment of adults with polycythemia vera (PV). (cliniexpert.com)
- BESREMi is an innovative monopegylated, long-acting interferon, which exhibits its cellular effects in polycythemia vera in the bone marrow. (cliniexpert.com)
- We are incredibly proud to deliver on our goal of bringing treatments like BESREMi to the polycythemia vera community where there is clear unmet need for more effective, tolerable and durable treatments to preserve patients' health and well-being," said Ko-Chung Lin, Ph.D., Co-Founder and Chief Executive Officer for PharmaEssentia and inventor of ropeginterferon alfa-2b-njft. (cliniexpert.com)
- Ropeginterferon alfa-2b-njft (Besremi) is approved by the US Food and Drug Administration (FDA) for treatment of adults with PV. (medscape.com)
Myeloproliferative neoplasm2
- In oncology, polycythemia vera is an uncommon myeloproliferative neoplasm (chronic leukemia) in which the bone marrow makes too many red blood cells. (wikipedia.org)
- Polycythemia vera is a chronic myeloproliferative neoplasm characterized by an increase in morphologically normal red cells (its hallmark), but also white cells and platelets. (msdmanuals.com)
Secondary1
- In polycythemia vera, in contrast to the secondary erythrocytoses, the red cell mass increase is often initially masked by an increase in the plasma volume that leaves the hematocrit in the normal range. (msdmanuals.com)
Testicular1
- Ovarian and testicular carcinoma, polycythemia vera. (medicscientist.com)
Thrombosis1
- People with untreated polycythemia vera have a substantial risk of Budd-Chiari syndrome (hepatic vein thrombosis). (wikipedia.org)
Contraindications1
- The medical indication test helps to avoid absolute contraindications. (gewebenetzwerk.de)
Therapeutics3
- The participants critically discussed the need for a precise definition of cancer stem cells, the requirement for new markers and more rapid and tractable in vitro and in vivo assays, and the need to develop drug screening strategies to selectively target cancer stem cells to generate therapeutics for this subpopulation of cells that could be resistant to classic treatments while possessing potent tumor-forming capacity. (aacrjournals.org)
- targeted therapeutics, complete with analysis by indications, stage of development, mechanism of action (MoA), route of administration (RoA) and molecule type. (researchandmarkets.com)
- Silence Therapeutics plc, a biotechnology company, focuses on the discovery and development of novel ribonucleic acid (RNA) therapeutics in hematology, cardiovascular, and other rare and metabolic indications. (financialmodelingprep.com)
Lymphomas1
- Indications Chronic lymphocytic leukemia, lymphomas. (medicscientist.com)
GVHD1
- The U.S. Food and Drug Administration (FDA) issued a complete response letter for ruxolitinib extended-release (XR) tablets for once-daily (QD) use in the treatment of certain types of MF, PV and GVHD. (businesswire.com)
Symptom3
- A classic symptom of polycythemia vera is pruritus or itching, particularly after exposure to warm water (such as when taking a bath), which may be due to abnormal histamine release or prostaglandin production. (wikipedia.org)
- A classic symptom of polycythemia vera (and the related myeloproliferative disease essential thrombocythemia) is erythromelalgia. (wikipedia.org)
- This individual continues on hydroxyurea based on the need for continued phlebotomies, and now he develops progressive pruritus, a common symptom of polycythemia vera. (targetedonc.com)
Clinical6
- Clinical symptoms of polycythemia vera are mostly due to hyperviscosity of blood. (wikipedia.org)
- Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval. (pharmaceutical-technology.com)
- GlobalData's Likelihood of Approval analytics tool dynamically assesses and predicts how likely a drug will move to the next stage in clinical development (PTSR), as well as how likely the drug will be approved (LoA). (pharmaceutical-technology.com)
- FDA concluded that, 'in light of the failed primary analysis and data deficiencies noted, the clinical trial does not by itself support the indication. (formularywatch.com)
- Off-label prescribing is very common, reason being it costs a lot to fund clinical trials to "prove" a drug is effective for an indication, but if a drug is reasonably safe and there is good reason to believe it might work for a problem (e.g. test tube studies or just general physiological plausibility) physicians try it out on patients, especially when no other medications work. (mygotodoc.com)
- Furthermore, quanti- then the clinical indications have carcinogens, Cogliano et al. (who.int)
Hydroxyurea4
- In many ways, this individual now meets the criteria established by the European LeukemiaNet that defines hydroxyurea resistance for an individual with polycythemia vera. (targetedonc.com)
- Jakafi is a prescription medicine used to treat adults with polycythemia vera who have already taken a medicine called hydroxyurea (HU) and it did not work well enough or they could not tolerate it. (jakafi.com)
- Ruxolitinib was approved by FDA (2014) to treat polycythemia vera patients who have an inadequate response to or cannot tolerate hydroxyurea. (org.in)
- This was a phase 3 open-label study to evaluate the efficacy and safety of ruxolitinib versus standard therapy in patients with polycythemia vera who had an inadequate response to or had unacceptable side effects from hydroxyurea. (org.in)
Ruxolitinib3
- In this RESPONSE trial it was shown that ruxolitinib was superior to standard therapy in controlling the hematocrit, reducing the spleen volume, and improving symptoms associated with polycythemia vera and hence it was approved for this indication. (org.in)
- RESPONSE-2 assessed the efficacy and safety of ruxolitinib in controlling disease in patients with polycythaemia vera without splenomegaly (no palpable spleen at enrollment) who need second-line therapy. (org.in)
- Ruxolitinib is the first and only FDA-approved treatment for this indication. (org.in)
Jakafi1
- Our first quarter results demonstrate continued year-over-year double-digit revenue growth driven by Jakafi, which grew across all indications, and Opzelura, which is on track to become one of the most successful dermatology launches in recent years. (businesswire.com)
Interferon1
- With the availability of an FDA-approved, next-generation interferon for this indication, it's time that we focus on preserving the long-term health of patients with polycythemia vera. (cliniexpert.com)
Adults3
- ADULTS: PO Dosage reductions are required if the patient has received full-dose radiation or myelotoxic drugs within the last month or has a low leukocyte or platelet count. (medicscientist.com)
- Hypothesis #1: The PK of understudied drugs in children will differ from adults and within children according to pediatric age groups or special population. (clinicaltrialsgps.com)
- Hypothesis #2: The PD of targeted drugs in children will differ from adults. (clinicaltrialsgps.com)
Peripheral1
- Thus, polycythemia vera is a panmyelosis because of elevations of all 3 peripheral blood components. (msdmanuals.com)
Kinase1
- A mutation in the JAK2 kinase (V617F) is strongly associated with polycythemia vera. (wikipedia.org)
Disease7
- The WHO criteria for polycythemia vera are specifically outlined in Table 4, and emphasis is given to accurate histological observations as proven predictors in the prognosis of the disease. (wikipedia.org)
- Data on the effect of life-span of an individual with treated polycythemia vera is inconclusive due to the rarity of the disease. (wikipedia.org)
- The reality of living with a rare and chronic cancer like polycythemia vera is that it is often underrecognized and the limited treatments available cannot properly address the disease beyond the symptoms. (cliniexpert.com)
- Polycythemia Vera (PV) is a cancer originating from a disease-initiating stem cell in the bone marrow resulting in a chronic increase of red blood cells, white blood cells, and platelets. (cliniexpert.com)
- Voxzogo has consistently been smashing each round and it was no different in its Final Four matchup against Horizon's thyroid eye disease drug Tepezza. (fiercepharma.com)
- It may be possible to treat the underlying disease by surgical correction of anatomic lesions leading to small bowel bacterial overgrowth, expulsion of fish tapeworm, discontinuation of drugs leading to vitamin malabsorption (see Drug/Laboratory Test Interactions ), use of a gluten-free diet in nontropical sprue, or administration of antibiotics in tropical sprue. (nih.gov)
- All orders and care relevant to the patient's underlying disease if applicable for the admission are given by his/her attending physician who are familiar with the patient's medical history, drug history and other such information. (rwjbh.org)
Dosage2
- Drug Dosage: The authors and the publisher have exerted every effort to ensure that drug selection and dosage set forth in this text are in accord with current recommendations and practice at the time of publication. (karger.com)
- However, in view of ongoing research, changes in government regulations, and the constant flow of information relating to drug therapy and drug reactions, the reader is urged to check the package insert for each drug for any changes in indications and dosage and for added warnings and precautions. (karger.com)
Inhibitor1
- The drug is a once-daily, oral JAK inhibitor discovered by Incyte and licensed to LLY. (yahoo.com)
Mechanism2
- Discusses mechanism of action, dosing and target INRs, drug and food interactions, and patient counseling. (pharmacologycorner.com)
- Limited data are available in reference texts regarding the mechanism of action of this drug. (empowerpharmacy.com)
Cyanocobalamin2
- An intradermal test dose is recommended before Cyanocobalamin Injection, USP is administered to patients suspected of being sensitive to this drug. (wikidoc.org)
- There is limited information regarding Cyanocobalamin Postmarketing Experience in the drug label. (wikidoc.org)
Subcutaneous injection1
- In late June, it submitted a new drug application to the FDA for lenacapavir, a potential subcutaneous injection regimen for HIV patients. (thickmarkets.com)
Essential1
- Bomedemstat's most advanced indication is the slow-growing blood cancer essential thrombocythemia. (evaluate.com)
Occur2
- The long-term risks of polycythemia vera (PV) include leukemic and fibrotic transformation, which occur in fewer than 5% and 10% of patients, respectively, at 10 years. (medscape.com)
- cholestatic hepatitis has been found to occur in patients taking both warfarin and ticlopidine (a drug that also helps to prevent blood clots), at the same time. (drugwatch.com)
Patients8
- Patients with polycythemia vera are more likely to have gouty arthritis. (wikipedia.org)
- Such itching is present in approximately 40% of patients with polycythemia vera. (wikipedia.org)
- Untreated, polycythemia vera can be fatal, with the median survival in patients being 1.5-3 years. (wikipedia.org)
- Exercise caution when administering chlorambucil to patients with a history of seizure disorders, head trauma, or to patients receiving other potentially epileptogenic drugs. (medicscientist.com)
- Voxzogo, as many voters pointed out, shouts "to go" and hints at "to grow," which of course is what the drug is designed to help patients do. (fiercepharma.com)
- And 14 patients discontinued the drug, 10 because of adverse events. (evaluate.com)
- While warfarin can be a lifesaver for patients suffering from atrial fibrillation and other conditions that may cause a fatal blood clot, the drug also has its drawbacks. (drugwatch.com)
- Patients on ACE INHIBITORS should be off the drug for 24 hours for plasma exchange to prevent serious complications during the procedure. (rwjbh.org)
20201
- The regulatory agency declined to approve filgotinib in August 2020, citing male fertility concerns associated with the drug as the reason. (thickmarkets.com)
Anticancer drugs1
- Follow procedures for proper handling and disposal of anticancer drugs. (medicscientist.com)
Hydroxocobalamin1
- Although methylcobalamin and vitamin B12 are terms used interchangeably, vitamin B12 is also available as hydroxocobalamin, a less commonly prescribed drug product (see Hydroxocobalamin monograph), and methylcobalamin. (empowerpharmacy.com)
Efficacy1
- The drug was also a political hot potato from the get-go and belief in its efficacy was almost guaranteed in red states and sorely lacking in blue ones. (mygotodoc.com)
Metabolic1
- Hypothesis #3: Genetic polymorphisms in drug metabolizing enzymes and metabolic and proteomic profiles will impact drug exposure in children. (clinicaltrialsgps.com)
Dose2
- AVP-825 ( Avanir Pharmaceuticals ) drug-device combination product consisting of low-dose sumatriptan powder delivered intranasally utilizing a novel Breath Powered delivery technology for the treatment of migraine. (formularywatch.com)
- The drug did not harm fetal monkeys at doses that were 10 times the maximum recommended human dose. (medscape.com)
Bone1
- Blunted or impeded therapeutic response to vitamin B 12 may be due to such conditions as infection, uremia, drugs having bone marrow suppressant properties such as chloramphenicol, and concurrent iron or folic acid deficiency. (drugs.com)
Medically1
- Medically reviewed by Drugs.com. (drugs.com)
Primary2
- Polycythemia vera (PV), being a primary polycythemia (increase in the fraction of volume occupied by red cells in the blood), is caused by neoplastic proliferation and maturation of erythroid, megakaryocytic and granulocytic elements to produce what is referred to as panmyelosis. (wikipedia.org)
- This drug is given by infusion into the butt cheek muscle as coordinated by your primary care physician, generally every 1 to about a month. (axiolabs.com)
Blood3
- Most of the health concerns associated with polycythemia vera are caused by the blood being thicker as a result of the increased red blood cells. (wikipedia.org)
- Taking advantage of procedures done as part of routine medical care (i.e. blood draws) this study will serve as a tool to better understand drug exposure in children receiving these drugs per standard of care. (clinicaltrialsgps.com)
- Polycythemia vera involves increased production of red blood cells (RBCs), white blood cells (WBCs), and platelets. (msdmanuals.com)
Common2
- While the four drugs treat very different conditions, their names bear many common features. (fiercepharma.com)
- The most common indication for surgical intervention in neonatal idiopathic nephrotic syndrome is the presence of hydrocephalus, hyperkalemia, and serum low-density lipoprotein levels.1 These neonates are more difficult to treat surgically. (startthechangenow.com)